FDA Action Update, August 2026: Approvals, Clearances, and Designations

The FDA was busy in August 2026, making a number of decisions on potential new therapeutic agents, including approvals, clearances, designations, an extension, a clinical hold, and a marketing authorization.

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 Approves Orexin Agonist Oveporexton for Narcolepsy Type 1

At the beginning of the month, on August 5, 2026, the FDA 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).

“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,” Julie Flygare, JD, the president and CEO at Project Sleep, told NeurologyLive.

FDA Clears Phase 2 Trial for Myelin Protective Agent Lucid-MS in Progressive Multiple Sclerosis

Almost a week later, on August 10, 2026, the FDA cleared Quantum BioPharma's investigational new drug (IND) application for Lucid-MS (Lucid-21-302), a first-in-class compound targeting demyelination, to advance the compound into a phase 2 trial in people with progressive multiple sclerosis (MS).²

Trial start-up activities, including site selection, are underway, with patient enrollment expected to begin as quickly as possible. The planned phase 2 study is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy, safety, and tolerability of Lucid-MS using clinical and radiological measures relevant to disability progression.

"The randomized, double-blind, placebo-controlled Phase 2 trial is intended to evaluate efficacy using clinical and radiological measures relevant to disability progression and disease biology, while also assessing safety and tolerability," Andrzej Chruscinski, MD, PhD, vice president of scientific and clinical affairs at Quantum BioPharma, said in a statement.²

FDA Approves Tau PET Tracer MK-6240 for Alzheimer Diagnostic Workup

A few days later, on August 14, 2026, the FDA approved Tauklarify (florquinitau F 18 injection, previously known as MK-6240; Lantheus), a tau-targeted PET imaging agent for adults with cognitive impairment who are being evaluated for Alzheimer disease (AD). The scan identifies patients with tau neurofibrillary tangle (NFT) pathology, helping clinicians better characterize where a patient stands in the disease process.³

Tauklarify's approval was based on 2 clinical studies in which 5 independent blinded readers per study classified scans as positive or negative for tau NFT pathology, compared against a pre-established reference standard based on cognitive status and amyloid beta PET pathology. Together, the studies included scans from 617 subjects: 152 with mild AD dementia, 157 with mild cognitive impairment, and 308 cognitively unimpaired individuals.

"Tauklarify imaging will enable improved understanding of the status of Alzheimer's disease patients across the spectrum of cognitive impairment, including those in the early stages of the disease," Samantha Budd Haeberlein, PhD, chief medical officer of EB USA, said in a statement.³ “Two large, independent clinical studies demonstrated the efficacy and safety of Tauklarify in assessing the status of NFTs in patients being evaluated for Alzheimer's disease. As Alzheimer research moves towards an increased focus on early disease detection and treatment, Tauklarify is uniquely suited to address those emerging needs.”

FDA Accepts NDA, Grants Priority Review to Ecopipam for Pediatric Tourette Syndrome

Almost a week later, on August 19, 2026, the FDA accepted Teva Pharmaceuticals' new drug application (NDA) for ecopipam, a first-in-class selective dopamine D1 receptor antagonist under investigation for pediatric patients with Tourette syndrome (TS), and granted the submission priority review. The company noted that the agency set a target action date under the Prescription Drug User Fee Act (PDUFA) for late in the first quarter of 2027.⁴

The NDA application is supported by data from the phase 2b and phase 3 D1AMOND clinical program. In the 12-week, randomized, double-blind, placebo-controlled phase 2b trial, 153 pediatric participants across 68 sites in North America and Europe were randomized to ecopipam or placebo. Findings showed that those receiving ecopipam had a statistically significant and clinically meaningful reduction in the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) versus placebo at week 12 (P = .01).⁴ A subsequent open-label extension followed 121 pediatric participants for up to 12 months and reported sustained tic control.

"We are encouraged by the FDA's acceptance of the ecopipam application and grant of Priority Review. Tourette syndrome is an area where innovation has been limited for far too long, with patients and clinicians often relying on treatments developed for other conditions,” Eric Hughes, MD, PhD, executive vice president, global R&D and chief medical officer at Teva, told NeurologyLive.

