Finnish pharmaceutical company Orion has recruited the first patients in a phase 3 clinical trial, dubbed REFALS, to evaluate orally administered levosimendan (ODM-109) for the treatment of symptoms of amyotrophic lateral sclerosis (ALS).The purpose of the trial is to demonstrate that orally administered levosimendan, by enhancing respiratory muscle function, can help maintain breathing capacity and so benefit overall functioning of patients with ALS.
ALS is a rapidly progressing, fatal neurological disease that causes the death of motor neurons, leading to the weakening of voluntary muscles and eventually paralysis. There is no cure for ALS, and death commonly occurs within 3-5 years of diagnosis, typically due to respiratory failure.Levosimendan does not cure ALS, but it is expected to maintain the patient’s breathing capacity for longer and thus improve the quality of life by delaying the need for ventilation support.
A total of 450 patients will participate in the placebo-controlled trial at around 100 clinical sites in Europe, North America and Australia. The patients will be treated for around one year after the first administration of the drug.Orion is investing around EUR 60 million in the study over approximately three years.If the results of the trial are positive, the aim is to file for marketing authorisation in the United States and Europe. Orally administered levosimendan has been granted an Orphan Drug Designation both in the United States and in the European Union.
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