Beren Therapeutics announced that the FDA has accepted its NDA for adrabetadex for the treatment of Niemann-Pick disease type C (NPC) and granted Priority Review. This is an important milestone for families affected by this devastating disease.
Progress like this reflects years of research, persistence, and partnership across the rare disease community. We are grateful for companies like Beren who are committed to advancing potential therapies and helping move hope closer to reality for those living with NPC.
Momentum matters. And today, it feels tangible.