Drug News 15 to 31st July 2024: approvals, clinical trials reported data

European Medicine Agency (EMA)

The EMA’s Committee for Medicinal Products for Human Use (CHMP) held their monthly meeting on the week from the 22nd to the 25th of July. Here are the key takeaways:

1. Vyloy (zolbetuximab, Astellas) received a recommendation for marketing authorization for the treatment of gastric or gastro-esophageal junction adenocarcinoma.
2. Yuvanci (macitentan / tadalafil, Johnson & Johnson) received a positive opinion for the treatment of pulmonary arterial hypertension.
3. Anzupgo (delgocitinib, LEO Pharma) received a recommendation for marketing authorization for the treatment of moderate to severe chronic hand eczema in adults
4. Vevizye (ciclosporin, Novaliq) received a positive opinion for the treatment of adult patients with moderate to severe dry eye disease unresponsive to tear substitutes.
5. Iqirvo (elafibranor, Ipsen Pharma) received a recommendation for marketing authorization for the treatment of primary biliary cholangitis
6. Loqtorzi (toripalimab, TMC Pharma) received a positive opinion for the treatment for the treatment of nasopharyngeal carcinoma and esophageal squamous cell carcinoma.
7. Kayfanda (odevixibat, Ipsen Pharma) received a positive opinion under exceptional circumstances for the treatment of cholestatic pruritus in patients with Alagille syndrome.
8. Finally, Leqembi (lecanemab, Eisai and Biogen) received a negative opinion for the treatment of Alzheimer’s disease.

The committee adopted positive opinions for six biosimilar medicines: Eksunbi (ustekinumab, Samsung Bioepis), Fymskina (ustekinumab, Formycon), Otulfi (ustekinumab, Fresenius Kabi), Ituxredi (rituximab, Dr. Reddy’s Laboratories), Ranibizumab Midas (ranibizumab, MIDAS Pharma) and Tuznue (trastuzumab, Prestige Biopharma). CHMP meeting highlights.

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US Food and Drug Administration (FDA)

The FDA did take the following decisions during the 2nd half of July 2024:

Voquezna (vonoprazan, Phathom Pharmaceuticals) label expansion was approved by the FDA for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD) in adults. Phantom press release [pdf].

The FDA rejected the New Drug Application (NDA) for OX124 (naloxone, Orexo) as a rescue treatment for opioid overdose. The Complete Response Letter (CRL) indicated the need for an additional Human Factors (HF) study and more technical data on the final commercial product. Orexo press release.

Brineura (cerliponase alfa, BioMarin) received a label expansion approval from the FDA to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. BioMarin press release.

Leqselvi (deuruxolitinib, Sun Pharma) was approved by the FDA for the treatment of adults with severe alopecia areata. Sun Pharma press release [pdf].

Epysqli (eculizumab-aagh, Samsung Bioepis) was approved as a biosimilar to AstraZeneca’s Soliris (eculizumab) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Samsung Bioepis press release.

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Data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data in second half of July 2024 per therapeutic area:

Neurology

1. Biogen and Sage Therapeutics announced that the Phase 2 study of SAGE-324 (BIIB124) for the treatment of essential tremor (ET) failed to meet its primary endpoint. Biogen press release.
2. Teva Pharmaceuticals reported positive data from the Phase 3 trial of Ajovy (fremanezumab) for the prevention of episodic migraines in children and adolescent patients aged six to 17 years. Teva press release.
3. Azafaros reported positive topline results from the Phase 2 trial of nizubaglustat to treat patients with either GM2 gangliosidosis or Niemann-Pick disease type C (NPC). Azafaros press release.

Oncology

1. Jaguar Health announced that the Phase 3 trial of its anti-diarrheal drug Mytesi (crofelemer) in cancer patients did not meet its primary endpoint. Jaguar Health press release.
2. Bayer announced that the Phase 3 trial evaluating Nubeqa (darolutamide) in patients with metastatic hormone-sensitive prostate cancer met its primary endpoint. Bayer press release.
3. AVEO Oncology announced that the Phase 3 trial of Fotivda (tivozanib) plus Opdivo (nivolumab, BMS) in patients with advanced metastatic renal cell carcinoma (RCC) did not meet its primary endpoint. Of note, the trial’s control arm using Fotivda as monotherapy demonstrated a clinically meaningful outcome in median progression free survival (PFS). AVEO press release.

Cardio-metabolic disorders

1. Calliditas Therapeutics reported that the Phase2Ib trial of setanaxib in patients with primary biliary cholangitis (PBC) and elevated liver stiffness met its primary endpoint. Callidatis press release.
2. Gan & Lee Pharmaceuticals announced positive results from the Phase 2b study of their glucagon-like peptide 1 (GLP-1) receptor agonist GZR18 in adults with obesity in China. Gan & Lee press release.
3. Lexeo Therapeutics announced positive interim data from the Phase 1/2 study of LX2006 for the treatment of Friedreich ataxia cardiomyopathy. Lexeo press release.
4. Roche announced that the long-term safety and efficacy extension study of Vabysmo (faricimab) in people with diabetic macular edema (DME) met all primary endpoints. Roche press release.

Other indications:

1. Merck reported positive topline results from its Phase 2b/3 trial of esrovimab as a prophylactic monoclonal antibody to protect infants from respiratory syncytial virus (RSV) disease. The trial met its primary safety and efficacy endpoints. Merck press release.
2. Ionis Pharmaceuticals published positive results from the Phase 1/2 study of ION582 for the treatment of Angelman syndrome (AS). The company plans to start a Phase 3 trial in the first half of 2025. Ionis press release.
3. ViiV Healthcare shared positive results from the Phase 4 trial evaluating the two-drug regimen Dovato (dolutegravir/lamivudine) compared to the three-drug regimen Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide fumarate) for the treatment of HIV in people who are virologically suppressed and could benefit from treatment optimization.. ViiV press release.
4. Pfizer reported positive data from the Phase 3 trial of giroctocogene fitelparvovec to treat adults with moderately severe to severe hemophilia A. Pfizer press release.
5. Camurus reported positive results from the Phase 3 study of octreotide SC depot (CAM2029) in patients with acromegaly. Camurus press release.
6. Sanofi announced that the Phase 3 study of ALTUVIIIO (efanesoctocog alfa) in children with severe haemophilia A met its primary and secondary endpoints. Sanofi press release.
7. Paratek Pharmaceuticals reported positive results from the Phase 3 post-marketing study of Nuzyra (omadacycline) for the treatment of patients with moderate to severe community-acquired bacterial pneumonia (CABP). Paratek press release.