2022 | CHMP Positive Opinions | EMA Drug Approvals | European Commission | Pharma | News
- ipharmaservices
- Feb 28, 2023
- 14 min read
Updated: Jun 26, 2023
DECEMBER 2022 CHMP POSITIVE OPINIONS
CSL's gene therapy, Hemgenix, received a positive CHMP opinion for treating Hemophilia B.
The CHMP gave a positive opinion for Hemgenix (etranacogene dezaparvovec), a gene therapy for treating severe and moderately severe Haemophilia B.
The approval was based on the HOPE-B trial, demonstrating the efficacy of Hemgenix in patients with hemophilia B. Hemgenix showed a stable and durable increase in mean Factor IX activity, and the annual bleeding rate was reduced by 64%.
Haemophilia B is a rare disease resulting in bleeding in joints, muscles, and internal organs, leading to pain, swelling, and joint damage. The current standard of care is the infusion of factor IX for a lifetime.
Imjudo and Imfinzi combination received CHMP positive opinion for advanced liver and lung cancers.
AstraZeneca's Imfinzi (durvalumab) and Imjudo (tremelimumab) combination received CHMP positive opinion as first-line therapy for patients with advanced or unresectable hepatocellular carcinoma (HCC), and Imfinzi, Imjudo, chemotherapy combination in patients with Stave IV non-small cell lung cancer (NSCLC).
The approval as first-line treatment in liver cancer patients was based on the Phase 3 HIMALAYA trial. STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab) reduced the risk of death by 22% versus sorafenib. The median OS was 16.4 months in patients on the STRIDE regimen versus 18.4 months in patients on sorafenib.
Positive opinion for Stage IV lung cancer s based on the POSEIDON Phase 3 trial. Anti-CTLA-4 antibody Imjudo, Imfinzi, and four cycles of platinum-based chemotherapy reduced the risk of death by 23% versus chemotherapy alone. After five years, 31% of patients on the Imjudo combination were alive versus 22% of patients on chemotherapy.
Pombiliti received CHMP positive opinion for Pompe disease.
Amicus Therapeutics' Pombiliti (cipaglucosidase alfa) received CHMP positive opinion for late-onset Pompe Disease.
The positive opinion was based on Phase 3 pivotal study (PROPEL) study, which recruited high unmet need enzyme replacement therapy (ERT)-experienced and ERT-naive patients.
NOVEMBER 2022 CHMP POSITIVE OPINIONS
GSK and Sanofi's VidPrevtyn received CHMP positive opinion as a booster dose to prevent COVID-19
GSK and Sanofi's VidPrevtyn Beta received CHMP positive opinion as a booster dose in adults previously administered with an mRNA or adenoviral vector COVID-19 vaccine.
Sanofi announced that it was the first protein-based adjuvanted COVID-19 vaccine approved in Europe. Further, the companies announced that the vaccine is effective against all variants of COVID-19.
The positive opinion was based on Phase 3 VAT08 Stage 2 trial, in which 23,726 individuals were recruited. Sanofi announced that the next-generation vaccine was more effective in stimulating immune response than Pfizer-BioNTech's COVID-19 vaccine.
INDICATION EXTENSIONS
Sanofi's Dupixent received CHMP positive opinion for prurigo nodularis
Dupixent (dupilumab) received CHMP positive opinion to treat adults with moderate-to-severe prurigo nodularis who are candidates for systemic therapy.
The CHMP positive opinion was based on two Phase 3 trials, PRIME and PRIME2; Dupixent reduced itching and skin lesions versus placebo. There was an improvement in health-related quality and a reduction in skin pain and symptoms of anxiety/depression.
Adverse events were higher in the Dupixent arm versus the placebo.
Enhertu received CHMP positive opinion for gastric cancer.
Daiichi Sankyo received CHMP positive opinion for Enhertu (trastuzumab deruxtecan) to treat adults with HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
Bayer's Eylea received CHMP positive for treating preterm infants with retinopathy of prematurity.