FDA Grants Fast Track Designation to Safusidenib for IDH1-Mutant Glioma

One day later, on August 20, 2026, the FDA granted Fast Track Designation to safusidenib, an investigational oral, brain-penetrant selective inhibitor of mutant IDH1, for the treatment of patients with IDH1-mutant glioma, according to developer Nuvation Bio. The designation comes as the agent advances into a pivotal phase 3 program.⁵

The Fast Track designation follows updated long-term findings from the phase 2 J201 (NCT04458272) study of safusidenib in patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant glioma. As previously reported, at a median follow-up of 38.8 months, data from 27 patients in Japan showed a centrally assessed confirmed objective response rate (ORR) of 51.9%, based on Response Assessment in Neuro-Oncology (RANO) criteria for low-grade gliomas. Overall, median progression-free survival (PFS) had not been reached, while the 36-month PFS rate was 79.1%.

For patients with IDH1-mutant gliomas, the designation represents an encouraging step toward therapies designed around the underlying biology of their tumors, according to Katy Peters, MD, PhD, FAAN, professor of neurology at Duke University in North Carolina. “This FDA Fast Track Designation is just very encouraging because it truly recognizes that [safusidenib] is a drug that has potential to meet a really critical unmet need for patients with IDH1-mutant glioma,” Peters said in an interview with NeurologyLive.

FDA Clears PrecivityAD2 Alzheimer Blood Test for Symptomatic Patients 40 and Older

On the same day, on August 20, 2026, the FDA cleared C2N Diagnostics' PrecivityAD2 blood test for adults 40 years and older who present with signs of cognitive impairment, making it the first FDA-cleared AD blood test authorized for patients this young of age.⁶ The clearance extends blood-based amyloid testing below the age thresholds set by the 2 previously cleared assays and adds a third option to a diagnostic category that did not exist in FDA-cleared form before 2025.

PrecivityAD2 uses high-resolution mass spectrometry to quantify plasma amyloid-beta 42/40 ratio and percent phosphorylated tau217 (p-tau217/np-tau217), combining both into a proprietary Amyloid Probability Score 2 that stratifies results into negative, positive, and an intermediate "likely positive" category. In C2N's clearance-supporting validation cohort of 1,142 symptomatic adults, the test showed a positive predictive value of 97.6% and a negative predictive value of 93.1% against amyloid PET or CSF findings; likely-positive results, occurring in 17.3% of patients, carried a 77.3% positive predictive value.

“Today’s clearance reflects how rapidly the field is advancing. Just over a year ago, there were no FDA-cleared blood tests for Alzheimer’s and now we have three,” Isobel Coleman, chief executive officer of the Alzheimer's Drug Discovery Foundation (ADDF), said in a statement.⁶ "This is a proof point that sustained, early investment in translational science plays a catalytic role in moving promising ideas from the lab to patients.”

FDA Places RGX-121 Gene Therapy for MPS II on Clinical Hold

A few days later, on August 24, 2026, the FDA placed a clinical hold on clemidsogene lanparvovec (RGX-121), an investigational gene therapy for mucopolysaccharidosis type II (MPS II, also known as Hunter syndrome), after spinal MRI abnormalities were detected in 5 participants from the CAMPSIITE study (NCT03566043).⁷ REGENXBIO stated it its company update that it does not expect to resubmit a biologics license application for the therapy in the near term.

The imaging findings consisted of either a small nodule or small cystic mass in participants who had received RGX-121 through intracisternal or intraventricular administration approximately 3 to 6 years earlier. Investigators classified the findings as nonserious, and reviewing radiologists considered them likely benign, according to the company. The affected participants remained asymptomatic and showed overall stability or improvement on neurocognitive and neurobehavioral assessments.

"We believe these findings are unique and limited to our Hunter Syndrome program, and require longer-term follow-up and additional data analysis to assess the benefit-risk profile of RGX-121," Curran Simpson, president and chief executive officer of REGENXBIO, said in a statement.⁷ "We remain focused on our Duchenne and retinal disease candidates, which utilize a different capsid and routes of administration, with near-term catalysts that are on track, including the planned submission of the Duchenne BLA this quarter and the wet AMD topline pivotal data announcement in the fourth quarter."

FDA Extends Deramiocel Review for Duchenne Muscular Dystrophy After HOPE-3 Amendment

On the same day, on August 24, 2026, the FDA extended the Prescription Drug User Fee Act (PDUFA) target action date for Capricor Therapeutics’ deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22 to November 22, 2026.⁸ The 3-month extension follows the agency’s acceptance of additional phase 3 HOPE-3 (NCT05126758) data as a major amendment to the biologics license application (BLA).

