Regulatory
As the FDA unveils a parade of initiatives aimed at accelerating drug development for rare diseases, experts appeal for a consistent approval process that will support and further catalyze momentum.
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.
Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials.
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.
Høeg is the fifth person to lead the Center for Drug Evaluation and Research this year.
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.
Experts unpack the implications of CBER Director Vinay Prasad’s claim that COVID vaccines have caused 10+ child deaths; the 2025 Clinical Trials on Alzheimer’s Disease conference continues following two disappointing readouts; and Novo Nordisk’s amycretin yields promising weight loss results.
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