Drug News: 19 to 25 November 2024

Intriguing Science

We enjoyed listening this week to the nature podcast, explaining how the squid’s ability to shoot ink inspire prototypes of drug delivery (insulin) in the intestine, needle-less. You want to know more: Nature Podcast.

European Medicine Agency

The PRAC (EMA safety surveillance committee) is due to meet this week. Stay tuned!

Hympavzi (marstacimab) was approved by the European Commission (EC) for the routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with severe hemophilia A without FVIII inhibitors or severe hemophilia B without FIX inhibitors. Pfizer press release.

US Food and Drug Administration (FDA)

1. Ziihera (zanidatamab-hrii, Jazz Pharmaceuticals) received FDA accelerated approval for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC). Jazz Pharmaceuticals press release.
2. Fludarabine Phosphate Injection (fludarabine phosphate, Sandoz) received an updated label as part of a combination to treat adults with B-cell chronic lymphocytic leukemia (CLL) and for the treatment of adults with B-cell CLL who have not responded to or whose disease has progressed during treatment with at least one alkylating-agent containing regimen. FDA press release.
3. Attruby (acoramidis, BridgeBio Pharma) was approved for the treatment of adults with Transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. BridgeBio Pharma press release.
4. Izervay (avacincaptad pegol intravitreal solution, Astellas Pharma) supplemental New Drug Application (sNDA) was rejected for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The complete response letter (CRL) cited statistical concerns related to proposed labeling language. Astellas 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 18th and 24th of November 2024:

1. Neurogene reported a treatment related SAE in its Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome. A high-dose cohort participant is critical condition after developing systemic hyperinflammatory syndrome, a risk associated with adeno-associated virus (AAV)-based therapies. Neurogene paused high-dose cohort enrollment and continues with the low dose cohort. Neurogene press release.
2. GSK reported that the Phase 3 trial evaluating linerixibat in adults with cholestatic pruritus (relentless itch) associated with primary biliary cholangitis (PBC) met its primary endpoint. GSK press release.
3. Biogen and UCB shared positive results from the Phase 3 study of dapirolizumab pegol (DZP) in patients with moderate-to-severe systemic lupus erythematosus (SLE). Biogen press release.
4. Merck announced positive results from the Phase 3 trial of subcutaneous pembrolizumab for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC). Merck press release.
5. NewAmsterdam Pharma reported positive data from the Phase 3 trial of obicetrapib and ezetimibe fixed-dose combination in adult patients with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD), inadequately controlled on maximally tolerated lipid-lowering therapy. NewAmsterdam press release.
6. Sage Therapeutics announced that the Phase 2 study of dalzanemdor (SAGE-718) in participants with cognitive impairment (CI) associated with Huntington’s Disease (HD) did not meet its primary endpoint. Based on these results, Sage will discontinue the development of dalzanemdor. Sage press release.
7. Johnson & Johnson shared positive results from the Phase 3 study of icotrokinra (JNJ-2113) in patients 12 years of age and older with moderate to severe plaque psoriasis (PsO). J&J press release.