May flashback: NICE paved the way for Ocrevus owing to the new lower price, recommended Spiranza for the treatment of spinal muscular atrophy
NICE has recommended nusinersen for the treatment of children with spinal muscular atrophy. Biogen markets Spiranza.
Patients with SMA usually die before the age of 2. Currently, there are no medications available for the treatment; only supportive care was possible to minimize the disabilities, improve quality of life, and address complications.
The prevalence of SMA in the UK is around 600 to 1200 children.
Earlier NICE was unable to make the recommendation because of the high cost of the product and uncertainties with the lost term cost.
On the other hand, NICE has recommended ocrelizumab (Ocrevus, Roche) for treating the patients with primary progressive multiple sclerosis (PPMS) in adults. The approval follows the commercial agreement between the company and NHS, which makes the product available at a lower price.
Celgene’s Revlimid in combination with rituximab is indicated for the treatment of previously treated follicular lymphoma and marginal zone lymphoma
Celgene has today announced that Revlimid has approved for the treatment of follicular lymphoma and marginal zone lymphoma in the combination of rituximab following priority review designation. This combination is the first chemotherapy-free combination indicated for the treatment of follicular lymphoma and marginal zone lymphoma who did not respond to the previous treatment.
The approval is based on the Phase 3 AUGMENT study comparing the combination of Revlimid and rituximab in combination with placebo and rituximab combination. The primary endpoint is progression-free survival. PFS was 39.4 months for patients treated with Revlimid and rituximab combination versus 14.1 months treated with rituximab and placebo. Overall survival is 16 in Revlimid + rituximab vs. 26 deaths in placebo +rituximab.
The approval is under consideration by the European Medicines Agency in Europe.
GSK announced the approval of Shingrix in China; vaccine indicated to prevent shingles
GSK has announced that National Medical Products Administration (NMPA) has approved Shingrix. The product is recombinant subunit adjuvanted vaccine intended for the prevention of shingles in adults of age more than 50 years.
Earlier the product was included in the list of 48 ‘clinically urgently needed new medicines’ in China.
Varicella-zoster virus reactivation causes shingles, which remains dormant in the nervous system and gets activated with the advancement of age. As per the company, there are around three million people affected with shingles in China.
The approval is based on Phase 3 clinical trial involving 38,000 people. ZOE-50 and ZOE-70 were the two trials in which the product has showed efficacy in more than 90% of patients; efficacy sustained for four years. Adverse events include redness and pain at the site of injection.
Most expensive gene therapy of Novartis was approved by the US FDA
Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of death in infants was approved by the United States Food and Drug Administration (FDA).
Survival motor neuron 1 mutation leads to spinal muscular atrophy (SMA). The gene codes for survival motor neuron (SMN) protein, the protein which is critical for the functioning and maintenance of motor neurons. Lack of the protein leads to the death of motor neurons causing death.
The product is indicated for the treatment of children of less than 2 years of age. The product is based on adeno-associated virus vector-based gene therapy.
The approval of the drug is based on the clinical trial involving 36 pediatric patients with infantile SMA. The primary evidence was obtained from the ongoing trial involving 21 patients. Considering the history, the patients who received Zolgensma developed major developments in the motor milestones (head control and ability to sit). Side effects include elevated liver enzymes and vomiting.
Earlier the product has received the breakthrough designation, fast-track designation and orphan designation from FDA.
UCB’s Nayzilam nasal spray was approved by US FDA for intermittent, stereotypic episodes of frequent seizure
Nayzilam is a benzodiazepine indicated for the treatment of intermittent, stereotypic episodes of frequent seizure in the patients' age of 12 and more. Patients can take the midazolam without the presence of healthcare professionals. This is the first nasal option available for patients in the United States.
There are around 150,000 people in the USA with uncontrolled epilepsy. This will be helpful for those patients who do not prefer other treatments.
The approval is based on the single study consisting of test dose phase and comparative phase. In the test dose phase, two 5 mg doses were administered and were checked for adverse events. The patients who had no adverse events were considered for comparative phase. 201 patients were considered for comparative phase. Blinded doses of Nayzilam and placebo were administered. If seizures were re-occurred, Nayzilam was administered in both the arms. The primary endpoint is the termination of seizures within 10 minutes and no seizures in the next 6 hours. Termination fo seizures within 10 minutes are 80.6 versus 70.1%. Absence of reoccurrence is 58.2 versus 37.3%.
SAMSUNG BIOEPIS’S HUMIRA BIOSIMILAR WAS APPROVED IN AUSTRALIA
Samsung Bioepis’s Humira Biosimilar Hadlima was approved by the Therapeutic Goods and Administration in Australia.
Samsung Bioepis’s has requested the approval of all the indications for Humira. Later, the sponsor has restricted the removal of other indications during the evaluation process.
The approval is based on Phase 3 52 weeks trial. The study compared the safety and efficacy of Hadlima with Humira. The studies were conducted in accordance with the European and United States guidelines.
Hadlima was available as Imraldi in Europe. It is under the evaluation process in Canada and was not submitted to the US FDA in the USA.
AbbVie’s cancer product Depatux-M (Depatuxizumab Mafodotin) did not reach the primary endpoint in the patients suffering from Glioblastoma
The pipeline has to perform better for AbbVie to overcome the revenues as sales erosion of Humira is increasing. However, Depatux-M troubled AbbVie’s pipeline. Depatux-M is late-stage product indicated for glioblastoma showed no efficacy.
Abbvie has released a press statement confirming that its product Depatuxizumab Mafodotin has not reached the primary endpoint in Phase 3 clinical trial. The drug is indicated for the patients suffering from glioblastoma which has EGFR (epidermal growth factor receptor) amplification. Phase 3 is INTELLANCE-1, conducted in collaboration with RTOG Foundation. Independent data monitoring committee has confirmed that the primary endpoint has not reached in the trial and suggested to end the trial as there was no survival benefit. The enrolment in the trial was halted.
