NOW AVAILABLE IN THE U.S.

Products

Our mission is to bring life-changing therapeutics to people living with rare diseases.

Product Name

Indication

Commercial Partner

Milestone

Niemann-Pick Disease Type C Commercial Product

MIPLYFFA^TM

arimoclomol capsules

Niemann-Pick disease type C in patients age two and older

Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
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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 older.

For more information visit www.miplyffa.com.

Urea Cycle Disorders Commercial Product

Olpruva^®

sodium phenylbutyrate for oral suspension

Certain Urea Cycle Disorders

Zevra acquires Acer Therapeutics in 2023
View Press Release

OLPRUVA (sodium phenylbutyrate) for oral suspension approved in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

For more information visit www.olpruva.com.

ADHD Commercial Product

AZSTARYS^®

serdexmethylphenidate and dexmethylphenidate

Attention Deficit Hyperactivity Disorder (ADHD) in patients age six and older

Corium Inc.

Potential to reach one or more milestones during 2024 and continued royalty revenue.

Approved by the FDA in 2021, AZSTARYS is a prodrug for the treatment of ADHD in patients age six years or older.

For more information visit www.azstarys.com.

AZSTARYS® is a federally controlled substance (CII) because it can be abused or lead to dependence. Keep AZSTARYS® in a safe place to prevent misuse and abuse. Selling or giving away AZSTARYS® may harm others and is against the law.

OLPRUVA is a registered trademark of Acer Therapeutics Inc., a wholly owned subsidiary of Zevra Therapeutics, Inc.