Eylea (aflibercept) received CHMP positive opinion for treating preterm infants with retinopathy of prematurity (ROP).
The CHMP positive opinion was based on Phase III study FIREFLEYE, in which Eylea failed to show non-inferiority versus laser photocoagulation (85.5% on intravitreal Eylea vs. 82.1% with laser photocoagulation).
Laser photocoagulation was associated with the destruction of retinal tissue, which might lead to high myopia (nearsightedness) and peripheral vision loss.
With the indication extension, Bayer aims to expand the patent protection period of Eylea by six months. Bayer seeks to extend the patent until November 2025 with the patent extension.
AstraZeneca's Imfinzi received a positive CHMP opinion for treating biliary tract cancer.
Imfinzi received CHMP positive opinion for treating adults with unresectable or metastatic biliary tract cancer. Imfinzi has to administer in combination with gemcitabine or cisplatin.
OCTOBER 2022 CHMP POSITIVE OPINIONS
Takeda's Dengue Tetravalent Vaccine received CHMP positive opinion.
Takeda's Dengue vaccine received CHMP positive opinion and is active against genotypes 1, 2, 3, and 4. The vaccine received CHMP positive opinion for four years and older.
Nearly 20,000 to 25,000 per year die because of dengue infection, primarily children. The vaccine protects against fever, hospitalization, and death. The vaccine also received CHMP positive opinion under the brand name Qdenga.
The first therapy for the post-transplant lymphoproliferative disease, Ebvallo, received positive CHMP opinion.
Atara Biotherapeutics and Pierre Fabre's Ebvallo (tabelecleucel) received CHMP positive opinion for Epstein-Barr virus-positive post-transplant lymphoproliferative disease. Ebvallo received a positive opinion for treating adults and pediatric patients two years and older.
Ebvallo is Epstein-Barr virus (EBV)-specific T-cell immunotherapy developed for EBV-driven diseases, including post-transplant lymphoproliferative disease (EBV + PTLD).
The approval was based on the Phase 3 ALLELE Study, demonstrating a favorable risk-benefit profile. The median overall survival was nearly 1.7 months in EBV + PTLD following hematopoietic cell transplant (HCT) and almost three months in solid organ transplant (SOT) recipients with EBV + PTLD. SOT and HCT patients received a median of two and three cycles. The objective response rate was 50% in the overall population and 50% each in SOT and HCT patients. The median overall survival was 18.4 months.
Novartis' Pluvicto received CHMP positive opinion for PSMA-positive metastatic castration-resistant prostate cancer.
CHMP has given a positive opinion for Pluvicto (lutetium (177Lu) vipivotide tetraxetan) to treat adult patients with PSMA-positive metastatic castration-resistant prostate cancer.
The CHMP positive opinion was based on the Phase III VISION study, in which Pluvicto plus standard of care showed improvement in overall survival versus standard of care alone in patients previously treated with AR pathway and taxane-based chemotherapy. The risk of death was reduced by 38%, and the risk of radiographic progression or death was reduced by 60% in patients on Pluvicto plus standard of care versus standard of care alone.
If approved, Pluvicto will be the first radioligand therapy available for prostate cancer in Europe.
After FDA approval, BI's Spevigo received CHMP positive opinion for generalized pustular psoriasis.
Boehringer Ingelheim's Spevigo (spesolimab) received CHMP positive opinion for patients with generalized pustular psoriasis (GPP) flares.
The approval was based on EFFISAYIL 1 trial, a 12-week Phase 2 trial in patients with GPP flares. 900 mg of IV spesolimab was tested versus placebo. One week after treatment with spesolimab, 54% of patients showed no visible pustules versus 6% of patients on placebo. After 12 weeks of treatment, 84.4% of patients had no visible pustules and almost had clear skin.
Spesolimab is an interleukin-36 receptor (IL-36R) inhibitor that received approval in the US and Japan.
INDICATION EXTENSION
BeiGene's Brukinsa received CHMP positive opinion for adults with CLL
BeiGene's Brukinsa (zanubrutinib) received a positive CHMP opinion for treating adult patients with chronic lymphocytic leukemia (CLL).
