FDA
The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term clinical data for the asset.
FEATURED STORIES
Here’s how companies can ensure they’re in compliance with new requirements that go into effect in August.
As the World Health Organization initiates a new agreement for coordinating global responses to future pandemics, the future of vaccine development in the U.S. faces growing challenges, including waning funding and regulatory changes, that threaten next-gen COVID-19 vaccine candidates and pandemic preparedness more broadly.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli Lilly’s Alzheimer’s antibody Kisunla, Sarepta’s DMD gene therapy Vyondys 53 and Gilead’s HIV drug Sunlenca.
The FDA will allow a new dosing schedule for Eli Lilly’s Alzheimer’s drug Kisunla that could lessen a known side effect of the monoclonal antibody drug class that has led to several deaths.
BrainStorm Cell Therapeutics issued a statement Tuesday supporting a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose BLA was withdrawn in 2023. A Phase IIIb trial was scheduled to begin last month.
Ekterly’s road to approval was not a smooth one. Last month, the FDA informed KalVista it would not meet its PDUFA date due to resource constraints and reports surfaced that Commissioner Marty Makary tried to have the application rejected.
While it trails Johnson & Johnson’s Tecvayli, Regeneron still hopes Lynozyfic can differentiate in terms of dosing convenience and efficacy.
The safety update for Vyvgart rattled argenx’s shares on Monday, a reaction that analysts at William Blair said was “overdone.”
Analysts believe that Gilead’s new PrEP drug Yeztugo could reach peak sales of $4.5 billion. Not if GSK has anything to say about it.
Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”
The FDA’s clunky launch of Elsa, an AI tool to increase efficiency, has sparked concern from agency employees and outside experts.
The FDA delivered two notable approvals for RSV immunization, UroGen overcame a negative advisory committee vote to secure an approval in bladder cancer, and more key regulatory nods from the past month.