EMA grants orphan drug designation to Mithra’s E4 treatment for neonatal encephalopathy


Mithra has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for its neonatal encephalopathy (NE) treatment of Estetrol (E4).

The company plans to develop E4 to be used in the treatment of hypoxic-ischemic encephalopathy (HIE), a subset of NE which accounts for 50% to 80% of cases and affects pre-term and newborn babies.

The ODD was granted based on E4's preclinical results in HIE, as well as improvements in pathophysiology, general well-being, and motor function.

Mithra is currently evaluating the capability of E4 in a wider range of indications, such as neuroprotection and wound-healing.

Over the next two years, the pharmaceutical firm intends to advance the preclinical study of the potential neuroprotective properties of E4 in HIE.

"Given the promising initial preclinical data and the ODD, we look forward to exploring this important indication further."

Mithra Pharmaceuticals CEO François Fornieri said: “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.”

HIE results from the reduction in the supply of blood or oxygen to the baby's brain before, during or shortly after birth.

In addition to causing death, it also results in severe neurological impairment and long-term disability among survivors.