Q1 2026 Clinical Recap: 7 FDA-Approved Treatments for Neurologists to Know

The first quarter of 2026 represented a notable period of progress in neurological care, highlighted by several FDA drug approvals targeting complex and rare conditions. These included first-of-their-kind therapies for ultra-rare disorders such as neurologic Hunter syndrome and cerebral folate transport deficiency, as well as new treatment formulations for spinal muscular atrophy (SMA). Collectively, these regulatory decisions reflect continued developments in the management of neurologic diseases.

Below is an overview of key FDA approvals from Q1 2026, including their clinical context, approved indications, and links to related coverage from NeurologyLive^®.

January

FDA Approves Generic Glatiramer Acetate Injection for Multiple Sclerosis Treatment

On January 5, 2026, the FDA approved ScinoPharm Taiwan’s glatiramer acetate (GA) injection as a treatment for relapsing multiple sclerosis (MS), making it the first complex injectable generic approval for this therapy.¹

GA, originally marketed as Copaxone in 1996, has been recognized as one of the most challenging complex synthetic polypeptides globally. This newly approved version is a nonbiological complex drug generic, meaning it has the same active, route, and typical strengths of traditional GA, mirroring the Copaxone label.

To date, there are no publicly registered phase 2/3 MS trials under ScinoPharm’s name and no mention of human efficacy trials in the company’s FDA-approval press communications. Instead, the company and the FDA emphasized that the product followed the nonbiological complex drug abbreviated new drug action pathway described in the FDA’s product-specific guidance for GA. That guidance allows waiving traditional clinical bioequivalence trials if the generic demonstrates tight structural and compositional matching, as well as in vitro and in vivo functional similarity.

FDA Approves Subcutaneous Copper Histidinate as First Treatment for Pediatric Menkes Disease

Months after the FDA issued a complete response letter (CRL) to the new drug application (NDA) for Sentynl Therapeutics’ copper histidinate, on January 12, 2026, the agency approved the agent as the first treatment for pediatric patients with Menkes disease, a rare genetic neurodegenerative disorder.² Marketed as Zycubo, the therapy is a subcutaneous copper replacement treatment that delivers copper in a form designed to bypass impaired intestinal absorption and support systemic utilization of the mineral.

The company resubmitted a revised NDA for copper histidinate on November 14, 2025, following receipt of a CRL from the FDA on September 30, 2025. In the CRL, the agency cited observations related to current good manufacturing practice compliance at the manufacturing site. According to Sentynl, no additional approvability concerns were identified, and the FDA did not note deficiencies in the efficacy or safety data, which showed improved overall survival among patients with Menkes disease who received early treatment.

"This milestone represents the culmination of decades of research into better understanding and ultimately finding an effective treatment for Menkes disease," Stephen Kaler, MD, a clinical genetics and genomics specialist, and professor of pediatrics at the Columbia University Medical Center, said in a statement.² "Increased awareness of Menkes disease and rapid testing upon suspicion are critical, as beginning copper histidinate therapy in affected neonates has been shown to reduce symptoms and prolong life."

Click here to view the January 2026 FDA Action Update.

February

Balt’s Squid Receives Premarket Approval for Patients with Chronic Subdural Hematomas

On February 4, 2026, the FDA granted premarket approval for Balt’s Squid liquid embolic agent for embolization of the middle meningeal artery as an adjunct to usual care in patients with large symptomatic chronic subdural hematomas. The approval was based on results from the prospective, randomized STEM trial (NCT04410146), which evaluated MMA embolization for cSDH and demonstrated a significant reduction in treatment failures in a large patient population.³

The STEM trial demonstrated that adjunctive embolization of the MMA with Squid significantly reduced treatment failure rates without increasing adverse events (AEs) in patients managed with either surgery or medical management. In the primary efficacy analysis, a primary outcome event occurred in 19 of 120 patients (16%) in the embolization group versus 47 of 129 patients (36%) in the control group, corresponding to a 64% relative reduction in risk (odds ratio [OR], 0.36; 95% CI, 0.20–0.66; P = .001). These findings underscored the clinical benefit of embolization in lowering recurrence and treatment failure rates among patients with cSDH.

