In June, the FDA took several regulatory actions, including approvals and expedited regulatory designations for a range of neurologic conditions, such as neurodegenerative, autoimmune, neuromuscular, and sleep disorders. For information on approvals in other specialties during the first quarter of 2026, see the Medscape Medical News FDA Drug Approvals page.

Approval Date Set for Immunoglobulin A (IgA) Nephropathy Therapy

The FDA accepted a biologics license application for povetacicept, an investigational dual B cell activating factor and a proliferation-inducing ligand inhibitor for adults with IgA nephropathy. A decision on approval is set for November 30, according to a statement from the drug’s manufacturer, Vertex Pharmaceuticals. The submission is supported by positive phase 3 RAINIER trial data that showed significant reductions in proteinuria compared with placebo. If approved, povetacicept could become a first-in-class disease-modifying therapy for IgA nephropathy.

Antisense Oligonucleotide for Spinal Muscular Atrophy (SMA) Moves Forward

The FDA granted breakthrough therapy designation to salanersen (Biogen), an investigational antisense oligonucleotide for SMA. The designation was based on clinical data showing improvements in motor function among patients previously treated with gene therapy. In a statement, the company said the agent is designed to increase survival motor neuron protein production with once-yearly dosing.

Orphan Drug Status for Idiopathic Hypersomnia Therapy

TRN-257, an investigational low-sodium, extended-release oral suspension of oxybate for idiopathic hypersomnia, received orphan drug status, manufacturer Tris Pharma reported. The once-nightly formulation eliminates the need for middle-of-the-night dosing that may be required with currently available oxybate therapies, which the company said could reduce treatment burden.

FDA OKs MRI Contrast Agent for Central Nervous System (CNS) Imaging

The agency cleared gadoquatrane (Ambelvist; Bayer), the lowest-dose intravenous macrocyclic gadolinium-based contrast agent for imaging of the CNS and other body regions, the company reported. The MRI agent can help detect and visualize lesions with abnormal vascularity in adult and pediatric patients, including term neonates. The approval is based on the global pivotal phase 3 QUANTI clinical studies.

Accelerated Pathway Approved for HD Gene Therapy

The FDA reversed course on an earlier decision and will allow data from a 3-year phase 1/2 trial of an investigational gene therapy for Huntington disease to serve as the primary basis for accelerated approval. The announcement followed a type B meeting between uniQure and the agency, in which regulators discussed the design of a confirmatory study of AMT-130. The company plans to submit the therapy for approval in the third quarter of 2026.

IND Cleared for Novel Remyelination Therapy in Multiple Sclerosis (MS)

The agency approved an investigational new drug application for PTD802 (Pheno Therapeutics), a selective GPR17 antagonist designed to promote remyelination in MS and other demyelinating disorders, the company announced. The move allows the first-in-human study of the oral small-molecule therapy in the US. The phase 1 study will evaluate the drug’s safety and tolerability in healthy volunteers.

Priority Review for Small Interfering RNA (siRNA) Therapy for Myasthenia Gravis (MG)

An investigational therapy for adults with anti-acetylcholine receptor antibody-positive generalized MG (gMG) has received priority review. If approved, cemdisiran would become the first siRNA therapy for gMG and the first complement-targeting therapy administered by quarterly subcutaneous injection, manufacturer Regeneron said in a statement. The decision was based on phase 3 NIMBLE trial data of subcutaneous cemdisiran every 12 weeks in adults with symptomatic gMG. A decision on approval is due in November 2026.

Exon-Skipping Therapies for Duchenne Muscular Dystrophy (DMD) Advance

The FDA accepted supplemental new drug applications for casimersen (Amondys 45) and golodirsen (Vyondys 53), manufacturer Sarepta Therapeutics announced. The move could convert existing FDA accelerated approvals to traditional approvals for patients with DMD with mutations amenable to exon 51, exon 45, or exon 53 skipping. The agency set a decision date for late February 2027.