US FDA approves Zevra's treatment for rare genetic disease

 The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the first treatment to get a nod for the condition, the health regulator said on Friday.

The company has been trying for years to bring the drug to market after the FDA previously declined to approve it and extended a review of the treatment.

The oral drug, branded as Miplyffa, has now been approved for the treatment of Neimann-Pick disease type C - a rare genetic condition that affects the nervous system and other organs.

On average, people affected by this disease only live for about 13 years.

"The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering," said Janet Maynard, a director at the FDA's Center for Drug Evaluation and Research.

Miplyffa, in combination with miglustat, which is branded as Zavesca, has been cleared to treat neurological symptoms associated with NPC in adults and children 2 years of age and older.

The drug is expected to be available in the U.S. in eight to 12 weeks, Zevra said.

The company did not immediately respond to a Reuters request for comment on pricing.

H.C. Wainwright analyst Oren Livnat expects the average net price of the drug to between $500,000 and $600,000 per year.

Livnat estimates peak sales of about $250 million in the U.S.

Miplyffa comes with a warning for hypersensitivity reactions including hives and angioedema, a condition that causes swelling under the skin.

Zevra gained access to the drug through its acquisition of Orphazyme, the original developer, in 2022.

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