NeurologyLive® Year in Review 2024: Most-Read FDA Approval Stories

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 2024, 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-viewed FDA coverage that appeared on NeurologyLive^® this year. Click the buttons to read more about these agency decisions.

1. FDA Approves Takeda’s Immune Globulin Infusion, Hyqvia, for Chronic Inflammatory Demyelinating Polyneuropathy

JANUARY 16, 2024 — The FDA approved immune globulin (IG) infusion 10% (human) with recombinant human hyaluronidase (Hyqvia; Takeda Pharmaceuticals), a liquid medicine, as maintenance therapy for adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to prevent relapse of neuromuscular disability and impairment.

“With the FDA approval of Hyqvia for CIDP, which builds on our expertise in rare neuroimmunological and neuromuscular disorders, we can now offer a personalized maintenance treatment option for adults with this debilitating disease. Research and clinical experience have shown that IG therapy is effective as maintenance treatment in adults with CIDP, and we hope that this approval for Hyqvia is the first of several around the world as we strive to deliver our broad and diverse IG portfolio to more people with complex neuroimmunological diseases.”

– Giles Platford
President of Takeda’s Plasma-Derived Therapies Business Unit

2. FDA Approves Italfarmaco's Givinostat for Duchenne Muscular Dystrophy

MARCH 21, 2024 — Months after an extended review process, the FDA has approved Italfarmaco SpA's givinostat (Duvyzat), a proprietary histone deacetylase (HDAC) inhibitor, as a treatment for patients with Duchenne muscular dystrophy (DMD). The treatment is designed to inhibit HDACs, enzymes that prevent gene translation by changing the 3-dimensional folding of DNA in the cell. The decision makes it the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

“We are pleased that the FDA approved givinostat for individuals with Duchenne age 6 and older. This adds to the list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation."

– Debra Miller
Founder and Chief Executive Officer of CureDuchenne

3. FDA Approves Alexion's Ravulizumab-cwvz for Neuromyelitis Optica Spectrum Disorder

MARCH 25, 2022 — The FDA approved ravulizumab-cwvz (Ultomiris; Alexion), a terminal compliment C5 inhibitor, for the treatment of patients with anti-aquaporin (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). With the decision, ravulizumab-cwvz becomes the fourth approved therapy for this patient population, following behind eculizumab (Soliris; Alexion), inebilizumab (Uplizna; Horizon Therapeutics), and satralizumab (Enspryng; Genentech).

“Complement inhibition is a very powerful treatment for prevention of NMOSD relapses. Now that ravulizumab-cwvz is approved, it makes it easier for patients to take a once every 2 months infusion rather than once every 2 weeks. The safety issues regarding infections with encapsulated organisms are essentially the same as eculizumab and other complement inhibitors. I am hopeful an improved [Risk Evaluation and Mitigation Strategy] program will help to address those concerns.”

– Michael Levy, MD, PhD
Associate Professor of Neurology at Harvard Medical School

4.FDA Approves One Pill, Once-Daily Tablets of Deutetrabenazine for Tardive Dyskinesia, Chorea

MAY 29, 2024 — The FDA approved a new one-pill, once-daily tablet administration option for deutetrabenazine (Austedo XR; Teva Pharmaceuticals), a medication for tardive dyskinesia and chorea associated with Huntington disease (HD). Deutetrabenazine, a vesicular monoamine transporter 2 (VMAT2) inhibitor, is now available in 4 tablet strengths (30, 36, 42, and 48 mg), adding on to its original dosing options of 6 mg, 12 mg, and 24 mg.

"Since our launch of Austedo in 2017, we have been committed to helping people living with TD and HD chorea treat these chronic, involuntary movements. Austedo, backed by the longest efficacy and tolerability data to date, has continued to evolve—having received approval for Austedo XR, our once-daily extended-release formulation in February 2023. This latest milestone offers a streamlined treatment regimen for clinically therapeutic doses with the broadest dosing flexibility."

– Dell Faulkingham
Senior Vice President, and Head of Innovative Medicines at Teva

5. FDA Grants Traditional Approval to Elevidys Gene Therapy for Ambulatory DMD, Accelerated Approval for Nonambulatory Patients

JUNE 20, 2024 — Months after the FDA accepted and filed Sarepta Therapeutics’ efficacy supplement to the biologics license application for SRP-9001 (Elevidys), otherwise known as delandistrogene moxeparvovec-rokl, the agency granted it traditional approval for ambulatory patients with DMD. With traditional approval, SRP-9001's indication has been expanded to include patients aged 4 years and older with DMD who have a confirmed mutation in the DMD gene.

“We are delighted that the FDA has expanded the label for Elevidys, Sarepta’s gene therapy for DMD to include ambulatory and non-ambulatory Duchenne patients who are older than 4 years of age. Families facing Duchenne have an urgent need for treatments that will delay the progression of the disease and this represents a significant treatment option for many boys and young men with Duchenne. We are grateful to Sarepta Therapeutics, the FDA, and of course the many families who have participated in clinical trials to advance this critical research."

