FDA awarded the title of elamipretide orphan drug

Source: Bio Valley October 10, 2017 / Bio Valley / recently, stearth biotherapeutics announced that the FDA awarded the company the title of elamipretide orphan drug, a drug for the treatment of primary mitochondrial myopathy (PMM) PMM is a group of inherited diseases caused by the deficiency of some enzymes in the process of mitochondrial metabolism At present, the treatment of PMM is mainly nutritional support treatment, symptomatic treatment, a large number of vitamins, coenzyme Q treatment and so on Reenie McCarthy, CEO of steadth, said: "the FDA's award of elamipretide as an orphan drug for PMM is a key milestone PMM is a gradually debilitating disease The symptoms of PMM are muscle weakness and fatigue If there is no appropriate drug treatment, the patients will suffer from the pain all the time We will continue to work with FDA to promote the completion of phase III clinical trials of elamipretide in the treatment of PMM " In June this year, steadth published the results of a phase II clinical trial called mmpower-2 The trial evaluated the safety, tolerability and efficacy of elamipretide in the treatment of PMM Finally, elamipretide reached multiple end points after treatment of PMM patients, supporting elamipretide to carry out phase III clinical trials Currently, steadth is recruiting patients for the power trial to observe and evaluate the condition of PMM patients after treatment with elamipretide Among them, patients with REpower have the opportunity to further participate in the clinical phase III trial This clinical trial will further evaluate the potential efficacy, safety and tolerability of elamipretide in PMM patients.