Modis Therapeutics announced today that the FDA has granted Breakthrough Therapy designation to MT1621, Modis’ investigational treatment for patients with thymidine kinase 2 deficiency (TK2d).
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious or life-threatening condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). A drug that receives Breakthrough Therapy designation is eligible for intensive guidance on an efficient drug development program, organizational commitment involving senior managers from the FDA, and all Fast Track designation features, including eligibility for Accelerated Approval and Priority Review, if relevant criteria are met.
Modis’ 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 in October 2018. MT1621 has also been granted PRIME designation by the EMA and Orphan Drug designation by both the FDA and EMA.
We are very pleased that – by granting MT1621 Breakthrough Therapy and PRIME designations – now both FDA and EMA have recognized the urgent need in TK2d patients and encouraging preliminary data that Modis and its collaborators have collected. We are looking forward to coordinating with FDA and EMA on the registration plan for MT1621 for TK2d patients,
said Joshua Grass,
Chief Executive Officer of Modis Therapeutics.