FDA Approves Oveporexton for NT1, Grants RMAT Designation to Sasineprocel in PD, Emerging Literature in Spinal Muscular Atrophy

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Starting with some FDA news, the administration has approved Takeda's oveporexton, an oral orexin receptor 2 (OX2R)-selective agonist, for the treatment of narcolepsy type 1 (NT1) in adults. Marketed as Orzeyful, the therapeutic is considered the first medicine approved for NT1 as a unified disorder, addressing the condition's full symptom range, and the first to work by directly targeting the loss of orexin signaling that causes the disease.¹

The approval was based on 2 randomized, double-blind, placebo-controlled 12-week studies, FirstLight (NCT06470828) and RadiantLight (NCT06505031), enrolling a combined 273 adults with NT1. Across both trials, oveporexton at the 2 mg dose met its primary endpoint, showing statistically significant improvement in the ability to stay awake during the day as measured by the Maintenance Wakefulness Test, relative to placebo (P <.001 in both studies).^1,2

Julie Flygare, president and CEO at Project Sleep, told NeurologyLive^®, “This is a historic moment for the narcolepsy community. The approval gives new hope to people living with type 1 narcolepsy, like myself, and marks the beginning of a new era of orexin innovation.”

Staying with FDA news, The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to sasineprocel (ANPD001), Aspen Neuroscience's lead investigational cell therapy for Parkinson disease (PD). The designation, which follows an earlier Fast Track designation for the therapy, provides increased FDA guidance and expedited development support, including potential eligibility for priority review and accelerated approval.³

The RMAT designation is based on results from the ongoing phase 1/2a ASPIRO trial (NCT06344026), an open-label, first-in-human study evaluating bilateral intracranial delivery of sasineprocel an autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell therapy, in patients with PD aged 50 to 70 years.^3,4

Damien McDevitt, PhD, president and chief executive officer of Aspen Neuroscience, said in a statement.¹“This significant milestone highlights the transformative nature of sasineprocel as a potentially disease-modifying therapy for patients facing a serious disease with substantial unmet medical need,"

Switching gears, spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disorder caused by biallelic variants in the survival motor neuron 1 (SMN1) gene, leading to degeneration of motor neurons in the spinal cord and progressive proximal muscle weakness. The approval of 3 SMN-enhancing therapies, including nusinersen (Spinraza; Biogen), onasemnogeneabeparvovec (Zolgensma; Novartis), and risdiplam (Evrysdi; Roche), has substantially altered the natural history of the disease, but it has not reduced the need for coordinated care across disciplines.

If anything, longer survival and improved motor function have expanded the range of clinicians involved. In recognition of SMA Awareness Month, held annually throughout August, NeurologyLive^® reviewed recent literature on multidisciplinary care in SMA, focusing on how care teams are structured, what each discipline contributes, and where the evidence base still falls short.

To read the full pieces and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. FDA Approves First Drug to Treat the Full Range of Narcolepsy Type 1 Symptoms. News release. US Food and Drug Administration. August 5, 2026. Accessed August 5, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms
2. Takeda Announces Positive Results from Two Pivotal Phase 3 Studies of Oveporexton (TAK-861) in Narcolepsy Type 1. News release. Takeda. July 14, 2025. Accessed August 5, 2026. https://www.takeda.com/newsroom/newsreleases/2025/positive-results-phase-3-oveporexton-narcolepsy-type-1/
3. Aspen Neuroscience Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Sasineprocel (ANPD001) to Treat Parkinson's Disease. News release. Aspen Neuroscience, Inc. July 30, 2026. Accessed July 31, 2026. https://www.prnewswire.com/news-releases/aspen-neuroscience-receives-fda-regenerative-medicine-advanced-therapy-rmat-designation-for-sasineprocel-anpd001-to-treat-parkinsons-disease-302838309.html
4. A Study of ANPD001 in Parkinson Disease (ASPIRO). ClinicalTrials.gov identifier: NCT06344026. Updated March 20, 2025. Accessed July 31, 2026. https://clinicaltrials.gov/study/NCT06344026