FDA
A surprising deal from Vertex Pharmaceuticals adds to Big Pharma’s acquisitive streak as Crinetics folds into the cystic fibrosis drugmaker. Meanwhile, IPOs and venture capital raises trend upward, but mostly for derisked companies. Plus, FDA decisions slow only slightly as the hunt for a permanent leader drags on.
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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 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.
Teams at facilities being developed by Eli Lilly, Regeneron and other companies will receive early technical guidance and additional perks from the FDA.
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
The vibe at BIO 2026 in San Diego last week was overwhelmingly positive, with attendees observing noticeable changes at the FDA and an uptick in dealmaking and IPOs. Plus, a top medical journal this week retracted a pivotal study for Amgen’s rare disease drug Tavneos, which has been in the FDA’s crosshairs since January.
Unicycive Therapeutics and Sobi received complete response letters for kidney disease and gout filings, respectively, after the FDA found fault with their manufacturing partners.
Vijay Kumar, acting director of CBER’s Office of Therapeutic Products, will leave his role at a tumultuous time for the FDA.
The delay is largely “benign” for Praxis Precision Medicines, according to Jefferies, which emphasized that the FDA did not flag safety or manufacturing issues.
Viridian Therapeutics’ Lumvoa is the first FDA-approved treatment for thyroid eye disease that includes data for both active and chronic forms of the illness.
Replimune’s resubmission for RP1 for melanoma comes after the departures of FDA leaders in place at the time of the drug’s first two rejections. The FDA expects to hold an advisory committee meeting in late July.
Policymaking at FDA has been anything but business as usual under the Trump administration, but former regulators cite the agency’s new investigational new drug pilot program as a sign of normalcy.