The CHMP positive opinion was based on a global head-to-head Phase 3 clinical study, ALPINE trial. Brukinsa (zanubrutinib) was tested versus ibrutinib in patients with relapsed or refractory chronic lymphocytic leukemia. In the SEQUOIA trial, Brukinsa was tested versus bendamustine plus rituximab in patients with previously untreated CLL or SLL.
Libtayo received CHMP positive opinion for cervical cancer
Libtayo (cemiplimab) received CHMP positive opinion for treating advanced cervical cancer. The recommendation was based on the Phase 3 trial, demonstrating significant improvement in survival versus chemotherapy, irrespective of PD-L1 status.
The approval was based on the Phase 3 EMPOWER-Cervical 1 trial, which recruited 608 patients. The primary endpoint was overall survival, which reduced the risk of death by 31% and 27% in squamous cell carcinoma.
Libtayo was approved for basal cell carcinoma, cutaneous squamous cell carcinoma (CSCC), and advanced non-small cell lung cancer (NSCLC).
SEPTEMBER 2022 CHMP POSITIVE OPINIONS
Beyfortus (nirsevimab) for preventing infection of respiratory syncytial virus (RSV)
CHMP gave a positive opinion for AstraZeneca's Beyfortus (nirsevimab) to prevent respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants during their first RSV season.
The positive opinion was based on MELODY Phase III, MEDLEY Phase II/III, and Phase IIb trials. In the studies, Beyfortus met the primary endpoint of reduction in the medically attending lower respiratory tract infection incidence versus placebo when administered as a single dose. There were no major differences in the safety outcomes between Beyfortus and placebo.
RSV infections are the most common respiratory infections leading to the hospitalization of infants. The current standard of care options is limited to treatments that provide symptomatic relief.
Enjaymo (sutimlimab) for hemolytic anemia in adult patients with cold agglutinin disease
Genzyme's Enjaymo (sutimlimab) received a positive CHMP opinion for treating hemolytic anemia in adult patients with cold agglutinin.
The approval was based on CADENZA and CARDINAL trials. In the CADENZA trial, the primary endpoints were the proportion of patients who met all the three components - improvement in hemoglobin ≥1.5 g/dL from baseline, avoiding transfusion from week 5 to week 26, and avoiding CAD-related therapies beyond what was permitted between Week 5 and Week 26. 73% of patients on Enjaymo met the primary endpoint compared to 15% of patients on placebo.
CARDINAL trial was a single-arm study aimed at assessing the safety and efficacy of Enjaymo. The primary endpoint includes the following components - improvement in hemoglobin ≥2g/dL from baseline or reaching ≥12 g/dL at the 26-week, avoiding transfusion from week 5 to week 26, and avoiding CAD-related therapies beyond what was permitted between Week 5 and Week 26. 54% of patients met the primary endpoint.
Livtencity (maribavir) received CHMP positive opinion for treating cytomegalovirus infection
Takeda's Livtencity (maribavir) received CHMP positive opinion for treating adult patients with cytomegalovirus (CMV) infection who are refractory to other treatments and have undergone hematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT).
The positive opinion was based on the SOLSTICE trial, which evaluated the safety and efficacy of Livtencity versus other antivirals.
Mycapssa (octreotide) for treating acromegaly
Amryt's Mycapssa (octreotide) received a CHMP position for the maintenance treatment of acromegaly, who has responded and tolerated with octreotide or lanreotide.
The CHMP positive opinion was based on MPOWERED Phase 3 Trial. The trial met the primary endpoint of showing non-inferiority versus injectable somatostatin receptor ligands (iSRLs). In patients who were stabilized with octreotide were treated with Mycapssa, 91% of patients maintained insulin-like growth factor 1 versus 100% of patients on iSRLs.
