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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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.
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
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.
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Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 for Pompe disease.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
Companies who run their early-stage clinical development outside the U.S. would “experience higher fees” according to an FDA proposal made during the negotiation process for the eighth cycle of the user fee program.
Last week, Center for Biologics Evaluation and Research Director Vinay Prasad claimed in an internal memo—without providing evidence—that COVID-19 vaccines were responsible for the deaths of 10 children between 2021 and 2024.
Høeg is the fifth person to lead the Center for Drug Evaluation and Research this year.
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.
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.