The U.S. Food and Drug Administration on Thursday introduced the Rare Disease Evidence Principles (RDEP) to provide greater speed and predictability in the review of therapies intended to treat rare ...
Last week, FDA Commissioner Marty Makary, MD, MPH, announced plans to officially lower the standard for how many clinical trials are needed to approve a new drug -- from the long-standing expectation ...
Cintredekin besudotox targets IL-13 alpha-2 receptor in glioblastoma, aiming to spare healthy brain tissue while destroying cancer cells. Orphan drug designation offers benefits like lower fees and ...
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GSK’227, now known as risvutatug rezetecan ...
Not so for common diseases." The FDA, citing a report from the National Organization for Rare Disorders, said about two-thirds of orphan drugs were approved with one adequate and well-controlled trial ...
SEATTLE, Oct. 2, 2025 /PRNewswire/ -- Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing novel therapies for rare and difficult-to-treat cancers, today announced ...
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