Pyrukynd (mitapivat) for treating pyruvate kinase deficiency
Pyrukynd (mitapivat) received CHMP positive opinion for treating pyruvate kinase (PK) deficiency in adult patients. Pyrukynd was a first-in-class oral PK activator and was recently approved by the US Food and Drug Administration (FDA).
The approval was based on ACTIVATE and ACTIVATE-T, in which it achieved its primary endpoint. In ACTIVATE trial, 40% of patients achieved hemoglobin response versus 0% of patients on placebo. In the Phase 3 ACTIVATE-T trial, 37% of patients achieved a transfusion reduction response. 22% of patients achieved transfusion-free during the fixed-dose period.
Zynlonta (loncastuximab tesirine) for treating relapsed or refractory diffuse large B-cell lymphoma
ADC Therapeutics and Swedish Orphan Biovitrum AB (Sobi)'s Zynlonta (loncastuximab tesirine) received CHMP positive opinion for treating relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
The CHMP positive opinion was based on LOTIS-2, a Phase 2 single-arm trial, which recruited adult patients with relapsed or refractory DLBCL who were treated with two or more prior lines of therapy. The overall response rate was 48.3%, the complete response rate was 24.1%, and the partial response rate was 24.1%.
The US Food and Drug Administration (FDA) granted accelerated approval for Zynlonta, the first CD19-targeted antibody-drug conjugate (ADC). It received approval in 2021.
EXTENSION OF INDICATIONS
Skyrizi (risankizumab) received CHMP positive opinion for moderate to severe Crohn's Disease.
AbbVie's Skyrizi received CHMP positive opinion for treating adults with moderate to severe Crohn's Disease. Skyrizi is the first IL-23 inhibitor that received a CHMP position opinion for treating Crohn's Disease.
The approval was based on ADVANCE, MOTIVATE (induction studies), and FORTIFY (maintenance study). Many patients achieved the co-primary endpoints of endoscopic response and clinical remission. In ADVANCE and MOTIVATE induction studies, the primary endpoint was met at week 12 compared to placebo. In the FORTIFY study, a significant portion of patients showed statistically significant improvement in the co-primary endpoints at week 52.
JUNE - CHMP POSITIVE OPINIONS
NEW INDICATION APPROVALS
VLA2001 COVID 19 VACCINE (Valneva)
CHMP has given a positive opinion for Valneva's VLA2001 COVID19 vaccine. Valneva announced that this is the first vaccine that received standard market authorization. The vaccine was approved for individuals of age 18 to 50 years.
Oncopeptides' Pepaxti for multiple myeloma (Oncopeptides)
Pepaxti (melphalan flufenamide), an antineoplastic agent, receives CHMP positive opinion for multiple myeloma.
Rayvow for migraine
Eli Lilly's Rayvow (lasmiditan) received a positive CHMP opinion for treating adult patients with migraine. Rayvow is available under three different strengths - 50 mg, 100 mg, and 200 mg film-coated tablets.
Roctavian for Hemophilia A
BioMarin's Roctavian (valoctocogene roxaparvovec) received CHMP positive opinion to treat adult patients with Hemophilia A. Roctavian is the first gene therapy approved in Europe for hemophilia A. The CHMP positive opinion was based on the GENEr8-1 Phase 3 study.
Scemblix for chronic myeloid leukemia
Novartis' Scemblix (asciminib) received CHMP positive for treating adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). It is indicated for patients who were previously treated with two or more tyrosine kinase inhibitors (TKIs).
The incidence of CML in Europe is 6,300. A significant percentage of patients experience intolerance or resistance to TKIs.
The approval was based on the Phase III ASCEMBL trial; the major molecular response rate in patients treated with Scemblix (asciminib) vs. Bosulif (bosutinib) was 25.5% vs. 13.2%.
Sunlenca for HIV
Gilead's Sunlenca (lenacapavir) received a positive CHMP opinion for human immunodeficiency virus type 1 infection. The approval was based on the CAPELLA trial - the viral load decrease of at least 0.5 log copies/ml was observed in 88% of patients versus 17% in patients on placebo.
