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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