FDA Action Update, June 2026: Acceptances, Clearances, and Alignments

The FDA was busy in June 2026, making a number of decisions on potential new therapeutic agents, including acceptances of new drug applications, reaching alignments, and scheduling an AdComm meeting.

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, we’ve compiled all the updates here. The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds.

FDA Allows AMT-130 Huntington Disease Data to Support Planned BLA Submission Under Accelerated Approval Pathway

In the middle of the month, on June 17, 2026, the FDA indicated that 3-year data from the phase 1/2 clinical program of AMT-130, an investigational gene therapy for Huntington disease (HD), may serve as the primary basis for a planned Biologics License Application (BLA) submission seeking accelerated approval. According to developer uniQure, the company expects to submit the application in the third quarter of 2026.¹

The regulatory update follows a recent Type B meeting between uniQure and the FDA, during which the agency requested additional alignment on the design of a confirmatory study that would follow a potential accelerated approval. According to the company, the FDA indicated that the confirmatory study may evaluate AMT-130 against standard-of-care therapy rather than requiring a sham procedure.

"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA's genuine commitment to addressing the unmet need of Americans living with Huntington's disease," Matt Kapusta, chief executive officer at uniQure, said in a statement.¹ "The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study."¹

FDA and EMA Accepts Regulatory Applications for Cemdisiran in Generalized Myasthenia Gravis

A few days later, on June 22, 2026, both the FDA and European Medicines Agency (EMA) have accepted regulatory applications for Regeneron Pharmaceuticals’ cemdisiran, an investigational small interfering RNA (siRNA) for the treatment of adult patients with anti-acetylcholine receptor (AChR) antibody–positive generalized myasthenia gravis (gMG). If approved, cemdisiran would represent the first siRNA agent indicated for gMG and the first complement-targeting treatment available via subcutaneous quarterly dosing in this population.²

The FDA accepted the company’s new drug application for cemdisiran under priority review following the use of a priority review voucher, with a PDUFA target action date of November 2026. In addition, the EMA has accepted the Marketing Authorization Application; a decision from the European Commission is anticipated in the second half of 2027. The company also noted that a regulatory submission in Japan is planned for early 2027.

“Generalized myasthenia gravis is a chronic debilitating disease with unpredictable symptoms that impact daily life. While current therapies have managed disease activity, there remains an unmet need for options that achieve rapid and sustained efficacy with reduced treatment burden,” Tuan Vu, MD, professor of neurology at the University of South Florida Morsani College of Medicine, said in a statement.²

FDA Clears Investigational New Drug Application for Remyelinating Agent PTD802 in Multiple Sclerosis

On the same day, on June 22, 2026, the FDA cleared an investigational new drug (IND) application for PTD802, an oral small-molecule therapeutic candidate designed to promote remyelination in neurologic diseases, including multiple sclerosis (MS). The decision allows developer Pheno Therapeutics to initiate a first-in-human clinical study in the United States evaluating the safety, tolerability, and pharmacokinetics of the agent in healthy volunteers.³

The regulatory milestone marks the entry of a novel therapeutic approach into clinical development at a time when most approved MS therapies primarily target inflammatory disease activity rather than repair of existing central nervous system (CNS) damage. Although disease-modifying therapies (DMTs) have substantially reduced relapse rates and MRI activity in relapsing forms of MS, disability progression remains an important unmet need, particularly in progressive disease, where neurodegeneration and incomplete remyelination contribute to long-term functional decline.

“FDA IND clearance is an important milestone for our PTD802 program, and a step further toward our ultimate goal of providing an effective treatment for neurological diseases associated with demyelination,” Fraser Murray, PhD, CEO of Pheno Therapeutics, said in a statement.³ “As the first company to gain approval to begin clinical trials for a selective GPR17 antagonist, we are proud to be leading the way, and believe this approach has the potential to offer real patient benefit, in MS and beyond.”

FDA Clears Path for NAVSUNLI BLA Resubmission in MPS II, No Additional Studies Required

On the same day, on June 22, 2026, REGENXBIO announced it has reached alignment with the FDA on a path forward for the potential accelerated approval of NAVSUNLI (clemidsogene lanparvovec-sngl; RGX-121), a one-time investigational gene therapy for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome.⁴ The announcement marks a significant reversal from the complete response letter (CRL) the company received in February 2026, in which the agency had cited multiple evidentiary concerns and suggested additional studies before approval could be considered.

In a recent collaborative discussion conducted as part of REGENXBIO's appeal of the February CRL, the FDA confirmed that existing NAVSUNLI clinical data are sufficient for the accelerated approval pathway and that no additional patient enrollment or new studies are required, including the untreated concurrent control arm previously recommended.¹ The FDA asked REGENXBIO to request a Type A meeting to review existing longer-term biomarker and clinical data, with a BLA resubmission to follow on an expedited review basis. REGENXBIO expects the Type A meeting in July 2026 and the BLA resubmission in Q3 2026.

"We are encouraged by recent signals from the new FDA leadership reinforcing a commitment to address the unique nature of rare diseases and use the accelerated approval pathway to bring transformative therapies to patients with serious, unmet medical needs," Curran Simpson, President and CEO of REGENXBIO, said in a statement.⁴

FDA Aligns With Cognition Therapeutics on Phase 3 Trial Design for Zervimesine in DLB-Associated Psychosis

A few days later, on June 24, 2026, the FDA indicated that psychosis associated with dementia with Lewy bodies (DLB) could serve as an approvable outcome for a future new drug application (NDA), following a formal meeting with Cognition Therapeutics. The agency reached alignment with the company on key design elements of a registrational phase 3 trial of zervimesine, an investigational oral sigma-2 receptor modulator, with the pivotal program expected to launch in mid-2027.⁵

As aligned with the FDA, the proposed phase 3 study will enroll adults with DLB who experience psychosis, defined as hallucinations and delusions. Patients currently receiving stable background treatment with off-label antipsychotic medications will be eligible to participate, a design that reflects real-world clinical practice. Following a screening period, participants will be randomized to receive either zervimesine 100 mg once daily or placebo for 9 months.

