Spinogenix Receives FDA Orphan Drug Status for ALS Drug

The FDA has granted Spinogenix’s SPG302 Orphan Drug designation for treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The agency has approved a phase 1/2 study in ALS patients, followed by a phase 2/3 pivotal trial assuming positive safety, pharmacokinetic and biomarker data, the San Diego, Calif., drugmaker said.

SPG302 is an oral, small-molecule drug that can penetrate the blood-brain barrier and regenerate synapses. It has been shown to improve cognitive and motor behavior in animals with neurodegenerative disorders.