U.S. FDA Approves Marinus Pharma’s Lead Drug To Treat Genetic Disorder – Reuters.com

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly

March 18 (Reuters) – The U.S. health regulator has approved Marinus Pharmaceuticals Inc’s (MRNS.O) lead drug to treat seizures associated with a rare genetic disorder in patients two years of age and older, the company said on Friday, sending its shares soaring around 36%.

The oral drug, which will be sold under the brand name Ztalmy, is for treatment of seizures associated with CDKL5 deficiency disorder, a rare genetic epilepsy.

Ztalmy is expected to be commercially available in the United States in July following scheduling by the U.S. Drug Enforcement Administration, Marinus said in a statement.

Marinus has priced the drug at about $133,000 per patient per year at the wholesale level, while after discounts, including for Medicaid patients, it will cost an average of $105,000 per patient per year, Chief Executive Officer Scott Braunstein told Reuters.

At the wholesale level, the chronic medication translates to $2,425 per bottle, Braunstein said.

Cowen and Co analyst Joseph Thome expects the drug to garner peak sales of $71 million in the U.S. in 2029 for the treatment of CDKL5 deficiency disorder.

Ztalmy exhibits anti-seizure and anti-anxiety activity by affecting a type of protein in the central nervous system called GABAA receptor.

The approval by the Food and Drug Administration was based on data from a late-stage study in 101 patients that showed a 30.7% median reduction in 28-day major motor seizure frequency in patients treated with the drug, as compared to a 6.9% decline in those receiving placebo.

Reporting by Leroy Leo and Mrinalika Roy in Bengaluru; Editing by Maju Samuel and Shailesh Kuber

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