FDA
Aiming to protect patients, the FDA sent lawmakers a wish list of legislative proposals intended to clarify and expand its oversight of updates to approved drug production processes.
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The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.
The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher.
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The FDA Adverse Event Reporting System, which Commissioner Marty Makary called “clunky,” previously published updates on a quarterly basis.
The drug, for hereditary angioedema, is Ionis’ second wholly owned asset.
Vanda claims that two generics to its sleep disorder drug Hetlioz were approved despite inadequate data and is requesting that FDA Commissioner Marty Makary revisit the decision.
Among the problems cited were cat hair, bacterial contamination and instrument defects.
Stealth BioTherapeutics originally submitted elamipretide for approval in January 2024, only to receive a rejection in May of this year. In addition to accepting the biotech’s resubmission, the FDA has promised to take action on the candidate by Sept. 26.
The delays, first revealed in Regeneron’s Q2 report, were chalked up to manufacturing issues at Novo Nordisk-owned plants.
In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.
The FDA recommends that companies use overall survival as a primary endpoint for clinical trials where feasible. The new guidance follows the surprising return of CBER Head Vinay Prasad, who has previously argued for prioritizing OS.
Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset.
The small molecule, vatiquinone, had already flunked a Phase III trial, but the company pushed ahead with an approval bid anyway.