Phase 3 study which compared the Depatux-M versus placebo, which is administered along with radiation and temozolomide in patients who are newly diagnosed EGFR-amplified GBM. The main efficacy endpoint was overall survival. The safety and efficacy were not approved by the regulators for the approval.
Celgene’s announced the European Commission approval for Revlimid and Imnovid combination for the treatment of Triplet Combination Regimens for Patients with Multiple Myeloma
New triplet combination was of Revlimid and Imnovid was approved by the European Commission for the treatment of patients with multiple myeloma.
As per the approval, Revlimid in combination with bortezomib and dexamethasone was indicated for the treatment of patients with multiple myeloma as first-line treatment.
Imnovid in combination with bortezomib and dexamethasone was indicated for the treatment of patients with multiple myeloma as first-line therapy.
First-line therapy was important in the treatment of multiple myeloma as later lines of treatment has less fast remission.
The approval is based on SWOG S0777 and OPTIMISMM
Phase 3 study compared Revlimid, bortezomib and dexamethasone combination was compared with lenalidomide and dexamethasone alone in a newly diagnosed with multiple myeloma (ndMM) who were not intending on immediately receiving ASCT. Patients have indicated both the treatments in the ratio of 1:1. The main efficacy endpoint was progression-free survival. It was 42 months in Revlimid, Bortezomib and dexamethasone combination where it was 30 months in Lenalidomide and dexamethasone combination. Complete response was observed within 52 months in Revlimid, bortezomib, and dexamethasone whereas it was 38 months in lenalidomide and dexamethasone combination.
Phase 3 study compared pomalidomide in combination with bortezomib and dexamethasone with lenalidomide and dexamethasone alone. The target population was patients with an early line of therapy in patients with relapsed and refractory multiple myeloma. Patients were randomized for 1:1 to receive both the treatments. 11.2 progression-free survival was reported in pomalidomide in combination with bortezomib and dexamethasone and 7.1 months in bortezomib and dexamethasone. Neutropenia, thrombocytopenia, and infections were the adverse events observed.
May 14, 2019
AbbVie’s Venclexta get the US FDA approval for chemotherapy free treatment of Chronic Lymphocytic Leukemia in first line
The application was reviewed under Oncology Priority Review Program
This is the fourth approval for Venclexta and fifth breakthrough designation
AbbVie has announced that Venclexta in combination with obinutuzumab for the treatment of the chemotherapy-free treatment of Chronic Lymphocytic Leukemia in the first line. The product has received the breakthrough designation and early submission was based on the Real-Time Oncology Review (RTOR) pilot program.
The approval is based on the CLL14 trial in which the combination of Venclexta plus obinutuzumab compared to patients who received chlorambucil plus obinutuzumab. The patient arm receiving the Venclexta plus obinutuzumab reduced the risk of disease progression by 67% compared with chlorambucil plus obinutuzumab combination.
49% of the patients taking Venclexta plus obinutuzumab showed severe adverse events including febrile neutropenia and pneumonia. The other adverse events reported were neutropenia (60%), diarrhea (28%), fatigue (21%), nausea (19%), anemia (17%), and upper respiratory tract infection (17%).
May 15, 2019
FDA approved Pfizer’s Fragmin for the treatment potentially life-threatening blood clots for pediatric patients
The United States Food and Drug Administration approved Pfizer’s Fragmin, a blood-thinner in order to reduce the recurrence of symptomatic venous thromboembolism. The product is indicated for children of one month and older.
VTE includes deep vein thrombosis and pulmonary embolism which leads to the death of the patient.
Venous catheter, cancer, infection, congenital heart disease, and trauma or surgery are some of the conditions which lead to VTE.
Fragmin was a heparin product approved by the US FDA in 1994.
The current approval is based on the study in 38 pediatric patients with symptomatic deep vein thrombosis and/or pulmonary embolism. At the end of the study, 21 patients achieved resolution, 7 showed regression, 2 patients had no change in the disease, no disease progression in any patients and 1 experienced recurrence.
The common side effects with the product include hemorrhage, thrombocytopenia, hematoma, or pain at the site of injection.
May 13, 2019
Humira patent litigation was resolved between AbbVie and Boehringer Ingelheim
Humira has announced that the patent litigation was resolved between AbbVie and Boehringer Ingelheim. Based on the resolution, AbbVie will provide a non-exclusive license to BI in the United States.
Biosimilar Cyltezo will be available from July 1, 2023. BI will pay royalties for AbbVie. Earlier AbbVie has sued the BI for infringing more than 70 patents in 2017 during the development of adalimumab biosimilar.
BI follows in the line of Coherus, Pfizer, Amgen, Mylan, Samsung Bioepis, Sandoz, Fresenius Kabi, and Momenta which got the rights for adalimumab biosimilar.
Amgen’s product Amjevita will be the first biosimilar that will be available in the United States market. It will be available from January 2023
FDA approved Jacobus Pharmaceutical’s Ruzurgi for the treatment of Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder
The United States Food and Drug Administration has approved Ruzurgi (amifampridine) in children of age from 6 years to 17 years for the treatment of Lambert-Eaton myasthenic syndrome (LEMS).
LEMS is an autoimmune disorder where the connection between the nerves and muscles is lost leading to weakness. In patients with LEMS, body immune systems attack the neuromuscular junction, leading to disruption in the nerve signals to the muscles.
LEMS is a rare disease with the prevalence in children was not established.
The approval is based on the double-blind, placebo-controlled withdrawal study in 32 adults. After taking Ruzurgi for 3 months, patients switched to placebo. The effectiveness is measured in terms of time the patient took to rise from the chair, walk three meters, get back to the chair for three consecutive times without a break. The scores were less in patients who switched to placebo.
Earlier the product received Priority review and Fast Track designation for the product. The product has also received the Orphan designation.
