Relief And Acer Therapeutics Secures Orphan Drug Designation From European Union For Metabolic Disorder Candidate


The European Commission has granted orphan medicinal product designation in the EU to Relief Therapeutics and Acer Therapeutics’ ACER lead asset ACER-001 (sodium phenylbutyrate) for the potential treatment of patients with Maple Syrup Urine Disease (MSUD).

ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). 

Raghuram (Ram) Selvaraju, Chairman, stated: "Orphan designation by the European Commission is another important milestone for the ACER-001 program that provides further validation of the important role we believe ACER-001 will play in the potential treatment of multiple rare diseases.”

Relief Therapeutics has a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001 (sodium phenylbutyrate).

ACER-001 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in the United States.

Maple Syrup Urine Disease (MSUD) is a rare inherited disorder caused by a deficiency of branched-chain alpha-keto acid dehydrogenase complex, If left untreated, that can cause neurological damage, mental disability, coma, or death.

Price Action : Acer Therapeutics shares are trading around 1 percent down at $1.47 on Friday at the time of publication.

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