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 […]
Help Shape the Future: Survey on Living with Childhood Dementia Niemann-Pick UK (NPUK) is dedicated to supporting individuals and families affected by Niemann-Pick diseases, a group of rare lysosomal storage disorders that can lead to childhood dementia, among other symptoms. We are proud to promote the International Niemann-Pick Disease Registry (INPDR) survey on the “Lived […]
Rebecca French’s Research on Niemann-Pick Community Perspectives on Cure vs. Prevention in Rare Diseases Rebecca French, a fourth-year student at the University of Bath, is conducting research titled “What are the Niemann-Pick Community in England’s perspectives and policy implications of investing in gene therapy cures versus pre-emptive genetic testing for rare diseases?” This ethics-approved study […]
Azafaros Releases Preclinical Efficacy Data for Nizubaglustat in GM2 25 February 2026 Leiden, Netherlands, February 25, 2026 – Azafaros, a company aiming to become a leader in lysosomal storage disorders (LSDs), focused on addressing especially neurological symptoms, today announced the publication of proof-of-concept preclinical data with its lead product, nizubaglustat, in GM2 gangliosidosis. The data, published […]
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 Use (CHMP) of the European Medicines Agency (EMA). AQNEURSA® is approved in the European Union for use […]
What is Breakthrough Therapy Designation? This FDA designation is reserved for medicines that treat serious or life-threatening conditions and show substantial potential to improve upon existing therapies. It helps to accelerate the development and review process. For adrabetadex, the designation is supported by survival data from treated patients compared with untreated individuals. About Adrabetadex Adrabetadex […]
NPUK supports individuals and families affected by Niemann-Pick diseases, a group of rare, life-limiting, genetic conditions. For over three decades, the charity has provided practical and emotional support, advocacy and information, bringing hope and uniting a community which often feels isolated by the rarity of these devastating conditions. Receiving The King’s Award for Voluntary Services […]
Urgent Appeal for Support After Malicious Attack on September 11th On the night of September 11th, a malicious fire completely destroyed Vermont House, the long-standing base for Niemann-Pick UK (NPUK)’s Central Office. This catastrophic attack has left our charity facing immense challenges, with the entire building reduced to ruins. Caught in the crossfire of ongoing […]
AQNEURSA® (levacetylleucine) recommended for EU approval by the CHMP to treat Niemann-Pick Disease Type C AUSTIN, Texas – 29 July 2025 – IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive […]
Zevra Therapeutics Submits Marketing Authorization Application to European Medicines Agency to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C CELEBRATION, Fla., July 28, 2025 — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing […]
Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively LEIDEN, Netherlands–(BUSINESS WIRE)–Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that the first patient has been dosed in the company’s pivotal, multicenter […]
A newly approved treatment is now giving hope to patients of the rare illness known as Niemann-Pick disease…” – THV11
