US FDA approves Zevra’s treatment for rare genetic disease

(Reuters) -The U.S. Food and Drug Administration 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 U.S. health regulator 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 disorder that affects the nervous system and other organs.

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