Regulatory
Gossamer Bio announced Monday that it is discontinuing the development of its lymphoma candidate, GB5121, following two patient deaths in the Phase Ib/II STAR-CNS study.
HUTCHMED has completed the rolling submission of its New Drug Application to the FDA for fruquintinib, its candidate for refractor metastatic colorectal cancer.
A federal court sided in favor of Vanda Pharmaceuticals in its Freedom of Information Act case against the FDA regarding the CRL for its sleep drug label expansion.
In an earnings call Wednesday, bluebird bio revealed it is unlikely to meet its first-quarter goal to submit a Biologics License Application for sickle cell disease (SCD) gene therapy lovo-cel.
The FDA has granted an advisory committee meeting for BrainStorm Cell Therapeutics’ investigational ALS therapy, NurOwn, the company announced Monday.
The FDA is expected to make decisions on Emergent BioSolutions’ sNDA for OTC Narcan and Roche’s Polivy in frontline DLBCL.
Upstaza is the first targeted therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. PTC expects to submit a Biologics License Application to the FDA in the first half of 2023.
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 9-0 that tofersen’s effect on neurofilament light chain (NfL) could be a reasonable predictor of clinical benefit.
Peter Marks described the steps the agency is taking to advance the development of gene therapies for rare disorders. This could spell good news in the near future for Sarepta Therapeutics.
Karuna Therapeutics released data Monday from the Phase III EMERGENT-3 trial showing that KarXT met the primary endpoint, significantly improving symptom severity.
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