May 08, 2019
EMA’s CHMP given a positive opinion on Lorviqua for the treatment of previously treated ALK-positive advanced non-small cell lung cancer in adult patients
European Commission has granted conditional approval for Loqviqua for the treatment of anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer as a monotherapy. The drug is indicated in patients whose disease has progressed after alectinib or ceritinib as first ALK tyrosine kinase inhibitor therapy. Loqviqua is a third-generation ALK tyrosine kinase inhibitor, which can penetrate the blood-brain barrier in spite of the resistant mutations.
The approval is based on multi-center Phase 1/2 study, B7461001. The study included 139 patients with ALK-positive advanced NSCLC, who had at least one second-generation ALK tyrosine kinase inhibitor (alectinib, brigatinib or ceritinib). The overall response rate was 42.9% for patients treated with one ALK tyrosine kinase inhibitor, 39.6% in patients who had two or more ALk tyrosine kinase inhibitors.
According to the conditional approval, Pfizer has to submit the Phase 3 CROWN data and additional information from post-marketing studies. In CROWn studies, Lorviqua is compared with crizotinib for the treatment of patients with ALK-positive NSCLC in the first line.
May 07, 2019
Pfizer acquires Therachon, a clinical stage biotech company
Therachon is developing therapies against rare gastrointestinal and musculoskeletal disorders, including developing first in class achondroplasia, a genetic condition which is a common form of short-limb dwarfism.
Pfizer has acquired all the shared of Therachon Holding AG which is developing therapies for achondroplasia and short bowel syndrome (SBS). Pfizer has paid $340 million upfront and pays $470 million while achieving the key milestones.
TA-46 is the leading compound, is soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy. The mechanism of action is believed to normalize the overactive FGFR3 signaling pathways. TA-46 has received orphan designation from the US FDA and the European Medical Agency. It has completed a Phase 1 trial.
Therachon will spin-off its apraglutide development into a different company. Apraglutide is a GLP-2 analog which is in Phase 2 study, targeted for the treatment of short bowel syndrome.
April 25, 2019
Pfizer's Talzenna(talazoparib) got CHMP positive opinion for the treatment of BRCA mutated locally advanced or metastatic breast cancer
Talazoparib is oral poly (ADP-ribose) polymerase (PARP) inhibitor. It is indicated as monotherapy for (gBRCA)1/2-mutations who have HER2-) locally advanced (LA) or metastatic breast cancer. The approval is based on EMBRACA trial.
EMBRACA trial: A Phase 3, open-label, randomized compared talazoparib compared with standard of care as per physician’s choice. The inclusion criteria are RCA1/2 mutation and triple-negative or HR+/HER2- LA or MBC who may have received up to three prior cytotoxic chemotherapy regimens.
Talazoparib is already approved by the US Food and Drug Administration (FDA).
Janssen’s CAR-T therapy JNJ-4528 received PRIME designation by European Medicines Agency
Janssen has announced that its B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR-T) therapy has received the PRIME (PRIority Medicines) designation from the European Medicines Agency. This helps in enhanced interaction and early dialogue of cutting-edge technologies that are intended to meet high unmet needs.
The PRIME designation was given based on Phase 1/2 LEGEND-2 clinical trial which evaluated the LCAR-B38M CAR-T cells, sponsored by Nanjing Legend Biotech Co., Phase 1b/2 CARTITUDE-1 study evaluating JNJ-4528.
JAJ-4528 is currently targeted to treat multiple myeloma. It is indicated for the patients who have failed three therapies including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 antibody. There are very few treatment options available after the failure of 3 therapies and have very poor outcomes.
US FDA approved Janssen’s Balversa for treatment of Locally Advanced or Metastatic Urothelial Carcinoma with Certain FGFR Genetic Alterations
Balversa (erdafitinib) is the first FGFR kinase inhibitor approved by the US Food and Drug Administration.
Johnson & Johnson’s pharmaceutical subsidiary Janssen has announced that it has received the approval of Balversa (erdafitinib) from the US FDA for the treatment of locally advanced or metastatic urothelial carcinoma (mUC) with fibroblast growth factor receptor (FGFR)3 or FGFR2 genetic alterations. The treatment is indicated after the failure of platinum therapy. Earlier the product has received the Breakthrough Therapy Designation in March 2018 and Priority Review Designation in September 2018.
The approval is based on the Phase 2 clinical trial (BLC 2001, NCT02365597), multicenter, open-label, single arm study who has one of the generic variations mentioned and had at least one prior therapy. The trials resulted in 32.2% objective response rate (ORR). In the trial, the median duration of response was observed to be 5.4 months.
Adverse events include ocular disorder, hyperphosphatemia, and embryo-fetal toxicity. Stomatitis, fatigue, created increase, diarrhea, dry mouth, onycholysis, increase in alkaline phosphatase, alanine aminotransferase, decrease in sodium and appetite, dry eye, alopecia, constipation.
Gilead announced 5 grants to support research in HCV, HBV, NASH, PSC and HCV HIV Co-infection
Gilead has announced the launch of 5 new industrial grants for research on chronic hepatitis C (HCV) hepatitis B virus (HBV), HCV and HIV co-infection, nonalcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC).
The proposals can include the addressing unmet needs, improving screening and linkage to care, easy HCV testing and treatment, developing access and care for NASH, key gaps in the HBV care and PSC epidemiology.
The new grant programs are
HCV STAT (Simplification and test and treat strategies toward HCV elimination0
HIV/HCV NoCo (NoCo-Infection)
NASH Models of Care
HBV CARE (Linkage to CARe & Epidemiology in Special Populations)
PSC PACE (Primary Sclerosing Cholangitis Epidemiology)
Amgen’s Evenity got US FDA approval for the treatment of postmenopausal women for osteoporosis
The US Food and Drug Administration has approved the Amgen’s Evenity for postmenopausal women who are at high risk of fracture. The risk is for those women are have the history of fractures, failed or intolerant to other osteoporosis treatment.
Postmenopausal women have weakened bones and are at high risk of developing fractures. There are 10 million in the United States with osteoporosis.
