(Reuters) – The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, becoming the first treatment to receive the green light 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 refused to approve it and extended a review of the treatment.
The oral drug, known 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, marketed as Zavesca, has been approved 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.
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