Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), and its collaboration partner, Acer Therapeutics Inc. (Nasdaq: ACER) (Acer), announced that the European Commission has granted orphan medicinal product designation in the EU to ACER-001 (sodium phenylbutyrate) for the potential treatment of patients with Maple Syrup Urine Disease (MSUD). ACER-001 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in the U.S. for MSUD in 2014.
Orphan drug designation is granted to medicines that treat or prevent a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of not more than 5 in 10,000, and with either no currently approved method of prevention or treatment, or with significant benefit to those affected by the disease. The designation potentially provides certain benefits to ACER-001, including the potential for up to 10-year EU market exclusivity upon regulatory approval, if received, reductions in EMA application fees, and access to study protocol assistance.
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