A Significant Step Forward in NPC Research We’re pleased to share an important update from Cyclo Therapeutics, marking a significant moment in ongoing research into Niemann-Pick disease type C (NPC) and offering a real sense of progress for the global community affected by this rare condition. Cyclo Therapeutics has announced that the final patient has […]
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 […]
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 […]
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 […]