Vyvgart for generalized myasthenia gravis
Vyvgart (efgartigimod alfa) received a positive CHMP opinion for treating adult patients with an anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG).
INDICATION EXTENSIONS
Crysvita for FGF23-related hypophosphatemia
Kyowa Kirin announced that the CHMP had given a positive opinion for Crysvita (burosumab) to treat children, adolescents, and adult patients with FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors (PMTS).
Enhertu for breast cancer
Enhertu (trastuzumab deruxtecan) achieved CHMP positive opinion for treating adult patients with HER2-positive breast cancer who were previously treated with one or more HER2-based treatment regimens. Previously, it got approval for adult patients treated with two or more HER2-based regimens.
Lonquex for neutropenia
Lonquex (lipegfilgrastim) received CHMP positive opinion for reducing neutropenia or incidence of febrile neutropenia in children of 2 years old and adults. Previously, it was approved only for adults.
Rinvoq for active Non-Radiographic Axial Spondyloarthritis
AbbVie announced that the CHMP gave a positive opinion for Rinvoq to treat active non-radiographic axial spondyloarthritis. The approval was based on the Phase 3 SELECT-AXIS 2 study, in which Rinvoq met the primary endpoint of ASAS40 response at week 14.
Zerbaxa for intra-abdominal infections, acute pyelonephritis; complicated urinary tract infections
Merck's Zerbaxa (ceftolozane / tazobactam) received CHMP positive opinion for treating pediatric patients with intra‑abdominal infections, acute pyelonephritis, and complicated urinary tract infections. Previously, the product was only approved for adult patients.
MAY - CHMP POSITIVE OPINIONS
NEW INDICATION APPROVALS
Cevenfacta (eptacog beta (activated)) received a positive CHMP opinion for treating bleeding episodes in patients with congenital hemophilia. Laboratoire Francais du Fractionnement et des Biotechnologies was the market authorisation holder.
PTC Therapeutics' Upstaza (eladocagene exuparvovec) received CHMP positive opinion for pediatrics with aromatic L‑amino acid decarboxylase (AADC) deficiency. AADC is a fatal genetic disease and causes severe disabilities, including mental, physical, and behavioral. The approval is based on the clinical trial conducted in Taiwan; pediatrics who showed no motor milestones showed several developmental activities.
CHMP positive opinion was given to Genzyme's Xenpozyme (olipudase alfa) for treating non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD). The approval was based on the clinical trials in which Xenpozyme demonstrated improvement in non-CNS clinical manifestations in adult and pediatric patients with ASMD.
EigerBio's Zokinvy (lonafarnib) received a positive CHMP opinion for treating patients with progeroid syndromes. In 62 patients from two single-arm cohorts, treatment with Zokinvy increased the lifespan compared to historical data.
APRIL 2022 - CHMP OPINIONS
NEW INDICATION APPROVALS
CHMP gave a positive opinion for Amryt Pharmaceuticals' Filsuvez (birch bark extract) for treating epidermolysis bullosa (EB). EB is a rare skin disorder impacting pediatric and adult patients. The approval is based on the Phase 3 EASE trial.
Roche's Lunsumio (mosunetuzumab) received a positive CHMP opinion for treating follicular lymphoma patients who were previously treated with two other therapies. The approval was based on the GO29781 study, in which Lunsumio showed high complete response rates. After a median follow-up of 18.3 months, the CR rate was 60%, and progression-free survival was 17.9 months. The median duration of response in patients who responded was 22.8 months.
CHMP gave a positive opinion for Novartis' Tabrecta (capmatinib) for treating patients with METex14 advanced non-small cell lung cancer. The approval was based on the Phase II GEOMETRY mono-1 study. In all the patients, the median duration of response was 9.7 months; the median overall survival was 13.6 months in the previously treated population.
INDICATION EXTENSIONS
Bydureon (exenatide) was extended to the indication for pediatric patients (adolescents and children aged ten years and above).
Elonva (corifollitropin alfa) received an indication extension for treating adolescent males (14 years and older) with hypogonadotropic hypogonadism.
