FDA Action Update, January 2026: Approvals, Acceptance, and Clearance

The FDA was busy in January 2026, making a number of decisions on potential new therapeutic agents, including granting approvals, a designation, an acceptance, a clearance, priority review, and requesting a clinical study report.

With all the treatments that have progressed through the pipeline of clinical development, the NeurologyLive^® team has been hard at work covering all the agency movements to make sure you are up to date on the latest news in neurology. To give you a chance to catch up on any of the headlines you may have missed in December, we’ve compiled all the updates into 1 place. The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds.

FDA Approves Generic Glatiramer Acetate Injection for Multiple Sclerosis Treatment

At the beginning of the month, 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 non-biological complex drug (NBCD) 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 their FDA-approval press communications. Instead, the company–and the FDA–emphasized that the product followed the non-biological complex drug ANDA pathway described in 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 Grants Orexin Agonist Alixorexton Breakthrough Therapy Designation for Narcolepsy Type 1

A day later, on January 6, 2026, the FDA granted breakthrough therapy designation to Alkermes’ alixorexton, an investigational oral, selective orexin 2 receptor (OX2R) agonist, for the treatment of narcolepsy type 1 (NT1), using positive phase 1 and phase 2 data, including the phase 2 VIBRANCE-1 trial (NCT06358950), to support the decision. The company noted that it plans to initiate a global phase 3 narcolepsy program for the agent in the first quarter of 2026.²

Findings from the VIBRANCE-1 study, a double-blind, placebo-controlled phase 2 trial, showed that treatment with alixorexton resulted in statistically significant, clinically meaningful, and dose-dependent improvements in the Maintenance of Wakefulness Test (MWT) after 6 weeks of treatment at all doses tested (4-mg, 6-mg, and 8-mg).² In addition, the data revealed statistically significant enhancements in excessive daytime sleepiness via the Epworth Sleepiness Scale (P <.0001 at all doses), a key secondary end point.

“Our team was encouraged by the FDA’s Breakthrough Therapy designation for alixorexton in patients with NT1, which recognizes the strength of our early clinical data and the potential of alixorexton to meaningfully improve care for people living with this complex condition,” Craig Hopkinson, MD, chief medical officer and executive vice president of Research & Development at Alkermes, told NeurologyLive^®. “Results from the Vibrance-1 phase 2, multiweek study in 92 patients with NT1 demonstrated clinically meaningful improvements in wakefulness and a generally well tolerated safety profile. These data support continued development of alixorexton and underscore the potential of orexin 2 receptor agonists for the treatment of NT1.”

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.³ 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.³ "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."

sBLA Acceptance Positions Efgartigimod as Potential First Therapy for Seronegative Myasthenia Gravis

A day later, on January 13, 2026, the FDA accepted Argenx’s supplemental biologics license application (sBLA) for efgartigimod (Vyvgart), positioning it as potentially the first treatment for adults with acetylcholine receptor antibody (AChR-Ab) seronegative generalized myasthenia gravis (gMG). The agency has granted a Prescription Drug User Fee Act target action date of May 10, 2026, for the decision.⁴

Efgartigimod’s sBLA was based on data from the phase 3 ADAPT SERON study (NCT06298552), a randomized, double-blind, placebo-controlled trial that featured 119 patients followed over a 5-week treatment period. In the study, patients were randomly assigned to receive 4 once-weekly intravenous (IV) infusions of efgartigimod or placebo, with change in Myasthenia Gravis Activities of Daily Living (MG-ADL) as the primary end point, recorded at 29 days.

“Patients living with seronegative gMG continue to face limited treatment options and there remains a significant need to meaningfully improve their lives. The FDA’s acceptance of our sBLA with Priority Review status reflects the potential of Vyvgart to address this need,” Luc Truyen, MD, PhD, chief medical officer at Argenx, said in a statement.⁴ “This development brings us closer to expanding the use of Vyvgart in a broad spectrum of patients with myasthenia gravis. We look forward to continuing our dialogue with the FDA as they review our application.”

FDA Requests HOPE-3 Clinical Study Report for Deramiocel BLA in DMD Cardiomyopathy

A week later, on January 20, 2026, Capricor Therapeutics announced that the FDA has formally requested the complete clinical study report and supporting data from the phase 3 HOPE-3 trial (NCT05126758) of deramiocel, as the agency continues to review of the company’s biologics license application (BLA). The investigational cell therapy, which remains under review, is aiming to become the first cell-based product for patients with Duchenne muscular dystrophy (DMD) cardiomyopathy.⁵

Capricor stated in its company update that preparation of the HOPE-3 clinical study report is already underway and that the requested materials are expected to be submitted to the FDA in February 2026. The company noted that it anticipates that this submission will support continued review of the BLA and may lead to the assignment of a new PDUFA action date.

“We are actively engaging with the FDA in order to facilitate an efficient review of the HOPE-3 data that directly address the issues raised in the CRL we received in July 2025. We were pleased that the FDA requested the HOPE-3 clinical study report, as this is an expected and appropriate next step following their initial review of the topline data,” Linda Marbán, PhD, chief executive officer at Capricor, said in a statement.⁵ “Our near-term priority is to address the FDA’s request and continue working collaboratively so that patients with late-stage DMD, who currently have very limited treatment options, may gain access to Deramiocel as soon as possible.”

