FDA Action Update, August 2025: Approvals and Complete Response Letter

The FDA was busy in August 2025, making a number of decisions on potential new therapeutic agents, including granting approvals and issuing a complete response letter.

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 over the last month, 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 Fremanezumab as First Anti-CGRP Preventive Therapy for Pediatric Episodic Migraine

On August 6, 2025, the FDA approved an expanded indication for Teva’s fremanezumab (Ajovy) to include the preventive treatment of pediatric patients with episodic migraine aged 6-17 who weigh 45 kilograms or more. With the approval, fremanezumab becomes the first anti-calcitonin gene-related peptide (CGRP)-targeting treatment approved for both pediatric episodic migraine prevention and migraine prevention in adults.¹

The expanded indication into pediatrics was based on data from the phase 3 SPACE study (NCT03539393), a double-blind, placebo-controlled, parallel-group study of pediatric patients with history of less than 14 headache days a month. SPACE, which comprised 237 pediatric patients aged 6-17, showed that fremanezumab treatment resulted in statistically significant superior efficacy over a 12-week period compared with placebo, while maintaining a safety profile that was consistent with prior observations.

"Migraines are a common yet invisible condition that can severely disrupt daily life for children and adolescents, often leaving them overlooked and misunderstood,” Chris Fox, executive vice president, U.S. Commercial and Innovative Franchise Lead, and head of Global Marketing Business at Teva, said in a statement.¹ "With this FDA approval, AJOVY now offers younger patients a new treatment option, addressing a long-standing gap in care and offering families added support as they navigate the challenges of this condition."

FDA Grants Approval of Hypoglossal Nerve Stimulation System for Obstructive Sleep Apnea Treatment

On August 8, the FDA approved Nyxoah’s Genio hypoglossal nerve stimulation system for patients with moderate to severe obstructive sleep apnea (OSA) who have an apnea-hypopnea index (AHI) of greater than or equal to 15 and less than or equal to 65.²

The approval was supported by positive data from the multicenter, prospective, open-label, interventional DREAM trial (NCT03868618) which assessed the efficacy and safety of the Genio system among 115 patients with OSA for 12 months. Overall, the study met both its primary and secondary end points, showing an AHI responder rate of 63.5% (n = 73, P = .002) and an Oxygen Desaturation Index (ODI) responder rate of 71.3% (n = 82, P <.001) at 12 months. Notably, findings from the trial revealed that 82.0% of all participants from DREAM had their AHI scores drop below 15 or lower.^3,4

"The Genio system's approval represents a major addition to the treatment options available to physicians treating patients with OSA,” Colin Huntley, MD, associate professor in the Department of Otolaryngology Head & Neck Surgery at Thomas Jefferson University, said in a statement.² “This unique bilateral stimulation technology has demonstrated consistent efficacy across all sleeping positions, including the challenging supine position, while maintaining an excellent safety profile.”

FDA Denies Approval for Friedreich Ataxia Agent Vatiquinone, Citing More Efficacy Needed

On August 19, the FDA handed PTC Therapeutics a complete response letter (CRL) for its investigational therapy vatiquinone, a first-in-class selective inhibitor of 15-lipoxygenase (15-LO), as a new treatment for both pediatric and adult patients with Friedriech ataxia (FA), a rare neuromuscular condition. In the CRL, the agency noted that the evidence of efficacy was not substantial enough for vatiquinone, and that another well-controlled study would be needed to support new drug application (NDA) resubmission.⁵

Vatiquinone was aiming to become the second marketed treatment for FA, second to Biogen’s omaveloxolone, an FcRn inhibitor, which gained green light in early 2023 for patients 16 years and older with FA. The 2 therapies have slightly different functions: Vatiquinone primarily works by reducing oxidative damage and ferroptosis, aiming to preserve mitochondrial integrity and neuronal survival in FA, whereas omaveloxolone enhances the body’s endogenous antioxidant and anti-inflammatory defenses, improving mitochondrial energy production and reducing oxidative stress.

"We are of course disappointed by the FDA's decision to not approve vatiquinone," Matthew B. Klein, MD, chief executive officer at PTC, said in a statement.⁵ "We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich's ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL."

