FDA
The approval of Tregzi—the first regulatory greenlight for Orca Bio—was based on a Phase 3 study in which patients on the therapy were twice as likely to survive without cancer relapse and without chronic GVHD compared with conventional allogeneic transplant.
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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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The FDA has greenlit Travere Therapeutics’ Filspari as the only available treatment for focal segmental glomerulosclerosis despite the drug’s loss to Sanofi’s Avapro in a Phase 3 study.
Some 30% of clinical trials that are mandated to report their findings have not posted results to clinicaltrials.gov, the federal government’s public repository for studies, according to internal data from the FDA.
The FDA has gained a reputation during the past year for being inconsistently flexible, particularly when it comes to rare diseases. Executives at Rezolute and CERo Therapeutics recently had positive interactions with the agency, in which they told BioSpace reviewers have been “collaborative” and “curious.
Since the FDA began publishing its rejections of drug approval filings in July last year, companies have become more forthcoming about the details of agency decisions in their own disclosures, according to biopharma and regulatory analysts.
The FDA in a complete response letter to Replimune maintained its original objection to the single-arm trial the biotech used to support the application for RP1.
A Louisiana court on April 7 asked the FDA to complete its internal review of mifepristone’s safety and gave the agency six months to provide the court with an update on the investigation.
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.
If the Trump administration’s proposal passes, the FDA’s budget will be more than $200 million bigger in 2027, with plans to launch new programs that expedite drug development, boost national security and promote “radical transparency.”
The upcoming FDA decision for Replimune’s advanced melanoma drug could be a litmus test for the agency’s future regulatory decision-making, analysts say, with implications stretching well beyond one company.
While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external controls—the application of this flexibility appears to be inconsistent. One former regulator says the situation is more nuanced.