European Medicine Agency (EMA)
The EMA’s Committee for Medicinal Products for Human Use (CHMP) held their monthly meeting this week from June 24th to June 27th. Here are the key takeaways:
- Eurneffy (epinephrine, ARS Pharmaceuticals) received a positive opinion from the CHMP to become the first emergency treatment against allergic reactions that is administered as a nasal spray, not as an injection.
- Winrevair (sotatercept, Merck) received a recommendation for marketing authorization for the treatment of adult patients with pulmonary arterial hypertension.
- Balversa (erdafitinib, J&J) received a positive opinion for the treatment of patients with unresectable or metastatic urothelial carcinoma.
- mResvia (Respiratory Syncytial Virus (RSV) mRNA vaccine, Moderna) received a positive opinion for prevention of lower respiratory tract disease and acute respiratory disease caused by RSV in adults 60 years of age and older.
- Ordspono (odronestamab, Regeneron) received a recommendation for conditional marketing authorization for the treatment of follicular lymphoma and diffuse large B-cell lymphoma.
- Piasky (crovalimab, Roche) received a positive opinion for the treatment of paroxysmal nocturnal haemoglobinuria.
- Tauvid (flortaucipir, Eli Lilly) received a positive opinion for positron emission tomography (PET) imaging of the brain in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease.
- Steqeyma (Ustekinumab, Celltrion), a biosimilar for J&J’s Stellara received a positive opinion for the treatment of adult patients with moderately-to severely active Crohn’s disease, plaque psoriasis, pediatric plaque psoriasis and psoriatic arthritis.
- Syfovre (pegcetacoplan, Apellis) received a negative opinion for the treatment of geographic atrophy secondary to age-related macular degeneration. The company said that it plans to seek a reexamination of the opinion, with a final decision expected in the fourth quarter of this year. Apellis press release.
- Masitinib (masitinib, AB Science) received a negative opinion for the treatment of amyotrophic lateral sclerosis (ALS). The company said that it plans to seek a reexamination of the opinion. AB Science press release.
- Translarna (ataluren, PTC Therapeutics) once again received a negative opinion on renewing the marketing authorization as a treatment for Duchenne muscular dystrophy. CHMP meeting highlights.
US Food and Drug Administration (FDA)
- The FDA rejected the Biologics License Application (BLA) for HER3-DXd (patritumab deruxtecan, Merck and Daiichi Sankyo) for the treatment of adult patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC). The complete response letter (CRL) cited problems found during an inspection of a contractor’s manufacturing facility. Merck press release.
- Epkinly (epcoritamab-bysp, AbbVie) was granted accelerated approval by the FDA to treat patients with relapsed or refractory follicular lymphoma. AbbVie press release.
- The FDA approved Ohtuvayre (ensifentrine, Verona Pharma) for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adults. Verona press release.
- The FDA rejected the New Drug Application (NDA) for ABBV-951 (foscarbidopa/foslevodopa, AbbVie) for the treatment of motor fluctuations in adults with advanced Parkinson’s disease. The CRL cited observations discovered during an inspection of a third-party manufacturer listed in AbbVie’s drug application. AbbVie press release.
- The FDA rejected the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). The CRL cited Chemistry, Manufacturing, and Controls (CMC) issues.
New data from clinical trials (Phase 2, Phase 3)
- Novo Nordisk announced that the Phase 3 trial of ocedurenone in patients with uncontrolled hypertension and advanced chronic kidney disease failed to meet its primary endpoint. Novo Nordisk press release.
- Alnylam Pharmaceuticals shared positive results from the Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study met its primary and all secondary endpoints. Alnylam press release.
- G1 Therapeutics announced that the Phase 3 trial of Cosela (trilaciclib) in patients with metastatic triple-negative breast cancer (TNBC) failed to meet the primary endpoint. The company stated they will focus efforts on small cell lung cancer instead. G1 press release.
- Merck discontinued the Phase 3 trial evaluating xevinapant in patients with unresected locally advanced squamous cell carcinoma of the head and neck. The decision followed a pre-planned interim analysis performed by the study’s Independent Data Monitoring Committee, which found that the trial would unlikely meet its primary endpoint. Merck press release.
- AstraZeneca reported results from the Phase 3 trial of Imfinzi (durvalumab) in patients with non-small cell lung cancer (NSCLC) showing that the trial did not meet its primary endpoint. AstraZeneca press release.
- Sanofi reported shared positive data from the ongoing Phase 2 trial of its riliprubart in chronic inflammatory demyelinating polyneuropathy (CIDP) patients. Sanofi press release.
- Savara announced that the Phase 3 study of molgramostim in patients with autoimmune pulmonary alveolar proteinosis (aPAP) met its primary endpoint. Savara press release.
- Johnson & Johnson announced positive results from the Phase 3 study of nipocalimab in patients with generalized myasthenia gravis (gMG). J&J press release.
- Sanofi published positive results from a Phase 3 trial of Dupixent (dupilumab) in one to 11-year-old children with eosinophilic esophagitis (EoE). Sanofi press release.
- Wave Life Sciences announced positive results from its Phase 1b/2a trial of WVE-003, which is being developed as a potential disease modifying therapeutic for Huntington’s disease (HD). Wave Life Sciences press release.
