Overview
- Scholar Rock announced on Sept. 11 that the FDA approved Isembyld (apitegromab-mstn) for people with SMA aged 2 and older who are already receiving an SMN2-targeted therapy.
- The FDA based its decision on a 52-week trial of 188 participants that found 34.2% of children ages 2–12 on Isembyld achieved clinically meaningful motor improvement versus 13.5% on placebo.
- Isembyld works by blocking activation of myostatin, is given as a 10 mg/kg IV infusion once every four weeks, and can be administered in hospitals, infusion centers, or at home.
- The label includes safety concerns such as an increased risk of fractures and common side effects like respiratory infections, vomiting, cough, and headache, plus warnings about potential fetal harm and effects on reproduction.
- Scholar Rock is starting commercial shipments and a patient support program to help with insurance, financial aid, and infusion arrangements, and advocacy groups are offering education and access planning for families and clinicians.