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US FDA approves Zevra's treatment for rare genetic disease
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US FDA approves Zevra's treatment for rare genetic disease
Sep 22, 2024 9:34 AM

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.

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