Updates

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

INPDA Global Niemann Pick Disease Initiatives

INPDA Global Niemann Pick Disease Initiatives INPDA is actively engaged in a number of initiatives and campaigns which  have a wide-spread impact on a global scale . These include: Facilitating an update of the Consensus Clinical Management Guidelines for...

Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively

First patient dosed in global pivotal clinical trial program for late infantile/juvenile onset NPC and GM1/GM2 gangliosidoses. Both studies are part of Azafaros’ mission to develop treatments for the unmet need of patients with rare lysosomal storage disorders The...

AQNEURSA® (levacetylleucine) recommended for EU approval by the CHMP to treat Niemann-Pick Disease Type C

Zevra Submits Arimoclomol MAA to EMA for Niemann-Pick Disease Type C Treatment