As per Hylton V. Joffe, M.D, M.M.Sc., director of the Center for Drug Evaluation and Research’s Division of Bone, Reproductive and Urologic Products, patients taking Evenity are at higher risk of developing heart attacks, stroke, and cardiovascular problems.
Evenity has an effect on the protein sclerostin and is mainly involved in the formation of new bone.
The approval is based on two clinical trials involving 11,000 women. In the first trial, Evenity reduced the risk of vertebral fracture by 73% compared to the placebo. In the second trial, one-year treatment followed by one alendronate reduced the risk of vertebral fracture by % compared to the two years treatment of alendronate monotherapy.
Joint pain and headache are the most common side effects observed.
Eli Lilly and ImmNext collaborate to research on autoimmune diseases
A global licensing and research collaboration announced by Eli Lilly and ImmuNext for developing new drugs for autoimmune diseases by regulating the immune cell metabolism.
As per Ajay Nirula, M.D., Ph.D., vice president of immunology at Lilly, immunology is an important therapy area and Lilly is interested in regulating immune cell metabolism as an important target for fighting autoimmune diseases.
Under this collaboration, ImmuNext will get $40 million in payment and $565 million during development and commercialization. As per the collaboration, an exclusive worldwide right will be given to Lilly by ImmuNext to develop and commercialize novel immunometabolism target.
There is a 3-year collaboration between Lilly and ImmuNext for the target’s development.
April 07, 2019
Japan’s MHLW grants SAKIGAKE designation to Eisai's E7090
Eisai has announced that their in-house fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor E7090 has received SAKIGAKE designation from Japan’s MHLW. The product is indicated to manage cancer.
In order to attain the SAKIGAKE designation, the product should have a new mechanism of action. These products have the advantage of getting prioritized consultation services and reviews for regulatory authorizations.
The product is currently in the Phase 1 study in Japan and is targeted in treating the patients with solid tumors including cholangiocarcinoma harboring FGFR2 gene fusion
April 07, 2019
Novartis bounces back on the Amgen’s termination notice for the treatment of migraine
Novartis replied on the Amgen’s termination notice for the treatment of migraine.
On Apil 2, Amgen has issued a notice asking to terminate the collaboration agreement between Novartis and Amgen citing the material breach. In reply, Novartis filed the lawsuit stating that Amgen is not authorized to terminate the agreement. As per Novartis, the agreement will be in force, unless the court terminated the agreement.
Aimovig is a product which is a preventive treatment for migraine. The product was developed in collaboration between the Novartis and Amgen.
March 31, 2019
China approved Merck’s Keytruda for the treatment of Metastatic Non-squamous Non-Small Cell Lung Cancer (NSCLC) as first-line therapy
Keytruda was approved in China for the treatment of metastatic non-squamous Non-Small Cell Lung Cancer (NSCLC) in combination with pemetrexed and platinum chemotherapy in the first line treatment. Keytruda (pembrolizumab) is anti-PD-1 therapy which is approved by multiple tumor types.
Merck has submitted the Phase 3 results of KEYNOTE-189 trial, showing the overall survival and progression-free survival as endpoints.
KEYNOTE-189 trial showed that Keytruda in combination with chemotherapy has reduced the risk of death to half compared to chemotherapy alone. The trail also highlighted the improvement in progression-free survival.
Lung cancer is the leading cause of death in China. The estimated incidence of lung cancer in China is 782,000 per each year.
April 05, 2019
Gottelib MD ends his tenure as FDA Commissioner
Scott Gottlieb, MD ends his tenure as the US Food and Drug Administration (FDA) commissioner yesterday (5th April). This is just a few days before he completes his two years as FDA Commissioner.
Earlier in the last month, Gottlieb has submitted his resignation to the Health and Human Services Secretary Alex Azar.
Earlier the US president Donald Trump has praised in his word in approving the generics and hence bringing the drug prices down.
Gottelib was now associated with the American Enterprise Institute. Gottelib is also interested in spending time on the drug pricing and policy-making for the FDA.
Gottelib tenure witnessed the approval of highest generics in a year and also the approval of new devices and advanced therapies.
Norman “Ned” Sharpless, MD, director of the National Cancer Institute will step in as the new FDA Commissioner.
April 03, 2019
Pfizer’s Ibrance is now indicated for treatment of breast cancer in males
The US FDA has approved Ibrance for the treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer in males.
As per Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence, the approval is based on the post-market studies which showed that the safety of in males was like that of the females based on the electronic records.
Some of the approved indications do not distinguish between the male and female patients, however, the extension of the label helps.
Breast cancer is rare in males, most of the cases are due to the expression of hormone receptors. If the patients are positive, they can be treated with hormone therapy or chemotherapy. There are several therapies available for hormone positive cancer patients, however, some the therapies are restricted for female patients only.
Ibrance is a kinase inhibitor, indicated along with aromatase inhibitors. Pfizer submitted the real word data from electronic health records.
The common adverse events with Ibrance are infections, leukopenia, fatigue, nausea, stomatitis, hair loss, thrombocytopenia, and fever. The blood count needs to be assessed before starting the treatment.
Iberville Parish filed suits on the pharmaceutical companies regarding the use of prescription opioids
The Iberville Parish Council filed suits on the pharmaceutical companies regarding the use of prescription opioids. The Council accused the pharmaceutical companies on over distribution of the prescription opioids and violating the conduct. The Council expressed that the distribution cannot exceed the medical need.
1,800 similar suits have been registered across the United States, Ohio judge seeks a solution applicable throughout the nation.
Iberville parish president J. Mitchell Ourso to an interview to WAFB said that it's not about the money but concerned with the safety and welfare of the patients.
The suit says that the pharmaceutical companies are not detecting the unusual distribution of the drugs. Also, the distributors are not stopping the suspicious sales and these drugs are not being used for pharmaceutical needs.