The amendment includes 24-month open-label extension findings and additional robustness analyses from HOPE-3. Capricor submitted the information after a July 2026 FDA advisory committee meeting and asked the agency to consider a refined proposed indication centered on preservation of upper limb function, the trial’s primary end point. The FDA’s Center for Biologics Evaluation and Research accepted the amendment for review and classified it as major, prompting the revised action date.

“With an additional year of follow-up from HOPE-3, we now have one of the most extensive clinical datasets evaluating upper limb function in Duchenne,” Linda Marbán, PhD, chief executive officer of Capricor, said in a statement.⁸ “HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, and we believe the additional open-label data and further analyses included in the amendment strengthen the evidence supporting a refined proposed indication. We appreciate the FDA’s continued engagement and look forward to working constructively with the agency as it completes its review.”

FDA Clears Roche's Single-Biomarker Elecsys pTau217 Blood Test for Alzheimer Disease

On the same day, on August 24, 2026, the FDA cleared Roche’s Elecsys pTau217, a blood test that uses a single biomarker to both rule in and rule out amyloid pathology in adults 55 years and older presenting with signs, symptoms, or complaints of cognitive decline and being evaluated for AD, across both primary and specialty care settings. Roche described it as the first and only FDA-cleared, single-biomarker blood test that supports both rule-in and rule-out assessment of amyloid pathology using the same validated cutoffs across primary and specialty care.⁹

Elecsys pTau217 quantifies plasma concentrations of tau phosphorylated at threonine 217, a biomarker whose elevation is strongly associated with amyloid pathology, rather than functioning as a general marker of neuronal injury. The assay is not intended as a stand-alone diagnostic; results are meant to be interpreted alongside clinical history, cognitive assessment, and other diagnostic information as part of the broader AD workup.

"FDA clearance of Elecsys pTau217 marks an important milestone in Alzheimer's disease diagnosis and underscores Roche's continued leadership in advancing innovative solutions that can help patients get answers sooner," Dan Malarek, president and CEO of Roche Diagnostics North America, said in a statement.⁹

FDA Grants Fast Track Designation to T-Cell Agent CK0803 for ALS

A day later, on August 25, 2026, the FDA granted Fast Track designation to CK0803, an investigational allogeneic, cord blood-derived T-regulatory (Treg) cell therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS), according to developer Cellenkos. The designation applies to the company's investigational new drug (IND) application (IND 28681) for CK0803, which is designed to express neurotropic homing markers that facilitate trafficking to sites of central nervous system (CNS) inflammation.¹⁰

The designation follows early clinical findings, as previously reported by NeurologyLive, from an ongoing phase 1/1b study (NCT05695521) in which 6 evaluable patients received up to 9 infusions of CK0803.² According to the company, the trial reported changes in measures of disease progression and inflammation, including ALS Functional Rating Scale–Revised (ALSFRS-R) decline, plasma neurofilament light chain (NfL), and plasma interleukin-10 (IL-10).

“By leveraging enriched, tissue-directed regulatory T-cells derived from healthy, allogeneic, umbilical cord blood, our therapy actively homes to inflamed microglia via the CXCR3/CXCL10 axis,” said Simrit Parmar, MD, founder of Cellenkos, in a statement.¹⁰ “This targeted approach interrupts the inflammation-injury cycle and restores neuroimmune homeostasis—offering transformative potential for neuroinflammatory diseases.”

FDA Grants De Novo Authorization to Avulux Lenses for Migraine Photophobia

On the same day, on August 25, 2026, Avulux received FDA De Novo marketing authorization as an over-the-counter precision optical filter intended to decrease the impact of light sensitivity in patients aged 12 years or older with episodic migraine, according to a company announcement.¹¹ The action creates a new device classification but does not position the lenses as a replacement for acute or preventive migraine treatment.

The lenses are designed to block light around 480 nm in the high-blue range and 590 nm in the red/amber range while transmitting the remainder of the visible spectrum. They may be produced with or without a corrective prescription and are worn like conventional eyeglasses. According to Avulux, the FDA reviewed clinical evidence that included randomized controlled studies and real-world experience.

“For a long time, light sensitivity was seen as just a symptom of migraine. That thinking has changed,” Charles Posternack, cofounder and chief executive officer of Avulux, said in a statement.¹¹ “This authorization gives patients and providers a clear, FDA-recognized option designed specifically to manage the impact of light in those with migraine.”

