May 29, 2019

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. 

May 28, 2019

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. 

May 23, 2019

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.

May 23, 2019

Cometriq was not recommended by NICE for treatment of hepatocellular carcinoma

NICE was unable to make the recommendation on Cometriq for the treatment of hepatocellular carcinoma as the Ipsen Limited has not submitted the evidence.

The company has confirmed that it has not made the submission for the approval as the company said there is no sufficient evidence to prove that the product is cost-effective for the NHS.

May 25, 2019

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.

May 19, 2019

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%. 

May 15, 2019


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.

May 16, 2019

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. 

May 15, 2019

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

SWOG S0777:

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%).