Mithra’s E4 Estrogen Wins EU Orphan Drug Designation for Treating HIE in Newborns

Belgium-based Mithra Pharmaceuticals received orphan drug designation (ODD) from the European Medicines Agency (EMA) for its estrogen candidate estetrol (E4) as a treatment for neonatal encephalopathy (NE). The firm intends to develop the drug for treating hypoxic ischemic encephalopathy (HIE), a form of NE that it says accounts for up to 80% of cases and affects about 30,000 neonates each year in the EU and U.S. E4 is a natural estrogen produced by the human fetal liver during pregnancy.

Mithra is a women’s health specialist, but says it plans to investigate the potential for E4 in a range of indications, including neuroprotection and wound healing. ODD designation for E4 in Europe for the NE indication was based on preclinical data from a study conducted by Mithra in collaboration with the University of Liège, Belgium, which demonstrated improvements in pathophysiology, general well-being, and motor

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