Joy at Egetis after positive CHMP opinion – ”The single most important milestone”
Av The announcement came after a review that has been ongoing within the European Medicines Agency (EMA) since October last year, and means that its Committee for Medicinal Products for Human Use (CHMP) recommends the European Commission to authorize Emcitate (tiratricol) for marketing.
“This is the single most important milestone in Egetis’ history and a major step forward in building a sustainable rare disease company. We look forward to launching tiratricol in the EU pending the European Commission grants the marketing authorization, says CEO Nicklas Westerholm in a statement.
Patients with MCT8 deficiency have low levels of thyroid hormone in the central nervous system and increased concentrations of active thyroid hormone T3 in peripheral tissues. This can lead to symptoms like neurological developmental delay and intellectual disability.
The first symptoms usually appear in infancy, when the first signs show that the child is not developing at the same pace as other children.
The disease is caused by a mutation on the X chromosome and affects approximately 1 in 70,000 boys. At present, there is no approved treatment for the disease.
The positive opinion for Emcitate is based, among other data, on a follow-up study that included long-term effects and safety in 67 patients. The results indicated that the key features of MCT8 deficiency were alleviated for up to six years in patients across all ages.
Egetis is currently conducting a pivotal, randomized, placebo-controlled study in patients with MCT8 deficiency to support the submission of a New Drug Application in the USA.
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