The complaint has been moved to the US courts as plaintiff expressed the view that federal courts have better solutions for this problem.
Bayer submits market authorization application to EMA for approval of darolutamide for treating non-metastatic castration-resistant prostate cancer
Bayer has submitted the market authorization application of darolutamide to European Medical Agency for treating non-metastatic castration-resistant prostate cancer (nmCRPC).
The submission is based on Phase III ARAMIS trial in men with nmCRPC. The results from the trial showed significant improvement in metastasis-free survival for darolutamide and androgen deprivation therapy.
Prior to submission in Europe, Bayer already submitted the new drug application to the US FDA and MHLW in Japan.
Darolutamide is being developed by Bayer in collaboration with Orion Corporation.
March 06, 2019
Roche’s Veclexta achieved breakthrough designation from US FDA for the treatment of previously untreated chronic lymphocytic leukemia
Roche submitted a supplemental new drug application to FDA of Venclexta plus Gazyva combination which is indicated for the treatment of previously untreated chronic lymphocytic leukemia. The application was reviewed under the FDA’s real-time Oncology Review Pilot Program.
The combination of Venclexta® (venetoclax) and Gazyva® (obinutuzumab) has achieved breakthrough therapy designation status and this is the fifth breakthrough therapy designation for Venclexta.
The designation was given based on results of the Phase CLL 14 study, which compared the combination of Venclexta plus Gazyva versus Gazyva and chlorambucil. The target population is the patients who are untreated and have co-existing medical conditions.
CLL 14 study:
CLL 114(NCT02242942) is a randomized phase III study which compared the combination Venclexta plus Gazyva versus Gazyva and chlorambucil. 432 patients were included in this study for a period of 12 months.
Arm 1: Obinutuzumab + Chlorambucil, patients received obinutuzumab for 6 cycles and chlorambucil for 12 cycles
Arm 2: Obinutuzumab + Venetoclax, patients received obinutuzumab for 6 cycles and venetoclax for 12 cycles
The primary endpoint is progression-free survival.
CMS proposes coverage of CAR T-cell therapy for the treatment of cancer
Centers for Medicare & Medicaid Services proposes to over the CAR T-cell therapy which has been approved earlier the US FDA.
Currently, CAR T-cell therapy is not being covered by any Medicare policy across the united states.
CAR T-cell therapy uses the patient's immune system for fighting against cancer and is proposed to cover under "Coverage with Evidence Development". This coverage enable to national wide coverage by Mediare
FDA takes action against 17 companies for illegally selling drugs to treat Alzheimer's disease
FDA issued 12 warning letters and 5 advisory letters to different companies across the United States and other countries for selling 58 products that are sold illegally claiming that they treat Alzheimer's disease. Most of the products are dietary supplements which claimed to treat Alzheimer's disease.
These products are available online and social media sites. These drugs are not assessed by the FDA for their efficacy and safety. US FDA states that the products might not be safe to use.
FDA Commissioner Scott Gottlieb stated that Alzheimer's disease is not treatable and drugs claiming that they can treat the disease are fake.
Gottlieb has also revelated the future plans of the US FDA on the regulations for dietary supplements including effective and fast communication of the safety issues with the various dietary supplements with the patients and steps to promote innovation.
FDA is seeking the reply within 15 days. Also, the FDA adviced the people to more vigilant towards the products while purchasing the products online.
February 05, 2019
Sanofi’s Cablivi was approved by US FDA for treatment of acquired thrombotic thrombocytopenic purpura
The US Food and Drug Administration has approved Cablivi for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressants.
Acquired thrombotic thrombocytic purpura (aTTP) is a rare blood disorder which causes the blood clotting the smaller blood vessels, restricting the flow of blood to the vital organs causing stroke and heart attacks. aTTP is developed in patients who had undergone surgery, bone marrow transplantation, and chemotherapy.
The approval is based on the clinical trial which enrolled 145 patients, patients were randomized to receive placebo and Cablivi. In both the groups, the patients were administered with immunosuppressants and plasma exchange. In the trial, it was observed that the platelet count increased in the Cablivi group compared to the placebo group.
The adverse events include the bleeding from the nose and gums, also causing the headache.
The approval of Cablivi was given to Ablynx. Ablynx was acquired by Sanofi earlier for a deal of $4.5 billion.
Mylan's generic version of Advair Diskus was approved by the US FDA
The generic version of Advair Diskus was approved in the United States and this is the first generic approved. the product consists of fluticasone propionate and salmeterol which is indicated as inhalation powder. The drug is indicated for the treatment of asthma in patients who are older than 4 years and for the maintenance treatment of chronic obstructive pulmonary disease.
The drug was released in three strengths
fluticasone propionate 100 mcg/ salmeterol 50 mcg
fluticasone propionate 250 mcg/ salmeterol 50 mcg
fluticasone propionate 500 mcg/ salmeterol 50 mcg
Asthma is chronic health disease which causes the obstruction of air passage causing wheezing, chest heaviness and coughing.
COPD is a chronic disease which causes hardening in breathing.
Novartis to apply Japanese authorities for approval of its gene therapy product indicated for SMA
Novartis is applying for Japan authorities for the approval of its $4 million gene product which is used to treat the neurological disorder.
Earlier FDA has approved the product which is expected to cost between $4-5 million in the United States.
The gene therapy is used to spinal muscular atrophy, a single time treatment. Switzerland based drugmaker is expecting to bring the product to the drug market in the next year.
SMA is the condition where patients lack proteins to move the muscles and die at an early age. However, in the clinical trial submitted by Novartis showed that the survival rate is 100%.
The gene therapy was initially developed by AveXis which was later acquired by Novartis for $8.7 billion.
Novartis is highly investing in research and development, with more than 500 clinical trials in therapeutic areas including cancer, heart disease, metabolic disorders, and rare diseases.