Keytruda got a positive opinion for triple-negative breast cancer. Keytruda was indicated in combination with chemotherapy as neoadjuvant treatment, followed by continuation after surgery as monotherapy.
NovoSeven (eptacog alfa (activated)) received a positive CHMP opinion for severe postpartum hemorrhage.
Tecentriq got a positive opinion for early-stage non-small cell lung cancer (NSCLC).
Yescarta received a positive opinion for follicular lymphoma after three or more lines of systemic therapy.
NEGATIVE OPINIONS
Biogen has withdrawn the market authorization application of Aduhelm (aducanumab), a medicine indicated for treating Alzheimer's disease.
ARS Pharmaceuticals withdrew the application of Neffy, a treatment indicated for treating allergic reactions, including anaphylaxis.
Orphazyme withdrew the market authorization application of its Niemann-Pick disease type C therapy, Miplyffa (arimoclomol).
MARCH 2022 - CHMP POSITIVE OPINIONS
Mar 25, 2022
Janssen's Carvykti received CHMP positive opinion for treating relapsed and refractory multiple myeloma patients.
CHMP gave position for treating Carvykti (ciltacabtagene autoleucel) for treating relapsed and refractory multiple myeloma.
The approval was based on a single-arm, open-label study in 113 adult patients with relapsed and refractory multiple myeloma. The patients were previously treated with three other therapeutic options. 84% of patients had a durable response, and 69% showed a complete response.
AstraZeneca's COVID-19 medicine Evusheld received CHMP positive opinion
AstraZeneca's Evusheld received CHMP positive COVID-19 in adults and adolescents from 12 years. The approval was based on the trial, which enrolled more than 5,000 people. The risk of COVID-19 infection was reduced by 77% after two injections of 150 mg tixagevimab and 150 mg cilgavimab.
CHMP Opinions - January 2022
BMS' Breyanzi (isocabtagene maraleucel) received a positive CHMP for treating relapsed or refractory diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma. Breyanzi is gene therapy indicated in patients previously treated with two other treatments. The drug has previously received PRIME designation from EMA. The positive opinion is based on TRANSCEND NHL 001 and TRANSCEND WORLD study.
Pfizer's Paxlovid received CHMP positive opinion for treating COVID-19. The approval is based on the decreased hospitalizations and deaths in patients infected with COVID19 infection. After a month of treating with Paxlovid, 0.8% of patients on Paxlovid were hospitalized compared to 6.3% of patients on placebo. No deaths were reported in the Paxlovid group compared to 12 deaths in patients on placebo. Most of the patients enrolled in this study have Delta variant; however, laboratory studies showed that the drug is also effective against the Omicron variant.
Further, there are eight indication extensions.
Ayvakyt: Blueprint Medicines Ayvakyt (avapritinib) received an indication extension for aggressive systemic mastocytosis (ASM). Previously, it is approved for unresectable or metastatic gastrointestinal stromal tumors.
Briviact: Briviact (brivaracetam) received positive opinion for two years and older patients with epilepsy
Dupixent: Dupixent (dupilumab) received positive opinion for indication extension. Now, Dupixent can be used for pediatric patients for treating asthma who are inadequately controlled with medium to high dose inhaled corticosteroids (ICS) in addition to other products.
Jardiance: Jardiance (empagliflozin) was previously approved for heart failure - it is now received a positive opinion for all patients with symptomatic chronic heart failure.
Lacosamide UCB: Lacosamide UCB received a positive opinion for approval for two years and older patients with epilepsy. Previously it was approved for four years and older patients.
Senshio: Senshio received a positive opinion for moderate to severe symptomatic vulvar and vaginal atrophy (VVA).
Tecfidera: Biogen's Tecfidera (dimethyl fumarate) received a positive CHMP for patients aged 13 years and older. Previously, it was approved for adults only.
Vimpat: UCB's Vimpat (lacosamide) received a positive CHMP for two years and older pediatrics. Previously, it was approved for patients of 4 years and older.
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