Sept 20 (Reuters) - The U.S. Food and Drug
Administration approved Zevra Therapeutics' ( ZVRA ) 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.