December 11, 2018
Rinoclenil was able to maintain its reimbursement in France
Chiesi's Rinoclenil was able to maintain its reimbursement in France. The product contains beclometasone 17α,21-dipropionate. The drug is indicated to treat perennial or seasonal allergic rhinitis and chronic inflammatory rhinitis with eosinophils.
The market authorization holder has not provided new data for the product. The earlier evaluation of the product has been conducted on 25 June 2014.
The product provided moderate therapeutic benefit.
The product was able achieve reimbursement rate of 30%.
December 04, 2019
One more infliximab biosimilar assessed by HAS sante in France
One more infliximab biosimilar Zessly was assessed by HAS in France.
The biosimilar got an ASMR V for
Crohn's disease of the adult and the child
Ulcerative colitis of the adult and the child
Inadequate response to DMARDs in case of rheumatoid arthritis
January 02, 2019
BMS to acquire Celgene for $74 billion
Bristol Myers-Squibb is buying Celgene for $74 billion
Bristol Myers-Squibb blockbuster product Opdivo is facing tough competition with Merck's Keytruda
Celgene is also facing challenges
BMS is acquiring Celgene, an oncology specialist company. The acquisition is based on the cash and stock transaction worth of $74 billion. As per the transaction, Celgene stock holder gets one BMS share and $50 per each share. The directors of both the companies has agreed to the deal.
As per the company this will help to develop the drugs in major therapy areas including cancer, inflammatory and immunologic disease and cardiovascular disease.
Stemline Therapeutics' Elzonris was approved by the US FDA for treatment of blastic plasmacytoid dendritic cell neoplasm
Stemline Therapeutics Elzonris has been approved by the US FDA for the treatment of blastic plasmacytoid dendritic cell neoplasm, a rare disease of bone marrow and blood.
Earlier the standard of care for this was intensive chemotherapy followed the by the stem cell transplantation. The disease usually affects the patients of age more than 60 years and affects mainly men.
The approval was based on the clinical trial which includes
FDA has granted breakthrough and priority status earlier along with the orphan designation.
Alexion's Ultomiris approved by FDA for the treatment of paroxysmal nocturnal hemoglobinuria
Alexion's Ultomiris (ravulizumab) has been approved for the treatment of paroxysmal nocturnal hemoglobinuria, a rare blood disorder which is life-threatening.
The approval is based on the clinical trial including 246 patients. The comparator arm was eculizumab, where ravulizumab showed non-inferiority compared to eculizumab. The incidence rate hemolysis in both the drugs was similar. In another study including 195 patients remained clinically stable who were treated for at least 6 months with eculizumab. Upper respiratory infections and headache were common side effects during the treatment.
About paroxysmal nocturnal hemoglobinuria (PNH):
Patients with PNH suffer from the destruction of the blood cells, a general mechanism to save the blood cells is missing in these patients. The patients generally suffer from the destruction of the blood cells where the patients suffer from fatigue and infections. The disease is diagnosed in all ages particularly in the young adulthood.
Earlier Soliris from Alexion has been approved for the treatment of this particular disease.
January 01, 2020
Pfizer and GSK combine their consumer health business to start £9.8 bn consumer drugs giant
Pfizer and GSK combined their consumer health business which is worth of around £9.8 bn to bring a new consumer health giant.
This paves the way for GSK to enter in two paths, one into the consumer health and the other into the development of pharmaceuticals and vaccines. Based on the deal, GSK has 68% shares and Pfizer has 32% shares.
This new venture includes brands like Panadol, Anadin and Voltarol.
The shares of GSK raised high with this news however this might lead to cuts in jobs. Though Emma Walmsley has not mentioned anything specific about the jobs, she has hinted that this could happen in the future.
As per George Salmon, equity analyst, this will raise the expectations from GSK on the development of the new drugs and expect better results from the labs.
Pharmaceutical companies are facing tough situations because of the price restrictions from different countries.
NICE recommends Gileads's CAR T-cell therpay for adults with non-Hodgkin lymphoma
NICE has recommended Kite Pharma's Yescarta (axicabtagene-ciloleucel) for treatment of certain types of non-Hodgkins lymphoma.
NICE recommended the CAR T-cell therapy for adult patients with relapsed or refractory diffuse large B-cell lymphoma or primary mediastinal large B-cell lymphoma who have not responded to intensive chemotherapy for at least two times or if the drug reoccurs after the stem cell transplant.
The drug will be available through the Cancer Drugs Fund. The company has also given commercial access agreement.
It is expected that there are around 200 people in England who gets benefited with this new CAR T-cell therapy.
NICE has mentioned that the drug has safety uncertainties in the long-term treatment.
EMA has approved another gene therapy Novartis' Luxturna
EMA approved Luxturna, another gene therapy, which has already approved by US FDA a year earlier.
Luxturna is indicated to treat the vision loss because of the genetic mutation in RPE65 genes. The mutation usually leads to the less vision in the earlier stage and then lost the final vision.
As per Novartis, this therapy helps in getting the vision back if the eye has enough retinal cells.
The approval is based on the Phase 1 trials and Phase 3 trails.
December 06, 2018
FDA has given approval for Roche's Tecentriq for treatment of metastatic non-squamous non-small cell lung cancer in combination with Avastin, paclitaxel and carboplatin
Roche released a press release that it has got the approval from US FDA for the treatment of metastatic non-squamous non-small cell lung cancer (NSCLC) with no EGFR or ALK a genomic tumor for Tecentriq (atezolizumab) combination with paclitaxel and carboplatin.
The approval is based on the Phase III IMpower150 study where the results are
Overall survival of
Tecentriq + Chemotherapy + Avastin - 19.2 months
Avastin + Chemotherapy - 14.7 months
The impower150 study was a phase 3 randomized open-label trial which evaluated the efficacy of Tecentriq in combination with Avastin and chemotherapy versus Avastin and chemotherapy.
MOA: Tecentriq is a MAb which is designed to block PD L1 protein blocking its interaction with both PD-1 and B7.1 receptors.