“It was clear from the initial cases that Dr. Adam Arthur and I performed that middle meningeal artery embolization showed promise for these patients; however, high quality data were required to confirm the effectiveness and safety of the procedure and to ultimately generate a paradigm shift towards this minimally invasive treatment becoming the standard of care,” principal investigator David Fiorella, MD, PhD, professor at the Stony Brook University Medical Center, said in a statement.³ “I am very thankful that Balt’s leadership team aligned with this vision and took the bold step to support STEM, the very first prospective randomized trial assessing the effect of liquid embolics in patients with symptomatic cSDH.”

FDA Expands Indication for Pitolisant to Treat Cataplexy in Pediatric Narcolepsy

On February 17, 2026, the FDA granted an expanded approval indication to Harmony Biosciences’ pitolisant (Wakix) tablets to include the treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy. With this decision, pitolisant becomes the only FDA-approved nonscheduled therapy indicated for both pediatric and adult patients with narcolepsy, regardless of the presence of cataplexy.⁴

Pitolisant's supplemental NDA for the pediatric approval in cataplexy was supported by findings from a phase 3 trial (NCT02611687), which demonstrated significant reductions in EDS and cataplexy in pediatric patients aged 6 to 17 years with narcolepsy treated with 5 mg to 40 mg a day of the therapy. Published in Lancet Neurology, these findings suggested that pitolisant is a safe and effective treatment for children and adolescents with narcolepsy.⁴

“Given that narcolepsy often onsets in children and young adults, having more FDA-approved treatment options for this younger population is absolutely critical. Finding a good treatment regimen that works well for each child and teen allows them their greatest chance at getting through school and pursuing their dreams,” narcolepsy patient advocate Julie Flygare, JD, the president and CEO at Project Sleep, told NeurologyLive.

Click here to view the February 2026 FDA Action Update.

March

FDA Approves Leucovorin for Cerebral Folate Transport Deficiency

On March 10, 2026, the FDA approved expanded use of leucovorin calcium tablets (Wellcovorin) for the treatment of adult and pediatric patients with cerebral folate transport deficiency associated with confirmed variants in the folate receptor 1 (FOLR1) gene. The regulatory decision marks the first FDA-approved therapy specifically indicated for this rare genetic neurological condition.⁵

The approval was supported by a systematic review of published literature that included case reports with patient-level data as well as mechanistic evidence supporting the biological rationale for leucovorin therapy. The FDA worked with GlaxoSmithKline, the new drug application holder for Wellcovorin, to update the drug’s labeling to include information supporting safe and effective use in patients with FOLR1-related cerebral folate transport deficiency. The approval was issued through a supplemental NDA for the existing product.

“Today’s approval represents a significant milestone for patients living with cerebral folate transport deficiency due to the FOLR1 variant, a rare genetic condition that has had no FDA-approved treatment options until today,” Marty Makary, MD, MPH, commissioner of the FDA, said in a statement.⁵ “This action may benefit some individuals with FOLR1-related cerebral folate transport deficiency who have developmental delays with autistic features.”

FDA Grants Accelerated Approval to Tividenofusp Alfa for Neurologic Hunter Syndrome

On March 25, 2026, the FDA granted accelerated approval to tividenofusp alfa-eknm (Denali Therapeutics) for the treatment of neurologic manifestations of Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), in pediatric patients. Marketed as Avlayah, it becomes the first therapy specifically engineered to cross the blood-brain barrier in this rare lysosomal storage disorder.⁶

The FDA’s accelerated approval of tividenofusp alfa was supported by data from an ongoing phase 1/2, open-label study of 47 patients with Hunter syndrome, with findings that have matured over time. Previously reported data demonstrated substantial reductions in both central nervous system and peripheral disease biomarkers.

