Notes from NPC Accelerator Meeting – 5-26-21

Sean Kassen
Joslyn Crowe
Phil Marella
Chris Andrews
Cristin Davidson
Dave Zook


Mandos acquisition of Adrabetadex – Discussed outreach with Mandos and connecting them to the community. We must wait until the bankruptcy court approves the deal

Orphazyme update – PDUFA date for Arimoclomol is June 17. Waiting to learn more about the pending review/approval. NNPDF helped to get letters to send to the FDA.

Edenbridge and Miglustat – Edenbridge is trying to get an indication for NPC disease. The NTA is helping them gather data for their communications with the FDA.

FDA – update on efforts to interact with the FDA. We will be meeting with other rare disease groups that also are trying to get a number of therapies approved by the FDA and sharing best practices.

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