November 22, 2018
EMA has approved another gene therapy Novartis' Luxturna
EMA approved Luxturna, another gene therapy, which has already approved by US FDA a year earlier.
Luxturna is indicated to treat the vision loss because of the genetic mutation in RPE65 genes. The mutation usually leads to the less vision in the earlier stage and then lost the final vision.
As per Novartis, this therapy helps in getting the vision back if the eye has enough retinal cells.
The approval is based on the Phase 1 trials and Phase 3 trails.
November 26, 2018
Loxo Oncology and Bayer's Vitrakvi tissue agnostic drug gets approval from US FDA
US FDA has approved Vitrakvi which is a new cancer drug that targets the genetic mutation but not the type of cancer.
The drug can target any cancer within the body that arise because of neurotrophic receptor tyrosine kinase gene fusion.
Vikatrakvi (larotrectinib) is originally developed by Loxo Oncology. Bayer has acquired Loxo Oncology's TRK inhibitor program for $400 million.
The cost of the drug is of $32,800 per month, which costs nearly $393,000 a year. To provide higher access to the drug, Bayer provides a refund if the patients don't respond in 90 days. Also, Bayer agreed to provide reimbursement and patient assistance services.
The challenge the drug will face is the diagnosing the patients as the techniques which can identify the specific genetic mutation are not widely available.
Australia's TGA has approved the first kit to test HIV at home
In Australia, TGA has approved the home testing kit for HIV, which is expected to be a significant move in eradicating the virus.
Home testing kits are being recommended by several AIDS-related organizations, as this is already in use in several other countries.
This is expected to be helpful in eliminating the virus as per Mr. Hunt.
Darryl O’Donnell, Australian Federation of AIDS Organisations chief executive has expressed his view that this a significant step for the future.
Atomo Self Test will be available online in the future.
This is subjected to TGA conditions, which means that the kit will be not available with the chemists. Mr. Parkhill has a view that restricting the kits to the online might restrict the use of the kits.
The kit is expected to cost $39.50 per script. It further cost $6.40 for people who are having the concession.
November 20, 2018
Pfizer's Daurismo got approval from US FDA for treating acute myeloid leukemia
The US FDA has approved Daurismo (glasdegib) for treatment of acute myeloid leukemia in combination with low dose cytarabine (LDAC). The drug is used in newly diagnosed patients who are older than 75 years of age or who have other chronic diseases.
Patients are usually treated with high intensive therapy, however, some patients cannot tolerate high intense therapies because of the toxicities. This drug can be given in older patients and other patients who cannot tolerate high intense chemotherapy.
The efficacy of Daurismo was studied in Phase 3 clinical trial where 111 patients were tested. Patients who have received the combination of Daurismo plus LDAC has an overall survival of 8.3 months compared to the LDAC which has an overall survival of 4.3 months.
About acute myeloid leukemia:
AML is cancer where there is the excess generation of the white blood cells by the bone marrow. 19,520 people are diagnosed with AML and 10,670 patients died in the year 2018.
US FDA approves Novoimmune's Gamifant for treatment of primary hemophagocytic lymphohistiocytosis (HLH)
US FDA has approved Novoimmune's Gamifant (emapalumablzsg) for treatment of primary hemophagocytic lymphohistiocytosis (HLH) in adults and paediatrics patients including the newborn. Gamifant is the first that has been specifically approved for primary hemophagocytic lymphohistiocytosis.
Primary hemophagocytic lymphohistiocytosis is a rare disease that is effecting the paediatric causing mortality. This drug could offer solution for the unmet need.
The approval is based on the clinical trial involving 27 paediatric patients who are refractory, recurrent or progressive to the current therapy indicated for HLH. In the trial, 63% of the patients showed a response with 70% patients proceeded to stem cell transplant.
Adverse events observed include hypertension, infections and reactions.
The drug was able to achieve priority review and breakthrough review along with orphan designation.
About primary hemophagocytic lymphohistiocytosis (HLH)
HLH is the immunological condition where the immune cells overreact release molecules that cause inflammation. This leads to the destruction of the cells within the brain, liver and bone marrow.
November 19, 2018
Tecentriq/Avastin combination for kidney cancer was pulled back by Roche in Europe
Roche has pulled out the combination of Tecentriq and Avastin combination for the treatment of renal cell carcinoma for EU approval citing that the studies were not enough for the approval.
Tecentriq (atezolizumab) is a PD-L1 inhibitor and Avastin (bevacizumab) is anti-VEGF drug.
Roche is expecting the approval of this combination for first-line treatment for renal cell carcinoma based on Phase 2 study IMmotion151. The progression-free survival was reduced by 26% compared to Pfizer's Sutent.
Earlier Roche has withdrawn the application from US FDA as well.
Ei-Lilly has filed for US FDA for the approval of its migraine product lasmiditan. The drug acts a 5-HT1F agonist and showed in the Phase 3 trials that the product can reduce migraine headache within 2 hours.
Earlier Ei-Lilly has acquired CoLucid for $960 million which is the inventor of lasmiditan. One of the two trials was done by CoLucid. The product is administered orally.
Lasmiditan is expected to the first in class product with a new mechanism of action for the treatment of migraine.
In September, FDA has approved Ei Lilly's product galcanezumab for treatment of migraine in adults. Galcanezumab is a subcutaneous injection injected once monthly.
November 15, 2018
Pfizer’s Mylotarg is now included in the NICE guidelines for treatment of CD33-positive acute myeloid leukaemia
In the current guidelines NICE has recommended the use of Mylotarg for the treatment of CD33-positive acute myeloid leukaemia.
Mylotarg is gemtuzumab ozogamicin which contains the antibody that direct the drug to cancer cells. The mechanism of action includes blocking of the CD33 antigen expressed by hematopoietic cells.
The drug got the market authorization from EMA on 18/04/2018.
