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Big News for APDS: FDA Grants Priority Review for Leniolisib in Younger Children
NASDAQ: PHAR Pharming Group has just shared exciting developments regarding their treatment for Activated PI3K-delta Syndrome (APDS). The U.S. Food and Drug Administration (FDA) has formally accepted the supplemental New Drug Application (sNDA) for leniolisib and, significantly, granted it Priority Review status. This is a pivotal moment for the APDS community, particularly for families managing…
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Rocket Pharmaceuticals | Unlocking the Potential: RP-L201 and its FDA Designations – A Breakthrough in Pediatric Medicine
NASDAQ: RCKT Rocket Pharmaceuticals has achieved significant milestones with their groundbreaking therapy, RP-L201. The FDA has granted several prestigious designations to RP-L201, including Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, Fast Track, and Orphan Drug designations. These designations highlight the immense potential of RP-L201 in treating pediatric patients and emphasize the urgent need for innovative…