FDA
As Sangamo runs out of cash, Eli Lilly and Astellas have emerged as stalking horse bidders for key assets, including a Fabry gene therapy currently being submitted for potential FDA approval.
FEATURED STORIES
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 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.
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After being rejected in June 2022, GSK paid Spero Therapeutics $66 million for an exclusive worldwide license to develop and commercialize the drug. It’s the second oral antibiotic GSK has brought to market since the beginning of last year.
After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application.
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.
The FDA rejected bitopertin in February amid reports of skepticism from former CBER director Vinay Prasad, who has since departed the agency.
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.
A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.