Biotechs developing therapeutics to treat rare diseases include two that received FDA approvals this year for drugs to treat spinal muscular atrophy and Alexander disease.

A recent case report details the diagnosis of Alexander disease in an obstetrics patient, contributing to the understanding of this rare condition. The report includes a review of existing literature, highlighting the complexities of diagnosing Alexander disease in atypical presentations.

Recent research demonstrates that photobiomodulation can improve conditions in Alexander disease by activating AMPK/mTOR-mediated mitophagy. This study highlights a potential therapeutic approach for a rare neurological disorder.

Ionis Pharmaceuticals secures the first targeted approval for Alexander disease, marking a significant milestone in rare disease treatment. Meanwhile, Novo Nordisk and Novartis face setbacks with late-stage trial cancellations and Phase 3 failures in cardiovascular and muscular dystrophy indications.

Between September 19th and September 30th, the FDA is due to rule on 8 rare disease therapies. Sanfilippo syndrome, Alexander disease, ataxia-telangiectasia, and MCT8 deficiency have never had an approved treatment. That could change within 3 weeks. By Jason Hunter, independent writer at Trial ... Between September 19th and September 30th, the FDA is due to rule on 8 rare disease therapies. Sanfilippo syndrome, Alexander disease, ataxia-telangiectasia, and MCT8 deficiency have never had an approved treatment. That could change within 3 weeks. By Jason Hunter, independent writer at Trial Friend After approval, companies finalize manufacturing release, insurers make coverage decisions, and gene therapies are administered only at qualified treatment centers that must be activated. Patient support programs typically open within days of approval and are the fastest first step for families. Drug companies disclose PDUFA dates in press releases and investor filings when the FDA accepts their applications. Aggregated calendars, including Trial Friend's rare disease FDA calendar, compile those disclosures and cite each company announcement as the source. 8 rare disease FDA decisions arrive by Sept 30th, 2026. Sanfilippo, Alexander disease, A-T, and MCT8 deficiency could all see their first approved treatment. Approval gave Alexander disease its first disease-modifying therapy. Emcitate for MCT8 deficiency (September 28th). Egetis Therapeutics' tiratricol treats a rare genetic disorder in which thyroid hormone cannot enter the cells that need it, causing severe developmental impairment in boys. No approved treatment exists.