Drug News 23 Sept 2024, EMA, FDA approvals, new Phase 2 Phase 3 clinical data

European Medicine Agency

After positive opinions of the CHMP:

1. Vyloy (zolbetuximab, Astellas Pharma) was approved by the European Commission (EC) as a first-line treatment for adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma. Astellas press release.
2. Iqirvo (elafibranor, Ipsen) received conditional approval from the EC for the treatment of primary biliary cholangitis (PBC). Ipsen press release.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) held its monthly meeting from the 16th to 19th of September. Here are the key takeaways:

1. Elahere (mirvetuximab soravtansine, ImmunoGen) received a positive opinion for the treatment of adults with ovarian cancer, fallopian tube cancer, or primary peritoneal cancer.
2. Hetronifly (serplulimab, Henlius) received a positive opinion for the treatment of extensive-stage small cell lung cancer.
3. Hympavzi (marstacimab, Pfizer) received a positive opinion for the treatment of bleeding episodes in patients aged 12 years and older with severe haemophilia A or B.
4. Penbraya (meningococcal vaccine (recombinant, adsorbed), Pfizer) received a positive opinion for immunization against invasive meningococcal disease.
5. Afqlir (aflibercept, Sandoz) and Opuviz (aflibercept, Samsung Bioepis) received a positive opinion as biosimilars intended for the treatment of age-related macular degeneration.

Syfovre (pegcetacoplan, Apellis Pharmaceuticals) received a negative opinion for the treatment of geographic atrophy secondary to age-related macular degeneration.CHMP meeting highlights.

US Food and Drug Administration (FDA)

The FDA granted the following approvals:

1. Kisqali (ribociclib, Novartis) was approved by the FDA for the treatment of patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) stage II and III early breast cancer (EBC). Novartis press release.
2. Keytruda (pembrolizumab, Merck) was approved as a first-line treatment of unresectable advanced or metastatic malignant pleural mesothelioma (MPM). Merck press release.
3. Fasenra (benralizumab, AstraZeneca) was approved for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). AstraZeneca press release.
4. FluMist (influenza virus subtypes A and B vaccine, AstraZeneca) was for self- or caregiver administration. FDA press release.
5. Miplyffa (arimoclomol, Zevra Therapeutics) was approved for the treatment of Niemann-Pick disease, type C (NPC). Zevra press release.
6. Dupixent (dupilumab, Regeneron and Sanofi) was approved as an add-on maintenance treatment for adolescent patients aged 12 to 17 years with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). Regeneron press release.
7. Rybrevant (amivantamab-vmjw, Johnson & Jonshon) was approved for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or L858R substitution mutations. J&J press release.
8. Sarclisa (isatuximab, Sanofi) was approved in combination with bortezomib, lenalidomide, and dexamethasone (VRd) as a first line treatment option for adult patients with newly diagnosed multiple myeloma (NDMM) who are not eligible for autologous stem cell transplant (ASCT). Sanofi press release.

Vanda Pharmaceuticals received a CRL for Tradipitant in the treatment of adults with gastroparesis. Vanda press release (pdf). Caution: despite Vanda’s Press Release tone, please check the results of the pivotal study that did not meet its primary endpoint according the to phase 3 results press release.

New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the 16th of September and 22nd of September 2024:

Zentalis Pharmaceuticals announced that the FDA lifted the partial clinical hold on studies of azenosertib. Zentalis press release.

Boehringer Ingelheim announced that the Phase 3 study of nerandomilast in patients with idiopathic pulmonary fibrosis (IPF) met its primary endpoint. The company plans to submit a New Drug Application (NDA) to the FDA. Boehringer Ingelheim press release.

Incyte and Syndax Pharmaceuticals announced that the Phase 2 trial of Niktimvo (axatilimab-csfr) in adult and pediatric patients with recurrent/refractory active chronic graft-versus-host disease (GVHD) met its primary endpoint. Incyte press releases.

Merck and Daiichi Sankyo shared positive results from the Phase 3 trial of patritumab deruxtecan in patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC). Merck press release.

AstraZeneca reported positive data from the Phase 3 trial of Imfinzi (durvalumab) plus Imjudo (tremelimumab) in patients with unresectable hepatocellular carcinoma (HCC). AstraZeneca press release.

Aligos Therapeutics announced positive results from the Phase 2a study of ALG-055009 in patients with metabolic-dysfunction associated steatohepatitis (MASH). Aligos press release.

Sanofi shared positive results from the Phase 3 study of tolebrutinib in patients with non-relapsing secondary progressive multiple sclerosis (nrSPMS). Sanofi press release.

Roche announced that the Phase 3 study of Xofluza (baloxavir marboxil) met its primary endpoint in reducing the transmission of influenza viruses. Roche press release.