Entities that already have received letters from the agency raising concerns about marketing unapproved products may not necessarily be the first to face enforcement actions, CBER official says.
The list of sponsors suspected of marketing unapproved human cell, tissue, and cellular and tissue-based products (HCT/P) that previously received notices from the US Food and Drug Administration may not represent the agency’s priority now that enforcement discretion has ended.
The most recent extension of the agency’s discretion policy expires 31 May and was not renewed. Sponsors of HCT/Ps without...
Madrigal Pharmaceuticals’ resmetirom could become the first approved treatment for non-cirrhotic metabolic dysfunction-associated steatohepatitis in the EU, if the European Medicines Agency issues a positive opinion for the drug later this week.
The Pediatric Advisory Committee will review postmarketing safety for three vaccines and the oncologic drugs panel will consider the return of GSK’s belantamab mafodotin. An FDA notice about a 30 July meeting on Capricor’s deramiocel for Duchenne muscular dystrophy was withdrawn.
Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.
Cell and gene therapy developers may not have to contend with HHS Secretary Robert F. Kennedy Jr.’s animosity like the traditional pharma industry, but his disinterest in distinguishing between the good and bad actors in the space could backfire.
