Breakthrough Therapy Designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an New Drug Apllication submission. THOUSAND OAKS, Calif.– December 9, 2025 – Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation …
Category
Showing: 4 - 6 of 51 RESULTS
Niemann-Pick Type C News
Great Data On Adrabetadex/Cyclodextrin Released By Mandos Health
Wednesday, 9/10/25 – In advance of the 2025 American Neurological Association (ANA) meeting, Mandos Health releases two scientific abstracts with promising data for the treatment of NPC patients with Adrabetadex. The lead abstract, designated by ANA as an “Abstract of Distinction,” presents data showing a statistically significant survival benefit in patients with infantile-onset NPC who …
Zevra Therapeutics to Sell Priority-Review Voucher for $150 Million related to NPC drug approval
February 27, 2025 at 7:31 AM EST CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease …