Pharmaceutical Technology on MSN

FDA accepts Beren Therapeutics’ NDA for Niemann-Pick disease

The submission includes data indicating a meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.
Business Insider

Zevra Therapeutics Presents Positive New Real-World Data on MIPLYFFA® in Patients with Neimann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium™

CELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
Seeking Alpha

Zevra Therapeutics Celebrates and Supports Global Niemann-Pick Disease Awareness Day on October 19th and Niemann-Pick Disease Awareness Month Throughout October

CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick ...
ALZFORUM

Multi-omics Spot Differences Between Pick’s and Alzheimer’s Diseases

In Pick’s disease, as in Alzheimer’s disease and frontotemporal dementia, tangled tau spells trouble. In this subtype of FTD, abnormal clumps of three-repeat tau called Pick bodies abound in neurons.
Morningstar

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C

--If approved, adrabetadex would be the only therapy to directly target the underlying pathophysiology of Niemann-Pick disease type C (NPC) --Comprehensive NDA application includes data showing ...
Yahoo

FDA accepts Beren Therapeutics’ NDA for Niemann-Pick disease

Add Yahoo as a preferred source to see more of our stories on Google. Adrabetadex is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation for NPC. Credit: Joyseulay/Shutterstock.com ...
Business Wire

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C. ®, the parent company of Mandos LLC ® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today ...