Regulatory
A gene therapy being developed by Eikonoklastes Therapeutics to treat ALS received Orphan Drug Designation, the company announced Wednesday.
Ipsen will seek a new approval for Onivyde as a treatment for metastatic pancreatic ductal adenocarcinoma (mPDAC) after posting positive Phase III progression survival data.
The FDA deemed AstraZeneca’s PT027 to have a favorable risk-benefit profile for treating asthma in patients aged 18 or older but was less keen on supporting the drug in younger populations.
The FDA presented briefing documents Monday outlining what it called “uncertainties” in the data presented by Veru regarding its COVID-19 antiviral. An advisory committee will meet Wednesday.
Verve Therapeutics’ base editing program has been forced into a detour after the FDA placed a hold on its investigational new drug application. Beam has also posted its own regulatory update.
Akebia Therapeutics has submitted a Formal Dispute Resolution Request with the FDA regarding the rejection of vadadustat in anemia due to chronic kidney disease.
Sanofi and AstraZeneca won European approval for the first preventative RSV treatment for a broad population of newborns and infants.
The FDA is gearing up for deadlines and PDUFA dates in mid-November with Seagen, Provention, Clarus and Tolmar, and Ligand and Travere.
Apellis is submitting additional 24-month efficacy data to supplement the NDA for its intravitreal candidate pegcetacoplan for GA secondary to AMD.
Abeona Therapeutics will head to the FDA next year for the potential approval of its experimental recessive dystrophic epidermolysis bullosa (RDEB) therapy following positive Phase III data.
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