Biopharmaceutical company Modis Therapeutics revealed on Tuesday the receipt of European Medicines Agency (EMA) PRIME (PRIority MEdicines) designation for its investigational MT1621 for patients with thymidine kinase 2 deficiency (TK2d).

Thymidine kinase 2 deficiency (TK2d) is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d may present at all ages and causes progressive and severe muscle weakness, respiratory insufficiency and is often fatal.

The company added that MT1621 is an investigational deoxynucleoside combination therapy that targets the underlying pathophysiology of TK2 deficiency. Deoxynucleoside combination therapy has been shown to improve cell function and prolong life in preclinical models of TK2d.

According to the company, the application was supported by data from initial clinical studies in TK2d patients, some of which was presented by Dr Michio Hirano at the International World Muscle Society (WMS) Congress last month. The data from the initial clinical studies suggest that this therapy may alter the course of disease in patients with TK2d. It is planning to conduct additional clinical studies with the goal of obtaining regulatory approval to make MT1621 available to patients globally.

Through PRIME, the EMA offers enhanced support to medicine developers including early interaction and dialogue and a pathway for accelerated evaluation by the agency. It is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible.