EMA grants orphan drug designation to spinal and bulbar muscular atrophy drug

The European Medicines Agency has granted orphan drug designation to a small molecule intended to treat spinal and bulbar muscular atrophy, according to the manufacturer.

In a press release announcing the decision, Nido Biosciences said that 54 individuals have been enrolled in a phase 2 clinical trial investigating the single-dose, daily oral therapeutic known as NIDO-361.

The study will be conducted at multiple sites in Europe, the United Kingdom and South Korea. The primary endpoint will be changes from baseline in total and thigh lean muscle volume (measured by MRI), with secondary endpoints including changes from baseline in a disease-specific functional rating scale, 6-minute walk test and grip strength assessment.

The company said the study will also help determine optimal dosing levels of NIDO-361, which is expected to lay a foundation for phase clinical 3 trials.

“There are no treatments currently approved for [spinal and bulbar muscular atrophy] in the E.U. or the U.S., so the orphan drug designation for NIDO-361 marks an important step towards developing a new medicine that can transform the lives of patients suffering from this rare and debilitating neuromuscular disease,” Vissia Viglietta, MD, PhD, chief medical officer of Nido Biosciences, said in the release. “At Nido Bio, we are at a critical juncture with a fully enrolled phase 2 study for this program, and we are grateful for the opportunities that this designation provides as we eye the next stages of its advancement.”