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 function. Mithra says it will continue to progress development of E4 for the HIE indication over the next two years and look for a clinical development partner.

François Fornieri, CEO of Mithra, commented, “The ODD for E4 in neonatal encephalopathy underlines the potential of our unique natural E4 estrogen platform in areas beyond women’s health, including neuroprotection. HIE is a serious and prevalent syndrome under the umbrella of NE that causes significant mortality and morbidity in infants. Limitations with current treatment options in terms of efficacy and access highlight the unmet medical need. Given the promising initial preclinical data and the ODD, we look forward to exploring this important indication further.”

Mithra is focused on developing therapeutics in the areas of contraception and fertility, menopause and osteoporosis, gynecological infections, and female cancers. Mithra’s lead development candidates are a fifth-generation oral contraceptive, Estelle®, and a next-generation hormone therapy, Donesta®, both of which are founded on Mithra's E4 platform. 

Earlier this month the firm reported signing an exclusive license and supply agreement for Tibelia®, its generic version of Livial® hormone therapy, in Canada, with an undisclosed Canadian partner.


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