Acer, Relief's metabolic disorder therapy ACER-001 gets orphan drug status in EU

Aug. 12, 2022 9:12 AM ETAcer Therapeutics Inc. (ACER), RLFTF, RLFTYBy: Ravikash, SA News Editor

Low angle view of the large banner on the facade of the southern wing of the Berlaymont building, seat of the European Commission in Brussels, Belgium.


  • The European Commission granted orphan medicinal product designation to Acer Therapeutics (NASDAQ:ACER) and Relief Therapeutics' (OTCQB:RLFTF) (OTCQB:RLFTY) ACER-001 (sodium phenylbutyrate) to treat patients with Maple Syrup Urine Disease (MSUD).
  • ACER-001 was had received the U.S. Food and Drug Administration's (FDA) orphan drug designation for MSUD in 2014, the companies said in an Aug. 12 press release.
  • MSUD is a rare genetic metabolic disorder characterized by deficiency of an enzyme complex which is required to break down certain amino acids in the body.
  • In the EU, the orphan drug status is granted to therapies aimed at treating or preventing a serious or life-threatening disease which affects not more than five in 10K people in the EU. The designation has benefits, including up to 10 years of market exclusivity for the product, if approved.
  • ACER +6.76% to $1.58 premarket Aug. 12

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