European Medicine Agency (EMA)
Following a positive opinion from the CHMP, the European Commission (EC) has approved Otsuka and Lundbeck’s Abilify Maintena (aripiprazole) as a once-every-two-months long-acting injectable formulation for the maintenance treatment of schizophrenia. Lundbeck press release.
The European Medicines Agency (EMA) has granted priority medicines (PRIME) status to 89bio’s pegozafermin for the treatment of metabolic dysfunction-associated steatohepatitis (MASH). 89bio press release.
US Food and Drug Administration (FDA)
The FDA approved the following drugs:
lahere (mirvetuximab soravtansine-gynx, ImmunoGen) for adult patients with FRα positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. FDA press release.
Winrevair (sotatercept, Merck) for adults with pulmonary arterial hypertension. Merck’s press release.
Vemlidy (tenofovir alafenamide, Gilead) has been granted an indication extension to a younger patient population. The new approval allows Vemlidy to be administered to patients as young as 6 years old with chronic hepatitis B infection. Gilead press release.
On a less glamourous note, the FDA declined to approve Regeneron Pharmaceuticals’ BLA for odronextamab in relapsed/refractory follicular lymphoma (FL) and in R/R diffuse large B-cell lymphoma (DLBCL). The FDA requires Regeneron to make further progress on confirmatory trials. Regeneron 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 March 25 and March 31, 2024:
- Axsome Therapeutics announced that AXS-12 (reboxetine) achieved the primary endpoint in its Phase 3 trial in patients with narcolepsy. Axsome press release.
- Stoke Therapeutics shared positive data from two Phase 1/2a Open Label studies of STK-001 (antisense oligonucleotide, ASO) in patients with Dravet syndrome. Stoke press release.
- The TUDCA-ALS consortium announced that tauroursodeoxycholic acid (TUDCA) failed to meet the primary and secondary endpoints in its Phase 3 study in patients with amyotrophic lateral sclerosis (ALS). TUDCA-ALS press release.
- BMS shared positive results from the Phase 3 trial of Krazati (adagrasib) in patients with pretreated locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring a KRASG12C mutation. BMS press release.
- Moderna reported positive interim results from the Phase 3 trial of its next-generation COVID-19 vaccine candidate (mRNA-1283). mRNA-1283 elicited a higher immune response against the SARS-CoV-2 virus compared to Spikevax (mRNA-1273.222). Moderna press release.
- Beckley Psytech announced initial positive findings from its Phase 2a trial of BPL-003 (psychedelic compound) in treatment-resistant depression (TRD). Beckley Psytech press release.