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IntraBio Media Release | March 26, 2024

IntraBio Announces U.S. FDA Accepts New Drug Application for IB1001 for the Treatment of Niemann-Pick disease type C


Read the full announcement from IntraBio here:



IntraBio has announced that the U.S. FDA has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The NDA for IB1001 has been granted Priority Review with a PDUFA target action date set for September 24, 2024.


The application is based on positive results from the IB1001-301 Phase 3 Pivotal Trial, which demonstrated improvement in neurological signs and symptoms, functioning, and quality of life in adult and pediatric patients with NPC.


The NDA also includes data from a multinational Phase IIb trial showing improvements in symptoms and quality of life in NPC patients. If approved, IB1001 is expected to address the high unmet medical need for NPC patients.

 

To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."


Ways to support NP-C in Australia:

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