New treatments and expanded indications offer additional options for patients with rare genetic disorders, kidney disease, severe hair loss and certain breast cancers.
September 2026 brought several significant U.S. Food and Drug Administration decisions, including first approved therapies addressing four rare diseases that previously lacked FDA-approved treatment options.
For Sanfilippo syndrome type A, the agency approved Fayuvi, a gene therapy for paediatric patients. The inherited condition progressively damages the brain and nervous system. Fayuvi delivers a working copy of the gene needed to produce an enzyme that patients lack.
The FDA also approved Zanvastro, or zilganersen, for children and adults with Alexander disease. It is the first approved treatment for the condition and targets the abnormal protein accumulation involved in its progression.
Two further decisions addressed particular manifestations of rare disorders. Emcitate, or tiratricol, was approved to treat excess circulating thyroid hormone in patients with MCT8 deficiency. Aqneursa, or levacetylleucine, gained approval for treating impaired coordination known as ataxia in patients with ataxia-telangiectasia weighing at least 15 kilograms. These indications should not be interpreted as cures for every aspect of either disease.
Kidney care also gained options. Gazyva, or obinutuzumab, was approved to reduce relapse risk in patients aged two and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in complete remission.
Kerendia, or finerenone, received an expanded indication for adults with chronic kidney disease associated with type 1 diabetes. Its approval concerns reducing urinary albumin-to-creatinine ratio—a marker of kidney damage—which is expected to reduce the risk of further kidney deterioration.
Elsewhere, Olumiant, or baricitinib, was approved for adolescents aged 12 and older with severe alopecia areata. It adds another oral treatment option; Litfulo, or ritlecitinib, had already been approved for this age group in 2023.
Diabetes technology advanced with FDA clearance of Beta Bionics’ Mint insulin patch pump, designed for three-day wear. In oncology, imlunestrant combined with abemaciclib was approved for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer following progression on at least one endocrine therapy.
Together, the decisions expand treatment choices across several specialties. Their practical value will depend on matching patients to the precise approved indications, alongside access, monitoring and individual clinical circumstances.
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