Updates

Global Milestone Reached in Niemann-Pick Type C Clinical Research

Advancing Research for the Global NPC Community The International Niemann-Pick Disease Alliance (INPDA) is pleased to share an important update from Cyclo Therapeutics, marking a significant milestone in global research efforts for Niemann-Pick disease type C (NPC). Cyclo Therapeutics...

Beren Therapeutics Secures $300M Financing to Support the Potential Commercial Launch of Adrabetadex and Long-Term Care Initiatives for Infantile-Onset Niemann-Pick Disease, Type C

— $135 million equity financing with participation from leading specialist investors, corporate investors, and long-term growth partners, including Wellington Partners, JIC Venture Growth Investments (JIC VGI), Founders Fund, Narya Capital, Eisai Co., Ltd., and other select institutional investors —...

Rafael Holdings Announces Completion of Last Patient Last Visit for Pivotal Phase 3 TransportNPC™ Study Evaluating Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C

Topline results are expected to be announced in second half of calendar 2026 The company has had a pre-NDA meeting with the FDA and expects to submit an NDA in second half of calendar 2026 The company has received...

2025 Consensus Clinical Management Guidelines for Niemann–Pick Disease Type C

Published in the Journal of Inherited Metabolic Disease May 2026 INPDA is delighted to announce the publication of the 2025 Consensus Clinical Management Guidelines for Niemann–Pick Disease Type C in the Journal of Inherited Metabolic Disease. This important update...

Beren Therapeutics’ Adrabetadex NDA Accepted by FDA for Infantile-Onset Niemann-Pick Type C

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann Pick Disease Type C Read this Clinical Trial update on the Beren Therapeutics website and learn more. February 23, 2026 • Accepted for priority...

NiemannPick India: Seen, Heard, Supported – Every Step Toward Diagnosis, Care & Hope

Azafaros Announces Publication of Preclinical Efficacy Data with Nizubaglustat in GM2 Gangliosidosis

25 February 2026 Preclinical proof-of-concept data in GM2 gangliosidosis demonstrated survival benefit, functional improvement and target engagement Results are published in the 7 January issue of the Journal of Inherited Metabolic Disease Nizubaglustat is currently in Phase 3 registrational studies...

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

February 23, 2026 • Accepted for priority review with Prescription Drug User Fee Act (PDUFA) action date of August 17, 2026 • If approved, adrabetadex would be the only therapy to directly target the underlying pathophysiology of Niemann-Pick disease...

IntraBio Receives European Commission Approval of AQNEURSA® for the Treatment of Niemann-Pick Type C Disease

AUSTIN, Texas–(BUSINESS WIRE)–IntraBio Inc. today announced that the European Commission granted marketing authorization to AQNEURSA® (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human...

Patient Information Sheet: NAVIGATE Phase 3 Clinical Trial