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.
When the variance can’t be modeled, even disciplined biotech investors stop deploying. Here’s the cheapest fix for biotech’s investability problem.
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Moderna appears to have aligned with the FDA ahead of an advisory committee meeting for its mRNA-based flu vaccine, which the regulator initially turned away in February; biotech IPOs are going gangbusters, including two new records raises in as many weeks; layoffs continue across biopharma; plus much more.
The Vaccines and Related Biological Products Advisory Committee will meet June 18 to discuss Moderna’s seasonal flu vaccine mRNA-1010 after the FDA initially refused to accept the application in February.
Sanofi makes no mention of the Commissioner’s National Priority Voucher. Tzield was awarded the ticket in October 2025, but Sanofi requested withdrawal from the program after former CDER head Tracy Beth Høeg reportedly expressed skepticism of the drug.
DemeRx is launching the first U.S. clinical trial of an ibogaine-derived drug candidate, marking a pivotal moment for a controversial psychedelic long sidelined by safety concerns.
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.
The FDA must provide consistent and predictable regulatory frameworks if the U.S. is to maintain its leadership in gene therapy, one of the most consequential therapeutic fields of our generation.
The FDA hasn’t been transparent and open enough with how it has implemented the Commissioner’s National Priority Voucher program, patient, industry and trade groups said Thursday.
FDA inspectors found that Medline, which raised billions of dollars last year, failed to prevent repeat bacterial contamination of finished drug products.