Benzinga.com

Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease

Friday, the FDA approved Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson's ...
Seeking Alpha

Zevra’s MIPLYFFA™ (arimoclomol) Receives Approval from U.S. Food and Drug Administration for Use in Patients with Niemann-Pick Disease Type C (NPC)

XOMA Royalty is entitled to receive a mid-single digit royalty on MIPLYFFA™ sales and up to $52.6 million in milestones MIPLYFFA™ is now the sixth commercial asset in XOMA Royalty’s portfolio ...
Hosted on MSN

FDA Approves Miplyffa for Niemann-Pick Disease, Type C

WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has approved Miplyffa (arimoclomol) for the treatment of Niemann-Pick disease, type C (NPC), marking the first drug sanctioned to address ...
Seeking Alpha

Zevra Therapeutics Announces Multiple Datasets on MIPLYFFA® (arimoclomol) to be Presented at the International Congress of Inborn Errors of Metabolism (ICIEM)

MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) ...
Monthly Prescribing Reference

Miplyffa Now Available for Niemann-Pick Disease Type C

To start patients on Miplyffa, prescribers can fill out an enrollment form on Miplyffa.com. Miplyffa™ (arimoclomol) is now available for use in combination with miglustat for the treatment of ...
FiercePharma

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. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...
Morningstar

Zevra Therapeutics Announces MIPLYFFA® (arimoclomol) Featured in Presentations at the National Niemann Pick Disease Foundation Conference

CELEBRATION, Fla., July 11, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
Reuters

Zevra Therapeutics prices rare genetic disorder drug at up to $106,000 per month

Sept 23 (Reuters) - Zevra Therapeutics' (ZVRA.O), opens new tab drug, Miplyffa, for treating a rare and fatal genetic disorder will have a wholesale acquisition cost ranging between $40,000 and ...
Mena FN

Zevra Therapeutics Executes Distribution Agreement To Broaden Access To MIPLYFFA® For The Treatment Of Niemann-Pick Disease Type C (NPC)

(MENAFN- GlobeNewsWire - Nasdaq) CELEBRATION, Fla., Dec. 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing ...
Morningstar

Zevra Therapeutics Announces Multiple Datasets on MIPLYFFA® (arimoclomol) to be Presented at the International Congress of Inborn Errors of Metabolism (ICIEM)

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and ...