Spinal muscular atrophy (SMA) is a rare disease causing progressive muscular weakness. Severe forms of the disease can be fatal to infants. Apitegromab is an experimental therapy that was successful in a Phase III trial in SMA, paving the way for the company to anticipate approvals in Europe and the United States in early 2025.
According to Scholar Rock, developing company for apitegromab, the drug, together with the standard therapy for SMA, demonstrated a significant improvement in motor function in one year as opposed to the typical SMA treatment plus placebo. Secondary factors involve research into apitegromab’s ability to help people with obesity preserve muscle. Positive Phase 3 findings, according to the company’s CEO, may position the therapy to be a part of the “new standard” for SMA.
A pooled analysis found that children ages 2-12 who received one of the two doses being tested showed a difference of 1.8 from baseline. The researchers used a scale that evaluates physical abilities.
A smaller group of teens ages 13-21 observed motor function benefits with no newly-found safety incidents reported. In addition, no one left the trial as a result of side effects.
CEO Jay Backstrom said that the data recently collected demonstrates that apitegromab has earned a place in the new standard of care.
