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Will the FDA approve another treatment for Niemann-Pick disease, type C by Dec 31, 2024?
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FDA official announcements and press releases
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FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C
Sep 20, 2024, 03:42 PM
The U.S. Food and Drug Administration (FDA) has approved the first oral medication to treat Niemann-Pick disease, type C (NPC). The drug, developed by Zevra Therapeutics and named Miplyffa (arimoclomol), is approved in combination with miglustat for patients aged 2 and older. This marks the first treatment to receive approval for this rare and fatal genetic disorder. The approval was announced on Friday. Zevra Therapeutics is listed under the ticker symbol ZVRA.
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