FDA Clears Pilavapadin for Phase 3 Development in Diabetic Peripheral Neuropathic Pain

A day later, on January 21, 2026, Lexicon Pharmaceuticals announced that it completed an End-of-Phase 2 meeting with the FDA to advance pilavapadin, an investigational agent, as a potential treatment for diabetic peripheral neuropathic pain (DPNP). The agency had no concerns over the phase 3 program, which will include two 12-week, placebo-controlled studies evaluating a 10-mg daily dose versus placebo, using change in average daily pain score (ADPS) over a 12-week period as the primary end point.⁶

Previously reported topline data from the phase 2b PROGRESS study (NCT06203002), a large-scale study of nearly 500 participants, demonstrated that treatment with pilavapadin led to reductions in pain compared with placebo in adults with moderate to severe DPNP. Along with findings from the earlier RELIEF-DPN-1 study (NCT04455633), these results supported selecting a 10-mg once-daily dose for advancement into phase 3 trials.

“People with DPNP are desperately in need of new treatment options. This progress puts Lexicon one step further toward making pilavapadin the first non-opioid DPNP medicine available to patients in over two decades. That’s why we are very pleased that the End-of-Phase 2 meeting with FDA was productive and provided us with the insights needed to design a robust Phase 3 program,” Craig Granowitz, MD, PhD, senior vice president and chief medical officer at Lexicon, told NeurologyLive^®.

Priority Review Granted for Weekly Subcutaneous Lecanemab Dosing in Early Alzheimer Disease

At the end of the month, on January 25, 2026, Eisai and Biogen announced that the FDA has accepted its supplemental biologics license application (sBLA) for the subcutaneous (SC) autoinjector formulation of lecanemab-irmb (Leqembi Iqlik) as a weekly starting dose for patients with early Alzheimer disease (AD), granting the application priority review with a PDUFA action date of May 24, 2026.⁷ If this new 500-mg dosing regimen is approved, it would offer an alternative to the currently available biweekly intravenous 250-mg dosing used at treatment initiation.

For background, in August 2025, the FDA approved a new 360-mg, once-weekly SC maintenance dosing option for lecanemab that follows 18 months of biweekly intravenous therapy. Administration of lecanemab-irmb using the autoinjector requires approximately 15 seconds per 250-mg injection. Compared with intravenous administration, the SC formulation may reduce health care resource utilization associated with infusion-based delivery, including infusion preparation and nurse monitoring.

“If the FDA approves a subcutaneous starting dose for Leqembi, it will mark a major step toward expanding access for patients and caregivers,” Laura Nisenbaum, PhD, executive director of drug development at the Alzheimer’s Drug Discovery Foundation, told NeurologyLive^®. “Start-to-finish subcutaneous delivery opens the door to at-home administration—similar to diabetes and GLP-1 therapies—representing a breakthrough that will ultimately make it easier to combine multiple treatments and target the full spectrum of Alzheimer’s pathobiology.”

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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 February 11, 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. Alixorexton Granted Breakthrough Therapy Designation by U.S. FDA for the Treatment of Narcolepsy Type 1. News release. January 6, 2026. Accessed February 11, 2026. https://investor.alkermes.com/news-releases/news-release-details/alixorexton-granted-breakthrough-therapy-designation-us-fda
3. Sentynl Therapeutics Inc. announces FDA approval of Zycubo (copper histidinate). News release. Sentynl Therapeutics. January 13, 2026. Accessed February 11, 2026. https://sentynl.com/news/zycubo-fda-approval/
4. Argenx announces FDA acceptance of supplemental biologics license application with priority review for Vyvgart in AChR-Ab seronegative gMG. News release. Argenx. January 13, 2026. Accessed February 11, 2026. https://www.globenewswire.com/news-release/2026/01/13/3217457/0/en/argenx-Announces-FDA-Acceptance-of-Supplemental-Biologics-License-Application-with-Priority-Review-for-VYVGART-in-AChR-Ab-Seronegative-gMG.html
5. Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Topline Data. News release. Capricor Therapeutics. January 20, 2026. Accessed February 11, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/335/capricor-therapeutics-provides-regulatory-update-on
6. Lexicon Pharmaceuticals Announces Successful End-of-Phase 2 Meeting with FDA For Pilavapadin in the Treatment of Diabetic Peripheral Neuropathic Pain. News release. Lexicon Pharmaceuticals. January 21, 2026. Accessed February 11, 2026. https://investors.lexpharma.com/news-releases/news-release-details/lexicon-pharmaceuticals-announces-successful-end-phase-2-meeting
7. FDA Accepts LEQEMBI® IQLIK^TM (lecanemab-irmb) Supplemental Biologics License Application as a Subcutaneous Starting Dose for the Treatment of Early Alzheimer's Disease under Priority Review. News release. Eisai and Biogen. January 25, 2026. Accessed February 11, 2026. https://www.eisai.com/news/2026/pdf/enews202605pdf.pdf