FDA
Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s approach to pre-approval inspections.
FEATURED STORIES
The FDA is becoming deeply compromised and increasingly at risk of being permanently transformed in ways contrary to its mission, history and culture.
The status could support staged transitions to new manufacturing processes, potentially mitigating some risks of high-stakes switches.
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.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
Blujepa is also approved for uncomplicated urinary tract infection in females 12 years and older.
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.
The FDA has informed Merck, Sanofi and AstraZeneca of the safety investigation into their RSV antibodies, though it remains unclear what regulatory action, if any, will be taken.
The Commissioner’s National Priority Voucher program was launched in June to shorten review times for companies that align with various national priorities, such as improving domestic drug production and supply.
Saol Therapeutics received a complete response letter for its pyruvate dehydrogenase complex deficiency treatment a week after the FDA unveiled its Rare Disease Evidence Principles program. On Dec. 18, in a Type A meeting, the biotech will attempt to convince the agency that its drug fits perfectly into the framework.
For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products.
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.