NeurologyLive® Year in Review 2025: Most Impactful FDA Decisions

Each year, the NeurologyLive^® team tracks the progress of hundreds of therapeutic potentials through the clinical development pipeline. Eager to update the clinical community on the available options for the treatment of neurologic diseases, the staff is quick to provide the latest on FDA decisions that add to that collection of therapies. Aligned with the various conversations with experts on the clinical trial data on these therapies, the team provides coverage the moment these decisions happen.

In 2025, particularly, there was much FDA activity to track—there were many agency decisions, advisory committee meetings, and application submissions and acceptances, among other notable undertakings. Some patient populations saw first-ever approvals, while others were granted access to more convenient administration modalities or new formulations. Rare diseases reported therapeutic advances that had been years in the making, and a number of devices and digital platforms that provide new tools to clinicians received clearance from the agency—and all the while, our team was there to share the news.

Here, we'll highlight the most-impactful FDA coverage that appeared on NeurologyLive^® this year. Click the buttons to read more about these agency decisions.

Click the listen now buttons for more details and information about each episode.

1. FDA Approves Axsome Therapeutics’ AXS-07 for Migraine Treatment

JANUARY 30, 2025 — Months after the FDA accepted Axsome Therapeutics’ resubmission of its new drug application (NDA) in September 2024, the agency approved AXS-07, an oral, rapidly absorbed, multi-mechanistic agent, as a new acute treatment of migraine with or without aura in adults. AXS-07, a novel agent, is thought to act by inhibiting calcitonin gene-related peptide (CGRP) release, reversing CGRP-mediated vasodilation, and inhibiting neuroinflammation, pain signal transmission, and central sensitization.

"In terms of use in clinical practice, we want to treat our patients with the simplest, most effective acute treatments that we're able to offer them. So, you would not use a combination triptan and non-steroidal product in a patient whose treatment needs were fully met, either by a triptan alone or by a non-steroidal anti-inflammatory alone," Richard B. Lipton, MD, professor of neurology and director of the Montefiore Headache Center at Albert Einstein College of Medicine, told NeurologyLive at the time of the approval. "In fact, the design for individuals with unmet needs on single, monotherapy acute treatments was specifically a reflection of the fact that those are the patients who need combination therapy. I think that's something most prescribers would do and should do."

2. FDA Approves Vertex Pharmaceuticals' Suzetrigine for Acute Pain Management

JANUARY 30, 2025 — The FDA approved suzetrigine (Vertex Pharmaceuticals), an oral selective NaV1.8 pain signal inhibitor, to treat patients who experience moderate-to-severe acute pain. Marketed as Journavx, the novel, non-opioid therapy became the first new class of medicine to treat acute pain in over 20 years.

"This is the first new and nonopioid pain class we've had at our disposal in over 20 years. It provides a selective, nonaddictive alternative to opioids for treatment of moderate to severe acute pain," Jessica Oswald, MD, MPH, steering committee member at Vertex and associate professor in pain management and emergency medicine at UC San Diego, told NeurologyLive. "This offers an effective alternative to opioids, and it expands our nonopioid armamentarium. In the emergency department, I'm frequently stuck. I have patients with acute pain, and I have opioids, and I have to get creative with some of our available options. So this really provides one additional medication that I can use in my toolbox."

3. FDA Approves Apomorphine Infusion Device SPN-830 as New Parkinson Treatment

FEBRUARY 4, 2025 — Nearly 4 and a half years since its original submission, the FDA approved Supernus Pharmaceuticals’ investigational agent SPN-830 (Onapgo) as the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD).

"People living with Parkinson's and their families are hungry for new treatment options, and the PMD Alliance community was thrilled to learn about SPN-830's approval. Every newly approved Parkinson's therapy is a win for our community," Andréa Merriam, CEO of the Parkinson & Movement Disorder Alliance, told NeurologyLive. "The introduction of this therapy brings with it an increased need for education, and underscores the importance of clinician-patient communication. PMD Alliance produces resources and education to help patients understand their symptoms, for personal empowerment, but also to streamline clinic visits. Our annual healthcare provider conference, Advanced Therapeutics in Movement and Related Disorders (ATMRD), equips clinicians with the information to integrate new therapies into practice."

4. FDA Approves Tenecteplase for Acute Ischemic Stroke

MARCH 3, 2025 — The FDA approved tenecteplase (TNKase; Genentech) for the treatment of acute ischemic stroke (AIS) in adults, according to a Genentech announcement. The thrombolytic medicine is an intravenous (IV) tissue plasminogen activator that is clot-dissolving, administered as a single 5-second IV bolus.

"Today’s approval is a significant step forward and underscores our commitment to advancing stroke treatment options for patients. TNKase provides a faster and simpler administration, which can be critical for anyone who is dealing with an acute stroke," Levi Garraway, MD, PhD, the chief medical officer and head of Global Product Development at Genentech, said in a statement, following the approval.

