Rare diseases
After the FDA flagged patient deaths linked to Amgen’s rare disease drug Tavneos and called for its voluntary removal, the pharma recruited an independent data analysis from Duke researchers to help build the case for the drug’s continued market approval.
The discontinuation of Sanofi’s Phase 3 study for its complement inhibitor drug in chronic inflammatory demyelinating polyneuropathy delivers an ‘obviously disappointing’ blow to the broader drug class, according to William Blair.
The FDA rejected bitopertin in February amid reports of skepticism from former CBER director Vinay Prasad, who has since departed the agency.
The FDA has expanded the regulatory toolkit during President Donald Trump’s second term, adding new mechanisms for rare diseases while putting the Biden-era platform technology designation into action.
As the FDA tries to clarify its intent for former FDA Commissioner Marty Makary’s plausible mechanism framework for bespoke therapies, experts emphasize the importance of expanding its scope to encompass rare diseases that affect more than just one or a few individuals.
After discontinuing development of its lead asset, Fulcrum Therapeutics has provided more details on its restructure, with 48 of 57 employees headed out the door.
Travere Therapeutics will gain an exclusive license to the oral BTK inhibitor civorebrutinib, which analysts at Guggenheim Securities said could be “complementary” to the biotech’s IgA nephropathy drug Filspari.
For Peter Pitts, a former associate commissioner at the FDA, the appointment to the board of BrainStorm Cell Therapeutics is an opportunity to fulfill a promise he made long ago to a patient with ALS.
Edgewise Therapeutics will now focus on a handful of cardiovascular programs including EDG-7500 for hypertrophic cardiomyopathy thanks to the non-dilutive capital from France’s Servier.
After trial flops in spinal muscular atrophy, depression and bipolar disorder—and a costly rare disease drug rejection—Biohaven is undergoing a reset, recasting its former SMA candidate for obesity.
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