Special Report: 2024 Neurology Drug & Device Approvals

ALZHEIMER DISEASE & DEMENTIA

In 2024, we saw the approval of Kisunla (donanemab-azbt; Eli Lilly and Company, Indianpolis, IN), a monoclonal antibody (mAb) administered as a once-monthly injection for intravenous (IV) use in people with early symptomatic Alzheimer disease (AD). Additionally, 510(k) clearances were granted to 3 software tools that assist in the clinical assessment of brain images: Brainsee (Darmiyan, San Francisco, CA), NeuroQuant v5.0 (Cortechs.ai, San Diego, CA), and Icobrain Aria (Icometrix, Boston, MA)

CHILD NEUROLOGY

In 2024, the FDA approved the first 2 drugs indicated for the treatment of adult and pediatric patients with Niemann-Pick disease type C (NPC). First, Miplyffa (arimoclomol; Zevra Therapeutics, Celbration, FL) was approved for use in combination with miglustat for the treatment of neurologic manifestations of NPC, administered as an oral capsule 3 times daily. Aqneursa (levacetylleucine; IntraBio, Austin, TX) was subsequently approved as the first ever stand-alone therapy for NPC, a suspension for oral administration or administration via gastronomy tube up to 3 times daily, depending on patient body weight.

EPILEPSY & SEIZURES

In 2024, the FDA approved Libervant (diazepam buccal film; Aquestive Therapeutics, Warren, NJ) for pediatric patients with epilepsy for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity. Libervant is a buccal film administered on the inside of the mouth on top of the surface of the cheek. Additionally, 510(k) clearances were granted to 2 EEG devices, ONE (Zeto, Santa Clara, CA) and BrainWatch (Natus, Middleton, WI), which both have applications in monitoring seizure activity.

HEADACHE & PAIN

One device received 510(k) clearance for headache in 2024: Nerivio (Theranica, Bridgewater Township, NJ), a transcutaneous electrical stimulator for the treatment of acute migraine. Nerivio is a self-administered device for both acute and preventive treatment in the home setting.

IMAGING & TESTING

There were 3 510(k) clearances in 2024 for devices that have indications for imaging and testing. The SmartFrame OR Stereotacatic System (ClearPoint Neuro, Solana Beach, CA) is a stereotaxic instrument with applications in surgical procedures, whereas NeuroLF (Positrigo, Zurich, Switzerland) is an emission computed tomography system that can assist in the diagnosis and management of neurologic conditions. Smart Skin (X-trodes System M; X-trodes, Tel Aviv, Israel) is a physiologic signal amplifier with utility for people with cardiovascular and sleep disorders.

NEUROMUSCULAR DISORDERS

For neuromuscular disorders, 3 new drugs received FDA approvals, including generic deflazacort (Tris Pharma, Monmouth Junction, NJ), Lenmeldy (atidarsagene autotemcel; Orchard Therapeutics, London, UK), and Duvyzat (givinostat; italfarmaco SpA, Milan, Italy). Additionally, the FDA broadened the indication for Elevidys (delandistrogene moxeparvovec-rokl; Sarepta Therapeutics, Cambridge, MA) to treat children aged ≥4 years with Duchenne muscular dystrophy (DMD).

MOVEMENT DISORDERS

In 2024, we saw the approval of Crexont (carbidopa and levodopa, Amneal Pharmaceuticals, Bridgewater, NJ), a novel formulation of carbidopa/levodopa for the treatment of Parkinson disease (PD). There also were 3 approvals for new indications or formulations for Ingrezza (valbenazine; Neurocrine Biosciences, San Diego, CA), Austedo (deutetrabenazine; Teva Neuroscience, Kansas City, MO), and Brineura (cerliponase alfa; BioMarin Pharmaceuticals, San Rafael, CA). Additionally, the Percept RC DBS system (Medtronic, Minneapolis, MN) received 510(k) clearance for the suppression of tremor using thalamic stimulation.

NEURO-ONCOLOGY

There was one approval in 2024 for a drug in neuro-oncology. Voranigo (vorasidenib; Servier, Boston, MA), an oral tablet, received approval for adult and pediatric patients with specific forms of Grade 2 astrocytoma or oligodendroglioma. Treatment with Voranigo is indicated only after surgery.

MS & IMMUNE DISORDERS

Ocrevus Zunovo (ocrelizumab & hyaluronidase-ocsq; Genentech, South San Francisco, CA), a novel subcutaneous formulation of ocrelizumab, received FDA approval in 2024 for the treatment of people with multiple sclerosis (MS). Additionally, the FDA approved new indications for HyQvia (Immune Globulin Infusion 10% [Human] with Recombinant Human Hyaluronidase; Takeda, Cambridge, MA), Gammagard Liquid (Immune Globulin Infusion [human] 10% solution; Takeda, Cambridge, MA), Ultomiris (ravulizumab-cwvz; Alexion, Boston, MA), and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc; Argenx, Amsterdam, Netherlands).

TBI

In 2024, the i-STAT TBI Cartridge (Abbott, Chicago, IL) blood test device received 510(k) clearance for use in evaluating mild traumatic brain injury (mTBI).

STROKE

In stroke, there were 4 new 510(k) device clearances in 2024. Viz ICH Plus (Viz.ai, San Francisco, CA), Cina-ASPECTS (Avicenna.AI, La Ciotat, France), and AngioFlow (RapidAI, San Mateo, CA) were approved for automated or computer-assisted analysis of brain images, while Neuroblade System (Clearmind Biomedical, San Jose, CA) was cleared as an endoscope for use during surgery.

SLEEP

In 2024, 2 oral tablets, Wakix (pitolisant; Harmony Biosciences, Plymouth Meeting, PA) and Lumryz (sodium oxybate; Avadel Pharmaceuticals, Dublin, Ireland) received FDA approvals for expanded indications in the treatment of excessive daytime sleepiness (EDS), cataplexy, and narcolepsy. Additionally, the SANSA (Huxley Medical, Atlanta, GA) breathing monitor device and the Happy Ring Health Monitoring System (Happy Health, Austin, TX) cardiac monitor device received 510(k) clearances for use in sleep