5. FDA Approves Diazoxide Choline Extended-Release Tablets for Hyperphagia in Prader-Willi Syndrome

MARCH 25, 2025 — The FDA approved Soleno Therapeutics' diazoxide choline (DCCR) extended-release tablets, marketed as Vykat XR, for the treatment of Prader-Willi syndrome (PWS) for patients 4 years and older who have hyperphagia. DCCR, a novel, proprietary extended-release dosage form containing diazoxide choline — the crystalline salt of diazoxide — became the first therapy to treat hyperphagia in PWS.

“My immediate reaction was relief - I have been working with PWS for [over] 25 years to try to find an answer to the life-limiting issue of hyperphagia in this syndrome, so I was grateful that we now have an effective medication. I was one of the [principal investigators] for the study and had 33 patients on trial at my site, so got to see firsthand how well this medication worked for patients,” Jennifer Miller, MD, professor of pediatric endocrinology at the University of Florida, told NeurologyLive, following the approval. "I think it will change the treatment landscape by offering hope to patients and families with this condition. The concept of having a starving child and not being able to feed them is devastating for parents to contemplate. For physicians it offers the possibility of treatment in a space where there is currently none. That is huge."

6. FDA Approves Prefilled Syringe Administration for FcRn Modulator Efgartigimod

APRIL 10, 2025 — The FDA approved a new prefilled syringe administration route for efgartigimod (Vyvgart; Argenx), a marketed medication for generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP). With the decision, it offers patients greater convenience and flexibility, allowing in-home administration and reducing the number of time-consuming trips that typically come with intravenous (IV) infusions.

“Today’s FDA approval provides a new self-injection option across both approved indications in the U.S. that is designed for patients who seek more independence with their treatment,” Luc Truyen, MD, PhD, chief medical officer, Argenx, said in a statement at the time of the approval. "We understand patients experience MG and CIDP in different ways, and our prefilled syringe is an important innovation that provides patients with more freedom and flexibility to self-administer Vuvgart Hytrulo. Whether patients prefer to receive their treatment in a physician’s office, at home, or while traveling, they can experience treatment on their own terms and continue to benefit from Vivgart Hytrulo’s favorable safety profile and strong efficacy.”

7. FDA Approves FcRn Blocker Nipocalimab for Broad Forms of Generalized Myasthenia Gravis

APRIL 30, 2025 — The FDA approved Johnson & Johnson’s nipocalimab, marketed as Imaavy, as a new treatment for adults and pediatric patients aged 12 years and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive.

"We consistently hear from individuals living with myasthenia gravis who are hopeful for new treatment options that may help bring greater stability, independence and predictability to their lives," Samatha Masterson, president and chief executive officer at Myasthenia Gravis Foundation of America, said in a statement. "Today's announcement provides another option which could help address the constant uncertainty and heavy physical and mental toll that MG symptom relapse presents to patients and their families."

8. FDA Approves Updated Label for Alzheimer Therapy Donanemab to Lower ARIA-E Risk

JULY 9, 2025 — The FDA approved an updated label for donanemab (Kisulna; Eli Lilly), an FDA-approved therapy for early-stage Alzheimer disease (AD), to now include a new recommended titration dosing schedule, which may lead to lowered risk of amyloid-related imaging abnormalities (ARIA). In the latest updated labeling, the new recommended dosing regimen involves a more gradual titration, while still preserving the drug’s effect on amyloid plaque removal and phosphorylated tau 217 (p-tau) reduction.

"We are confident that this label update for Kisunla will significantly aid healthcare professionals in evaluating appropriate treatment options for their patients," Brandy Matthews, MD, FAAN, vice president of Global & US Medical Affairs for Alzheimer’s Disease at Eli Lilly, said in a statement. "This update underscores our unwavering commitment to patient safety and the advancement of Alzheimer's disease treatment by potentially mitigating the risk of ARIA-E."

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

AUGUST 8, 2024 — 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. The system is powered and controlled by a wearable component, which is fully upgradable, allowing patients with OSA to access the latest technology without needing additional surgeries for updates or battery replacements.

"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 at the time of the approval. “This unique bilateral stimulation technology has demonstrated consistent efficacy across all sleeping positions, including the challenging supine position, while maintaining an excellent safety profile.”

10. FDA Approves Doxecitine and Doxribtimine Combination Therapy as First Treatment for Thymidine Kinase 2 Deficiency

NOVEMBER 3, 2025 — The FDA approved UCB’s doxecitine (dC) and doxribtine (dT), a fixed-dose combination therapy, as the first treatment for patients with thymidine kinase 2 deficiency (TK2d), an ultra-rare neuromuscular disorder that causes issues with myopathy, difficulty walking, and breathing, among others. Marketed as Kygevvi, this combination treatment targets the root cause of TK2d, giving patients and families newfound hope for treating the disease.

"The approval of doxecitine and doxribtimine represents a pivotal moment for the TK2d community who previously had no FDA-approved treatment options for this rare genetic mitochondrial disease beyond supportive [palliative] care," Donatello Crocetta, chief medical officer at UCB, said in a statement at the time of the approval. "We extend heartfelt thanks to the patients, families and friends, advocates, healthcare providers and dedicated clinical trial teams who have partnered with us on this important journey."

Click here to view more of our FDA coverage.