“The approval of AVLAYAH is a new era for the Hunter syndrome community as we deliver the first FDA-approved therapy designed to cross the brain’s protective barrier for individuals and families living with this debilitating disease,” Ryan Watts, PhD, co-founder and chief executive officer at Denali, said in a statement.⁶ “This approval reflects the determination and partnership of the MPS community, as well as the FDA’s collaborative engagement to incorporate biomarker evidence to help accelerate the development of urgently needed treatments.”

FDA Approves Higher Strength, More Effective Nusinersen Dose for Spinal Muscular Atrophy

On March 30, 2026, the FDA approved a new higher dose strength for nusinersen (Spinraza; Biogen) as a treatment for patients with SMA. This new dosing regimen, which is comprised of 50 mg/5 mL and 28 mg/5 mL doses, is designed to deliver a higher concentration of drug through both the loading and maintenance dosing phases, leading to more efficacious results for patients with the disease.⁷

The high-dose regimen of nusinersen, expected to become available in the coming weeks, introduces a faster loading schedule for treatment-naïve patients. This approach includes two 50 mg doses given 14 days apart, followed by ongoing maintenance injections of 28 mg every four months. For patients switching from the low-dose regimen, treatment would continue on the same four-month dosing interval after completing a single high-dose loading phase.

“I think it’s incredibly exciting,” study author Crystal Proud, MD, director of neuromuscular medicine at Children's Hospital of the King's Daughters, told NeurologyLive regarding the approval. “We’re nearing 10 years of approval of Spinraza later this year, and our patients have had incredible success over that time period. It’s natural to then say, well, what’s next? Where can we do better?”

Click here to view the March 2026 FDA Action Update.

The first quarter of 2026 saw several monumental FDA approvals in neurology, which of the following milestones do you believe will have the most profound impact on neurological care in your practice? Answer in the poll below!

Click here to view the latest of our FDA coverage.

REFERENCES
1. ScinoPharm secures U.S. FDA approval for glatiramer acetate injection for the treatment of multiple sclerosis. News release. ScinoPharm. January 5, 2025. Accessed April 6, 2026. https://www.prnewswire.com/news-releases/scinopharm-secures-us-fda-approval-for-glatiramer-acetate-injection-for-the-treatment-of-multiple-sclerosis-302652370.html
2. Sentynl Therapeutics Inc. announces FDA approval of Zycubo (copper histidinate). News release. Sentynl Therapeutics. January 13, 2026. Accessed April 6, 2026. https://sentynl.com/news/zycubo-fda-approval/
3. Balt’s Squid™ Liquid Embolic Receives FDA Approval for Adjunctive Treatment for Patients with Chronic Subdural Hematomas. Balt. News release. February 4, 2026. Accessed April 6, 2026. https://www.globenewswire.com/news-release/2026/02/04/3232016/0/en/Balt-s-Squid-Liquid-Embolic-Receives-FDA-Approval-for-Adjunctive-Treatment-for-Patients-with-Chronic-Subdural-Hematomas.html
4. Harmony Biosciences Receives U.S. Food and Drug Administration Approval for WAKIX® (pitolisant) for the Treatment of Cataplexy in Pediatric Narcolepsy. News release. Harmony Biosciences. February 17, 2026. Accessed April 6, 2026. https://ir.harmonybiosciences.com/news-releases/news-release-details/harmony-biosciences-receives-us-food-and-drug-administration-1
5. FDA approves first treatment for patients with cerebral folate transport deficiency. U.S. Food and Drug Administration. News Release. March 10, 2026. Accessed April 6, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-cerebral-folate-transport-deficiency
6. Denali Therapeutics Announces U.S. FDA Approval of AVLAYAH™ (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II). News release. Denali Therapeutics. March 25, 2026. Accessed April 6, 2026. https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-us-fda-approval-avlayahtm
7. FDA Approves New High Dose Regimen of SPINRAZA® (nusinersen) for Spinal Muscular Atrophy. News release. Biogen. March 30, 2026. Accessed April 6, 2026. https://investors.biogen.com/news-releases/news-release-details/fda-approves-new-high-dose-regimen-spinrazar-nusinersen-spinal