FDA approves Isembyld in the US to treat muscle loss in SMA
The US Food and Drug Administration (FDA) has approved Scholar Rock's Isembyld, the first therapy targeting muscle loss in spinal muscular atrophy (SMA). The drug has been authorized for use in adults and children aged two and older who are already receiving SMA therapy targeting the SMN2 gene, STAT News reports.
Study results
In a late-stage clinical trial, Isembyld was used together with a drug targeting SMN2. This gene is crucial for neurons that control movement.
After one year, young patients receiving the combination treatment showed improved motor skills. Participants in the placebo group, by contrast, showed worsening in these measures. The difference between the groups was statistically significant.
More current news is available on the UA.News Telegram channel Telegram.
Company statement
Scholar Rock CEO David Hallal called the FDA decision a defining moment for the spinal muscular atrophy community in a press release. According to him, after decades of unsuccessful industry efforts to unlock the potential of myostatin inhibition, the company has achieved a therapeutic breakthrough.
As STAT News notes, the approval may give patients with this rare neurological disease hope for better opportunities to move independently and walk.