November 15, 2018
HeartFlow FFRct Analysis gets reimbursement approval in Japan
HeartFlow, Inc. mentioned that it received the reimbursement approval by Ministry of Health, Labor and Welfare (MHLW) for HeartFlow FFRct Analysis. Earlier the reimbursement recommendation has been made by the Central Social Insurance Medical Council (Chuikyo). Earlier HeartFlow FFRct Analysis has received the approval from Pharmaceuticals and Medical Devices Agency (PMDA).in Japan. This will help the patients who are suspected to have coronary artery disease.
The device will enable to diagnose the patients who are suspected to have coronary artery disease. The method is invasive and helps in the easy diagnosis
The economic burden of coronary artery disease is quite high with half the deaths in related to hear diseases in Japan are because of coronary artery disease. This technique could significantly reduce the cost of the CAD diagnosis.
Intellectual property rights of pharmaceuticals were revised by European Commission
The European Commission is targeting to refine the intellectual property rights to encourage the pharmaceutical companies in conducting research which enable the growth and rapid employment generation. The commission is committed in developing Europe to be the headquarters for the development of pharmaceuticals.
Supplementary protection certificates are helpful in protecting the patent for the products that takes longer time. However with the waiver, the generics can be developed by European markets and can export to the companies where the patent has expired.
Other information from the news:
In 2017, the pharmaceutical market encompassed for nearly 1 trillion euros. This made the markets to search for the generics and biosimilars. EU is the center for the development of generics and biosimilars, however the commission feel that this place is under threat.
Amgen’s Migraine drug Aimovig received the US FDA approval
On May 17, 2018 the US Food and Drug Administration approved Amgen’s Aimovig (erenumab) for the prevention of migraine.
It is the first in class drug, targeting Calcitonin gene-related peptide (CGRP). The drug helps in reducing the number of days with migraine.
A self-administered injection, it is priced at USD6,900 a year, or USD575 a month.
May 07, 2018
Shire board OKs for Takeda's £46bn deal
Shire board has recommended the deal of Takeda's £46bn deal to acqure Shire, the rare disease specialist.
This could make the Japanese drug maker, a top global player in the field of pharmaceuticals. b
However the approval from the 75% of Takeda and Shire shareholders seems to be difficult.
The shares of Takeda dropped after the release of the news.
April 30, 2018
University of Bristol researcher won Career Development Award worth £1.2 million
Dr Borko Amulic got the prestigious Career Development Award from Medical Research Council (MRC) worth of £1.2 million.
This is for the research to conduct on neutrophils. The fund is to understand the underlying mechanism behind the defense mechanism of the neutrophils.
Neutrophils are defense cells that are present in the body and of highest number. Without them the body becomes immune-less.
Dr Borko Amulic responded that he was happy and honored to receive the award and was confident that with the talent present at University of Bristol, he would be able to achieve good results.
The award is for 5 years.
April 13, 2018
EU says OK for Roche’s Alecensa as first line treatment for NSCLC but NICE says NO
NICE has not recommended Roche’s Alecensa (alectinib) as first line treatment for ALK positive NSCLC. EU has approved Alecensa for as first line option for treating patients with NSCLC.
The current standard of care for ALK positive NSCLC is crizotinib for which progression free survival was 10.4 months where as for Alecensa it is 25.7 months.
However NICE was not satisfied with the company's submission of cost effectiveness, drug wastage, CNS care during disease progression.
April 04, 2018
Regulatory authorities on either side of atlantic accepted for review of Pfizer's Dacomitinib
Dacomitinib has received priority review from FDA for treating locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR-activating mutations in the first line.
For the same indication, European Medical Agency (EMA) has also accepted the Marketing Authorization Application.
FDA gives priority reviews for products that it believes to give a significant advance in the treatment. Mace Rothenberg, M.D., chief development officer, Oncology, Pfizer Global Product Development said that "While significant progress has been made in the treatment of patients with non-small cell lung cancers harboring GFR-activating mutations, it remains a challenging disease and new treatment options are needed”
This is the second investigation product to get the regulatory acceptance within two months, showing the Pfizer's commitment in treating the lung cancer.
Pfizer's submissions were based on Phase 3 ARCHER 1050 study where dacomitinib (n=227) compared to gefitinib, dacomitinib treated patients showing significant development than gefitinib treated patients.
The median progression free survival was 14.7 months in dacomitinib arm compared to 9.2 months of gefitinib.
The side effects observed includes diarrhea, nail changes, rashes and mouth sores.
2% of patients experienced grade 4 adverse events.
BREXIT IMPACT - Update on EMA relocation to Amsterdam:
EMA will move from London to Amsterdam before the end of March 2019, when the United Kingdom withdraws from the European Union (EU). The Dutch authorities have committed to building completely new, tailor-made premises for EMA in the business district Zuidas. The building will not be completed by 30 March 2019, when EMA has to be fully operational in Amsterdam. Therefore, the selection and availability of a temporary building was part of the Netherland’s bid to host the Agency. Management Board started building approval process of EMA premises in Amsterdam
February 18, 2018
EMA Drugs safety Highlights
Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 5-8 February 2018:
PRAC recommends new measures to avoid valproate exposure in pregnancy:
PRAC has recommended new measures to avoid exposure of babies to valproate medicines in the womb, due to risks of malformations and developmental problems. Medicines containing valproate have been approved to treat epilepsy, bipolar disorder and for the prevention of migraine. PRAC also recommended to introduce pregnancy prevention programme to avoid the exposure.
PRAC recommends women taking ulipristal acetate for uterine fibroids to have regular liver tests:
The PRAC has recommended regular liver monitoring for women taking Esmya (ulipristal acetate) for uterine fibroids (non-cancerous growths that develop in or around the womb), following reports of serious liver injury, including liver failure leading to transplantation.
PRAC recommends that the marketing authorisations of the painkiller flupirtine be withdrawn:
The PRAC has recommended that the marketing authorisations for the painkiller flupirtine be withdrawn, as the benefits of this medicine are no longer considered to outweigh its risks, particularly the risk of liver problems.