Zevra Therapeutics, Miplyffa and Niemann-Pick Disease
After prior snub, Zevra clinches FDA approval for rare lysosomal storage disorder drug Miplyffa
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. | The FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage disorder Niemann-Pick disease type C in conjunction with Johnson & Johnson’s enzyme inhibitor miglustat.
US FDA approves Zevra's treatment for rare genetic disease
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.
FDA scraps adcomm for Applied's rare disease drug ahead of November decision
After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat, the FDA has now decided that a planned advisory committee meeting won’t be required. | After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat,
FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C -- 2nd Update
Zevra Therapeutics has won Food and Drug Administration approval of Miplyffa for Niemann-Pick disease type C, or NPC, making it the first drug cleared in the U.S. for the rare neurodegenerative disorder.
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive
Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease
Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson's (NYSE:JNJ) enzyme inhibitor Zavesca (miglustat), is approved to treat neurological symptoms associated with NPC in adults and children two years of age and older.
Legionnaires’ disease, Gaston County
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Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
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