– Debra Miller
Founder and CEO of CureDuchenne

6. FDA Approves Efgartigimod as New Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

JUNE 21, 2024 — The FDA approved Argenx's coformulation therapy efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo) as a new treatment for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The therapy is the first and only neonatal Fc receptor (FcRn) blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.

“Argenx continues to pursue our ambition to turn science into solutions for patients with severe autoimmunity. Patients have been waiting, and today, argenx is delivering the first innovative treatment for CIDP in more than 30 years. Vyvgart Hytrulo is a precision tool that has been shown to drive meaningful benefits for patients. Today’s FDA approval means that CIDP patients have a transformational new treatment option and further affirms the therapeutic profile of Vyvgart Hytrulo and the potential of FcRn blockade in IgG-mediated autoimmune diseases.”

– Luc Truyen MD, PhD
Chief Medical Officer of Argenx

7. FDA Approves Pitolisant for Excessive Daytime Sleepiness in Pediatric Narcolepsy

JUNE 24, 2024 — Months after being granted priority review designation by the FDA, the agency has granted approval to Harmony Biosciences' pitolisant (Wakix), a selective histamine H3 receptor inverse agonist, for the treatment of excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy. In addition, the agency issued pitolisant a complete response for the treatment of cataplexy in pediatric patients with narcolepsy.

"Following the FDA's decision to grant priority review, we are very pleased with the agency's timely review and approval of Wakix for pediatric narcolepsy patients with EDS. EDS is the primary symptom experienced by all patients with narcolepsy and this approval for Wakix, as the first-and-only FDA-approved non-scheduled treatment option for narcolepsy, makes this important treatment option available to pediatric patients 6 years and older living with narcolepsy."

– Jeffrey M. Dayno, MD,
President and chief executive officer at Harmony Biosciences

8. FDA Approves New-Generation Acetylcholinesterase Inhibitor ALPHA-1062 for Mild-to-Moderate Alzheimer Disease

JULY 29, 2024 — The FDA approved Alpha Cognition’s ALPHA-1062 (Zunveyl), a prodrug of an approved acetylcholinesterase inhibitor (AChEI), galantamine, as a treatment for patients with mild-to-moderate Alzheimer disease (AD). ALPHA-1062, a delayed-release oral tablet formulation, is considered a new-generation AChEI inhibitor, with expected minimal gastrointestinal adverse events (AEs).

“I am very excited about the approval of Zunveyl, which we believe offers better tolerability for patients with AD. We have always believed in the efficacy of galantamine but have been limited in its use because of tolerability issues. To now have an agent with the efficacy of galantamine, but that also offers the hope of better tolerability, will provide physicians a great option to treat patients. This advancement marks a meaningful step forward in improving the quality of life for those living with Alzheimer and their families. As a geriatric psychiatrist specializing in AD, I am eager to incorporate this new treatment into our practice and see the positive difference it will make.”

– Elaine Peskind, MD
Friends of Alzheimer’s Research Professor of Psychiatry at the University of Washington School of Medicine

9. FDA Approves Carbidopa/Levodopa ER Capsules Formulation IPX203 for Parkinson Disease

AUGUST 7, 2024 — The FDA approved Amneal Pharmaceuticals’ investigational agent IPX203, an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules, as a treatment for patients with Parkinson disease (PD), according to an announcement from the company. Marketed as Crexont, the therapy is an oral formulation CD/LD which includes immediate-release (IR) granules as well as extended-release (ER) pellets.

"The treatment goals for people living with Parkinson’s disease include achieving a more robust duration of benefit per dose of LD, reducing OFF time, and simplifying dosing regimens. Crexont’s longer Good ON time per day and per dose represents a substantial advancement in managing motor symptoms and maintaining more consistent therapeutic effects, which is very encouraging for both patients and the Parkinson’s community."

– Robert A. Hauser, MD
Professor of Neurology at the University of South Florida

10. FDA Approves Subcutaneous Formulation of Ocrelizumab for Relapsing and Progressive Multiple Sclerosis

SEPTEMBER 13, 2024 — The FDA approved a new subcutaneous formulation of ocrelizumab (Ocrevus; Roche), a humanized monoclonal antibody designed to target CD20-positive B cells, as a treatment for relapsing forms of multiple sclerosis (MS) and primary progressive MS (PPMS), its original indication. Marketed as Zunovo, this expands on the administration options of ocrelizumab, which was originally approved in 2017 as an infusion.

"Ocrevus Zunovo gives patients and providers another option for receiving Ocrevus, building on a decade of robust safety and efficacy data for Ocrevus in multiple sclerosis. Today’s approval may offer greater flexibility for healthcare providers and people living with multiple sclerosis, based on their individual treatment needs."

– Levi Garraway, MD, PhD
Chief Medical Officer and Head of Global Product Development at Genentech