FDA News

Six FDA PDUFA decisions this fall could reshape care in Alzheimer disease, DMD, Sanfilippo syndrome, Alexander disease, and SMA, here's what clinicians need to know about the data behind each.

The FDA has approved oveporexton, marketed as Orzeyful, as the first therapy to directly restore orexin signaling and address the full symptom range of narcolepsy type 1.

The FDA has accepted Saol Therapeutics' resubmitted NDA for SL1009 in pyruvate dehydrogenase complex deficiency, setting a new PDUFA target action date of December 30, 2026.

Catch up on any of the neurology headlines you may have missed in July 2026, compiled into 1 place by the NeurologyLive® team.

The nonbinding vote casts doubt on the therapy's path to approval ahead of an August 22 FDA decision, capping over a year of regulatory back-and-forth over the strength of its phase 3 data.

The submission is backed by phase 3 FoCus trial data showing ALXN1840 tripled copper mobilization and produced significant, sustained neurologic improvement compared with standard of care over 48 weeks.

The FDA granted priority review to zeleciment rostudirsen for exon 51-amenable Duchenne muscular dystrophy, with a PDUFA target action date of January 21, 2027.

If approved, AD109 could become the first medication to address both the nighttime airway obstruction and oxygen deprivation central to OSA, as well as its daytime symptoms, such as fatigue.

The FDA files the NDA for Axsome’s AXS-12 for cataplexy in narcolepsy, backed by phase 3 data wins showing fewer attacks and better daytime alertness.

Catch up on any of the neurology headlines you may have missed in June 2026, compiled into 1 place by the NeurologyLive® team.

The 510(k) clearance was supported by a 3-study, 159-patient registrational program showing a 45.5% increase in response rate vs physical therapy alone, and includes Medicare coverage at launch.

The FDA accepted supplemental applications seek to convert the accelerated approvals of casimersen and golodirsen to traditional approvals in patients with Duchenne muscular dystrophy.

Additional analyses from a phase 2 study evaluating the effects of zervimesine on hallucination in DLB will be presented at the Alzheimer's Association International Conference 2026 .

The FDA will review Capricor Therapeutics' Deramiocel for the treatment of Duchenne muscular dystrophy on July 29, 2026, ahead of the therapy's PDUFA target action date of August 22, 2026.

Following the FDA's expanded approval of efgartigimod, the neuromuscular physician at Erlanger Health Systems discusses the implications for seronegative disease, treatment sequencing, and the evolving definition of antibody-negative MG. [WATCH TIME: 5 minutes]

REGENXBIO expects to resubmit the BLA for clemidsogene lanparvovec in Q3 2026 after the FDA confirmed existing CAMPSIITE data are sufficient for accelerated approval consideration.

The European Commission has approved Cenrifki (tolebrutinib) for nrSPMS without relapses in the last two years, a milestone that arrives despite the drug's failed US submission and a regulatory pathway that has been anything but linear.

FDA’s clearance for PTD802, a first-in-class GPR17 antagonist designed to promote remyelination, enables initiation of a first-in-human phase 1 study in healthy volunteers and advances a novel regenerative approach for multiple sclerosis.

Bevacizumab becomes approved in Japan for neurofibromatosis type 2 following phase 2 data that suggested potential effects on hearing preservation and tumor volume despite the study not meeting its primary efficacy end point.

The FDA has accepted Regeneron Pharmaceuticals’ new drug application of cemdisiran's for generalized myasthenia gravis under priority review, with a target action date of November 2026.

Phase 3 data showed a 53% reduction in relapse risk versus placebo, and ecopipam could become the first non-antipsychotic option ever approved for Tourette syndrome.

The FDA indicated that 3-year phase 1/2 data may support a BLA submission for AMT-130, an investigational gene therapy that previously demonstrated significant slowing of Huntington disease progression in treated patients.

Catch up on any of the neurology headlines you may have missed in May 2026, compiled into 1 place by the NeurologyLive® team.

New exon‑51 skipping therapy boosts dystrophin and patient strength, with upcoming trial readouts aiming to support accelerated FDA approval.

The FDA's acceptance and priority review of levacetylleucine for ataxia-telangiectasia positions the therapy to potentially become the first approved treatment for the rare neurodegenerative disorder.

FDA priority review of asundexian marks a key regulatory milestone for factor XIa inhibition in secondary stroke prevention following positive phase 3 OCEANIC-STROKE results.

Catch up on any of the neurology headlines you may have missed from last week, compiled into 1 place by the NeurologyLive® team.

The FDA has expanded the use of efgartigimod alfa-fcab and Hytrulo to include adults with AChR-Ab seronegative generalized myasthenia gravis, marking the first approved FcRn-targeted therapy for this patient population.