“We reached an important agreement with the FDA that DLB psychosis could be an approvable outcome and that key aspects of our registrational trial design are appropriate and supportive of an NDA,” Anthony Caggiano, MD, PhD, chief medical officer at Cognition, said in a statement.⁵ “To date, few drugs have been researched for DLB and none have been approved. The only recourse for DLB patients experiencing psychosis is the off-label use of potentially dangerous antipsychotics.”

FDA Advisory Committee Schedules Meeting to Review Deramiocel's BLA in Duchenne Muscular Dystrophy

Months after the FDA rescinded its complete response letter (CRL), on June 26, 2026, the agency's Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to review Capricor Therapeutics' biologics license application (BLA) for deramiocel, an investigational allogeneic cardiac-derived cell therapy for Duchenne muscular dystrophy (DMD), on July 29, 2026.⁶

In the company’s issued CRL in July 2025, the agency stated that it was unable to review the BLA because it did not meet the requirements for substantial evidence of efficacy. In addition, the FDA noted outstanding items in the Chemistry, Manufacturing, and Controls section of the application, most of which the company stated it believed had been addressed in prior communications with the FDA. For context, the original BLA submission for deramiocel was based on data from the HOPE-2 trial (NCT03406780), its open-label extension (NCT04428476), and natural history comparisons from FDA-funded datasets.

“We are encouraged by the opportunity to bring Deramiocel before the Advisory Committee and engage directly with the FDA, the DMD patient community, and the physicians who care for them. We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy,” Linda Marbán, PhD, chief executive officer at Capricor, said in a statement.⁶

FDA Accepts Sarepta's sNDAs for Casimersen and Golodirsen for Duchenne Muscular Dystrophy

At the end of the month, on June 30, 2026, the FDA accepted Sarepta Therapeutics’ supplemental new drug applications (sNDAs) for casimersen (Amondys 45) and golodirsen (Vyondys 53), 2 exon-skipping therapies indicated for Duchenne muscular dystrophy (DMD), and has assigned a PDUFA target action date of February 28, 2027. The submissions seek conversion of the existing accelerated approvals for both agents to traditional approvals, which will require the FDA to weigh confirmatory phase 3 data that narrowly missed its primary end point alongside an extensive body of real-world evidence.⁷

The sNDAs are anchored to data from ESSENCE (NCT02500381), a global, randomized, double-blind, placebo-controlled phase 3 confirmatory study enrolling 228 ambulatory boys with DMD amenable to exon 45 or exon 53 skipping, aged 6 to 13 years. Participants were randomized 2:1 to combined phosphorodiamidate morpholino oligomer (PMO) treatment (casimersen 30 mg/kg or golodirsen 30 mg/kg intravenously once weekly) or placebo over a 96-week double-blind period, followed by a 48-week open-label extension.

“The FDA’s acceptance of these applications for review is an important step for the Duchenne community. The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease,” Louise Rodino‑Klapac, PhD, president of research & development and technical operations at Sarepta, said in a statement.⁷ “We look forward to working with the FDA throughout the review.”

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REFERENCES
1. uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease. uniQure. News Release. June 17, 2026. Accessed June 30, 2026. https://www.uniqure.com/investors-media/press-releases
2. Cemdisiran Regulatory Submissions Accepted for Review by FDA and EMA for the Treatment of Generalized Myasthenia Gravis (gMG). News release. Regeneron Pharmaceuticals. June 22, 2026. Accessed June 30, 2026. https://newsroom.regeneron.com/news-releases/news-release-details/cemdisiran-regulatory-submissions-accepted-review-fda-and-ema
3. Pheno Therapeutics Granted FDA IND Clearance for Lead Multiple Sclerosis Therapeutic Candidate PTD802. Pheno Therapeutics. June 22, 2026. Accessed June 30, 2026. https://www.businesswire.com/news/home/20260622772175/en/Pheno-Therapeutics-Granted-FDA-IND-Clearance-for-Lead-Multiple-Sclerosis-Therapeutic-Candidate-PTD802
4. REGENXBIO announces alignment with FDA on path forward for NAVSUNLI BLA resubmission for accelerated approval; first potential gene therapy for MPS II. News release. REGENXBIO Inc. June 22, 2026. Accessed June 30, 2026. https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-alignment-fda-path-forward-navsunli-bla
5. Cognition Therapeutics Aligns on Key Aspects of Pivotal Study for Zervimesine (CT1812) in DLB Psychosis Following Receipt of FDA Meeting Minutes. News release. Cognition Therapeutics. June 24, 2026. Accessed June 30, 2026. https://ir.cogrx.com/press_releases/cognition-therapeutics-aligns-on-key-aspects-of-pivotal-study-for-zervimesine-ct1812-in-dlb-psychosis-following-receipt-of-fda-meeting-minutes/
6. Capricor Therapeutics announces FDA advisory committee meeting to review BLA for deramiocel for the treatment of Duchenne muscular dystrophy. Capricor Therapeutics. June 26, 2026. Accessed June 30, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/348/capricor-therapeutics-announces-fda-advisory-committee
7. Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®. News release. Sarepta Therapeutics. June 30, 2026. Accessed June 30, 2026. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fda-acceptance-sndas-amondys-45r-and-vyondys