FDA
The FDA intends to place a black box label—its most serious warning—on COVID-19 vaccines, according to reporting by CNN. It is unclear if the warning would apply to Moderna’s, Pfizer/BioNTech’s and Novavax’s shots, or to all age groups.
Generalized myasthenia gravis is Uplizna’s second new indication this year, after the FDA cleared the anti-CD19 antibody for IgG4-related disease in April.
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 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.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
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.
The agency’s sweeping rollout and staff challenge underscore rising momentum behind agentic AI: advanced, multiagent systems now fueling early pilots in medical writing, patient engagement and regulatory workflows across the industry.
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.
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.
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