FDA Drug Approvals, Neurology — 2022 Midyear Review

August 24, 2022

Ztalmy (ganaxolone)

Ganaxolone is a GABAA receptor–positive modulator. It binds specifically to GABAA receptors to enhance their inhibitory effects. It is indicated for seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2 years and older. CDD, a rare developmental epileptic encephalopathy, is largely a disease of pediatric and young adult patients.

Approval was based on the phase 3 MARIGOLD trial. Patients treated with ganaxolone (n = 49) showed a median 30.7% reduction in 28-day major motor seizure frequency, compared to a 6.9% reduction for those receiving placebo (n = 51) (P=0.0036). In the open-label extension study, patients treated with ganaxolone for at least 12 months (n = 48) experienced a median 49.6% reduction in major motor seizure frequency. Prescribing Information

Other neurology approvals

Adlarity (donepezil transdermal) – New transdermal patch indicated for treatment of mild, moderate, and severe Alzheimer dementia; originally approved as tablet and oral disintegrating tablet.

Hyftor (sirolimus topical) – New dosage form indicated for treatment of facial angiofibroma associated with tuberous sclerosis in patients aged 6 years and older.

Fintepla (fenfluramine) – New indication for seizures associated with Lennox-Gastaut syndrome (previously approved for seizures associated with Dravet syndrome) in patients aged 2 years and older.

Ultomiris (ravulizumab) – C5 complement inhibitor, new indication for generalized myasthenia gravis.

Radicava ORS (edaravone) – New oral suspension dosage form for adults with ALS.


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