FDA Approves Lecanemab Autoinjector, Marking First At-Home Treatment for Alzheimer Disease

At the end of the month, on August 29, FDA has approved a new autoinjector formulation for lecanemab (Leqembi; Eisai), an antiamyloid therapy indicated for patients with early-stage Alzheimer disease (AD) and mild cognitive impairment (MCI) from AD. With the decision, the subcutaneous autoinjector (SC-AI) becomes the first-ever at-home, self-administered option for ongoing AD treatment, improving convenience and access for patients in the United States.⁶

The new SC-AI, approved based on data from the pivotal phase 3 Clarity AD trial (NCT03887455), provides quicker administration for patients, lasting about 15 seconds total. In clinical trial settings, it demonstrated comparable exposure, improved tolerability, enhanced amyloid clearance, and better user-friendliness in patient, caregiver, and healthcare surveys. In addition, there is a belief that this new SC-AI will have significant societal cost savings, lowering treatment and administration expenses.

"This FDA action demonstrates continued progress in the field of Alzheimer's treatment," said Maria C. Carrillo, PhD, chief science officer and medical affairs lead at Alzheimer's Association, said in a statement.⁷ "We are seeing the evolution of amyloid targeting antibody treatments, including improvements in drug delivery and acknowledgment that this class of treatments continues to demonstrate clinical benefit beyond the 18-month clinical trial data."

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REFERENCES
1. FDA Approves Expanded Indication for AJOVY® (fremanezumab-vfrm), The First Anti-CGRP Preventive Treatment for Pediatric Episodic Migraine. News release. Teva Pharmaceuticals. August 6, 2025. Accessed September 8, 2025. https://www.tevapharm.com/news-and-media/latest-news/fda-approves-expanded-indication-for-ajovy-fremanezumab-vfrm-the-first-anti-cgrp-preventive-treatment/
2. Nyxoah Receives Approval from FDA for Genio® System for the Treatment of Obstructive Sleep Apnea. News Release. Nyxoah. Published August 8, 2025. Accessed September 8, 2025. https://www.globenewswire.com/news-release/2025/08/08/3130374/0/en/Nyxoah-Receives-Approval-from-FDA-for-Genio-System-for-the-Treatment-of-Obstructive-Sleep-Apnea.html
3. Nyxoah Announces DREAM U.S. Pivotal Study Meets Primary Endpoints. News Release. Nyxoah. Published March 19, 2024. Accessed September 8, 2025. https://www.globenewswire.com/news-release/2024/03/19/2848963/0/en/Nyxoah-Announces-DREAM-U-S-Pivotal-Study-Meets-Primary-Endpoints.html
4. Woodson BT, Suurna MV, Gillespie MB, et al. Multicentre study conducted across centres in the USA, Europe and Australia to assess the safety and effectiveness of a bilateral hypoglossal nerve stimulation system for the treatment of obstructive sleep apnoea in adults: a protocol for a pivotal, multicentre, open-label, single-arm study. BMJ Open. 2024;14(12):e085218. Published 2024 Dec 20. doi:10.1136/bmjopen-2024-085218
5. PTC Therapeutics receives complete response letter for vatiquinone NDA. News release. PTC Therapeutics. August 19, 2025. Accessed September 8, 2025. https://www.prnewswire.com/news-releases/ptc-therapeutics-receives-complete-response-letter-for-vatiquinone-nda-302533332.html
6. FDA Approves LEQEMBI® IQLIK™ (lecanemab-irmb) Subcutaneous Injection for Maintenance Dosing for the Treatment of Early Alzheimer's Disease. News release. August 29, 2025. Accessed September 8, 2025. https://www.prnewswire.com/news-releases/fda-approves-leqembi-iqlik-lecanemab-irmb-subcutaneous-injection-for-maintenance-dosing-for-the-treatment-of-early-alzheimers-disease-302542371.html
7. Alzheimer’s Association Welcomes FDA Action Approving Leqembi Weekly Subcutaneous Maintenance Dosing. News Release. Alzheimer’s Association. Published August 29, 2025. Accessed September 8, 2025. https://www.alz.org/news/2025/fda-action-approving-leqembi-subcutaneous-maintenance-dosing