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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.

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

Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug ...
Monthly Prescribing Reference

Miplyffa Approved for Niemann-Pick Disease Type C

Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...
Healio

FDA approves second Niemann-Pick disease type C treatment

Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...
Nasdaq

Zevra Launches Niemann-Pick Disease Type C Drug Miplyffa In The U.S.

(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with ...
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 ...
MedPage Today

First Drug Approved for Niemann-Pick Disease Type C

The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the genetic disorder Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative ...