FDA
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval.
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Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator.
A week into his tenure as head of the FDA’s Center for Drug Evaluation and Research, experts agree that Rick Pazdur is the “ideal fit” to stabilize the agency. And, according to one ex-FDA official, if his CBER counterpart Vinay Prasad tries to supersede Pazdur’s authority, “there will be hell to pay.”
Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program.
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In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for new approach methologies (NAMs) development.
Nearly 90% of senior leaders who were at the FDA a year ago are no longer with the agency, a BioSpace analysis shows. None remain from the Office of the Commissioner.
For certain monospecific antibodies, three-month toxicology studies plus other supportive evidence will suffice, eliminating the need for six-month testing.
Newly appointed Center for Drug Evaluation and Research director Richard Pazdur has filed retirement papers with the agency—three weeks after accepting the role—amid clashes with Commissioner Marty Makary over certain FDA programs.
With no evidence to support the claim that 10 children died due to COVID-19 vaccines, experts unpack the impact of CBER chief Vinay Prasad’s leaked vaccine memo.
Agentic AI can help FDA staff manage meetings, conduct pre-market reviews and validate reports, among other tasks, though the agency emphasized that using this technology is optional for its employees.
The alleged deaths were detected by the FDA’s Vaccine Adverse Event Reporting System, reports from which “generally cannot be used to determine” causation or even contribution, according to the agency.
If approved, Ascendis’ TransCon CNP would become the second therapy for achondroplasia, challenging BioMarin’s Voxzogo.
The FDA’s docket in December includes decisions for two big biologic franchises: BMS’s Breyanzi and Amgen’s Uplizna.
Analysts at Guggenheim Partners expect Voyxact to see “broad commercial uptake” given its relatively broad label compared with previous accelerated approvals for IgA nephropathy.