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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 Septemeber 2, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-full-range-narcolepsy-type-1-symptoms
2. Quantum BioPharma Receives United States FDA Clearance To Proceed with Lucid-MS Phase 2 Trial in Progressive Multiple Sclerosis. News release. Quantum BioPharma Ltd. August 10, 2026. Accessed Septemeber 2, 2026. https://www.globenewswire.com/news-release/2026/08/10/3341697/0/en/quantum-biopharma-receives-united-states-fda-clearance-to-proceed-with-lucid-ms-phase-2-trial-in-progressive-multiple-sclerosis.html
3. Enigma Biomedical USA Announces FDA Approval of TAUKLARIFY™ (florquinitau F 18 injection) for Use in Patients Being Evaluated for Alzheimer's Disease. News release. Enigma Biomedical USA. August 14, 2026. Accessed Septemeber 2, 2026. https://www.businesswire.com/news/home/20260814732640/en/Enigma-Biomedical-USA-Announces-FDA-Approval-of-TAUKLARIFY-florquinitau-F-18-injection-for-Use-in-Patients-Being-Evaluated-for-Alzheimers-Disease
4. U.S. FDA Accepts Teva's New Drug Application (NDA) and Grants Priority Review for Ecopipam, a First-in-Class Investigational Therapy for Pediatric Patients with Tourette Syndrome. News release. Teva Pharmaceuticals. August 19, 2026. Accessed Septemeber 2, 2026. https://www.tevapharm.com/news-and-media/latest-news/u.s.-fda-accepts-tevas-new-drug-application-nda-and-grants-priority-review-for-ecopipam-a-first-in-cl
5. Nuvation Bio Granted FDA Fast Track Designation for Safusidenib for Treatment of IDH1-Mutant Glioma. Nuvation Bio. News Release. August 20, 2026. Accessed Septemeber 2, 2026. https://investors.nuvationbio.com/news/news-details/2026/Nuvation-Bio-Granted-FDA-Fast-Track-Designation-for-Safusidenib-for-Treatment-of-IDH1-Mutant-Glioma/default.aspx
6. FDA Clearance of C2N Diagnostics' PrecivityAD2 Blood Test Advances Precision Medicine for Alzheimer's, Building on Groundwork Laid by Early ADDF Investment. News release. Alzheimer's Drug Discovery Foundation. August 20, 2026. Accessed Septemeber 2, 2026. https://www.alzdiscovery.org/news-room/announcements/fda-clearance-of-c2n-diagnostics-precivityad2-blood-test-advances-precision-medicine-for-alzheimers-building-on-groundwork-laid-by-early-addf-investment
7. REGENXBIO Announces Regulatory Update on RGX-121 for MPS II. News release. REGENXBIO. August 24, 2026. Accessed Septemeber 2, 2026. https://regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-announces-regulatory-update-rgx-121-mps-ii
8. Capricor Therapeutics announces extension of PDUFA target action date as FDA continues review of deramiocel BLA. Capricor Therapeutics. August 24, 2026. Accessed Septemeber 2, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/354/capricor-therapeutics-announces-extension-of-pdufa-target
9. Roche receives FDA clearance for Elecsys® pTau217, advancing Alzheimer's disease assessment across primary and specialty care. News release. Roche Diagnostics. Septemeber 2, 2026. Accessed August 24, 2026. https://www.prnewswire.com/news-releases/roche-receives-fda-clearance-for-elecsys-ptau217-advancing-alzheimers-disease-assessment-across-primary-and-specialty-care-302857619.html
10. Cellenkos Receives FDA Fast Track Designation for CK0803 in Amyotrophic Lateral Sclerosis (ALS). Cellonkos Inc. News Release. August 25, 2026. Accessed Septemeber 2, 2026. https://www.prnewswire.com/news-releases/cellenkos-receives-fda-fast-track-designation-for-ck0803-in-amyotrophic-lateral-sclerosis-als-302859365.html
11. Avulux granted FDA De Novo authorization, creating the first lens classification for migraine-associated light sensitivity. News Release. Avulux. August 25, 2026. Accessed Septemeber 2, 2026 https://www.prnewswire.com/news-releases/avulux-granted-fda-de-novo-authorization-creating-the-first-lens-classification-for-migraine-associated-light-sensitivity-302857820.html