FDA Action Update, May 2025: Approval, Clearance, and Authorization

The FDA was busy in May 2025, making a number of decisions on potential new therapeutic agents including granting an approval, a clearance, and an 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 over the course of the last month, we’ve compiled all the updates into one place. The coverage includes the latest FDA approvals, new designations, submissions and resubmissions, and clinical trial initiations and holds.

Click the read more buttons for more details and information about each update.

FDA Authorizes Expanded Access Program for ALS Treatment SPG302

Early in the month, on May 5, the FDA authorized Spinogenix’s expanded access program (EAP) for its investigational amyotrophic lateral sclerosis (ALS) agent SPG302 in the United States for patients living with the disease that meet the program’s eligibility criteria. The company also noted that it recently completed a phase 1/2 trial (NCT05882695) in Australia, and patients were offered continuation of the treatment in the open label extension trial (NCT05882695).¹

The phase 1/2 clinical trial, cleared by the FDA in June 2024, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of SPG302 in healthy volunteers and patients living with ALS.² Regarding healthy volunteers, previously announced results showed that treatment with the agent was tolerable and led to changes in plasma levels that aligned with its efficacy in animal models. In the study, investigators randomly assigned healthy volunteers aged between 18 and 55 years to either SPG302 or a placebo. The first part of the trial featured only single ascending doses of SPG302 and the second tested multiple ascending doses of SPG302 for 5 days.

“Every 90 minutes someone is diagnosed with ALS, the leading adult-onset motor neuron disease. Tragically, most patients survive only 2 to 5 years post diagnosis and current therapies offer only modest extensions of survival and are often poorly tolerated. Spinogenix is working toward improving the standard of care by addressing, for the first time, synapse loss as a fundamental contributor to disease onset and progression,” Stella Sarraf, PhD, founder and chief executive officer at Spinogenix, told NeurologyLive^®.

FDA Clears IND to Study Gene Therapy CAP-002 in STXBP1 Developmental Epileptic Encephalopathy

About a week later, on May 12, the FDA cleared Capsida’s investigational new drug (IND) application to test CAP-002, an intravenously administered gene therapy, as a potential treatment for patients with syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE), a rare epileptic disorder impacting 1 in every 26,000 births globally.²

The agent, a wholly owned, first-in-class gene therapy, will be studied in a phase 1/2 trial dubbed SYNRGY, that will begin dosing in the third quarter of this year. CAP-002, manufactured through Capsida’s Good Manufacturing Practice (GMP) facility, is the first such treatment to enter human trials using an IV-administered, blood brain barrier-crossing engineered capsid that also is detargeted from off-target issues, such as liver and dorsal root ganglia.

"STXBP1-related disorders present devastating challenges in communication, development, motor function and seizures," Charlene Son Rigby, president and cofounder of STXBP1 foundation, said in a statement.² "We are in dire need of targeted therapies that can improve the lives and functioning of our children and families."

FDA Approves First Autoinjector Form of DHE for Acute Migraine and Cluster Headache

A few days later, on May 15, the FDA granted approval to Amneal Pharmaceuticals’ dihydroergotamine mesylate injection, marketed as Brekiya, as the first dihydroergotamine (DHE) autoinjector for the acute treatment of migraine with or without aura and cluster headache in adults.³

The company noted that the therapy contains the same DHE used in hospitals but in a ready-to-use, subcutaneous autoinjector form that can be self-administered.⁴ The device delivers 1 mg dose into the thigh and does not require refrigeration, assembly, or priming. The hope is that the formulation may provide sustained pain relief and is intended for patients who experience issues with oral therapies, such as inefficacy, nausea, vomiting, or delayed dosing.⁵

“The DHE autoinjector is a really important milestone for patients living with acute migraines and cluster headaches. For the first time, it puts this type of treatment into a ready-to-use autoinjector that patients can administer themselves—giving them more control and the ability to act quickly during these painful attacks," Joe Renda, senior vice president and chief commercial officer – specialty at Amneal Pharmaceuticals, told NeurologyLive^®. "This approval is especially meaningful to me, having seen my sibling struggle with cluster headaches for many years and the toll it can take. Making treatment more accessible and actionable is a step forward for so many families."

FDA Clears Lumipulse Plasma Ratio as First Blood Test for Diagnosing Alzheimer Disease

A day later, May 16, the FDA cleared Fujirebio’s Lumipulse G p-tau217/ß-Amyloid 1-42 Plasma Ratio, a diagnostic blood test, for the early detection of amyloid plaques associated with Alzheimer disease (AD) in adults aged 55 and older. With the decision, it becomes the first such in vivo blood test used in the diagnosis of AD.⁶

The test operates on the Lumipulse G platform, a fully automated system that uses chemiluminescent enzyme immunoassay technology to quantify biomarkers with high sensitivity and precision. The p-tau217 protein, associated with neurofibrillary tangles in AD, and ß-Amyloid 1-42, which forms amyloid plaques, are measured in plasma using the Lumipulse G p-tau217/ß-Amyloid 1-42 Plasma Ratio to distinguish amyloid-positive individuals from amyloid-negative ones, giving patients a non-invasive alternative for early AD detection.

"Alzheimer disease impacts too many people, more than breast cancer and prostate cancer combine," Martin A. Makary, MD, MPH, FDA Commissioner, wrote in a statement.⁶ "Knowing that 10% of people aged 65 and older have Alzheimer's, and that by 2050 that number is expected to double, I am hopeful that new medical products such as this one will help patients."

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REFERENCES
1. Spinogenix Announces FDA-Authorized Expanded Access Program for SPG302, the First Synaptic Regenerative Therapy to Treat ALS. News Release. Spinogenix. Published May 5, 2025. Accessed June 9, 2025. https://www.spinogenix.com/spinogenix-announces-fda-authorized-expanded-access-programfor-spg302-the-first-synaptic-regenerative-therapy-to-treat-als/
2. Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy. News release. Capsida Biotherapeutics. May 12, 2025. Accessed June 9, 2025. https://capsida.com/capsida-receives-fda-ind-clearance-for-its-first-in-class-iv-administered-gene-therapy-for-stxbp1-developmental-and-epileptic-encephalopathy/
3. Amneal Receives U.S. FDA Approval for Brekiya® (dihydroergotamine mesylate) injection for the Acute Treatment of Migraine and Cluster Headaches in Adults. News Release. Amneal Pharmaceuticals. Published May 15, 2025. Accessed June 9, 2025. https://investors.amneal.com/news/press-releases/press-release-details/2025/Amneal-Receives-U-S--FDA-Approval-for-Brekiya-dihydroergotamine-mesylate-injection-for-the-Acute-Treatment-of-Migraine-and-Cluster-Headaches-in-Adults/default.aspx
4. Silberstein SD, Shrewsbury SB, Hoekman J. Dihydroergotamine (DHE) - Then and Now: A Narrative Review. Headache. 2020;60(1):40-57. doi:10.1111/head.13700
5. Aurora SK, Papapetropoulos S, Kori SH, Kedar A, Abell TL. Gastric stasis in migraineurs: etiology, characteristics, and clinical and therapeutic implications. Cephalalgia. 2013;33(6):408-415. doi:10.1177/0333102412473371
6. FDA Clears First Blood Test Used in Diagnosing Alzheimer’s Disease. News release. FDA. May 16, 2025. Accessed June 9, 2025. https://www.fda.gov/news-events/press-announcements/fda-clears-first-blood-test-used-diagnosing-alzheimers-disease