First Therapy to Target Muscle Loss in Spinal Muscular Atrophy Gets FDA Approval
The approval gives patients a new muscle-targeted option after prior therapies improved survival but left motor function gains limited, company executives said.
- On Friday, the FDA approved Scholar Rock's Isembyld, the first muscle-targeted treatment for spinal muscular atrophy. Cure SMA president And Kenneth Hobby noted the clearance grants access to a "broad population" of patients with the rare genetic disease.
- The FDA rejected Isembyld last September due to manufacturing issues at a fill-finish facility. Scholar Rock spent the last year implementing an alternative production plan and securing a new facility, which led to Friday's approval.
- On Monday, Scholar Rock set the list price at $11,659 per single-use vial, with typical annual costs around $310,000. The drug targets the 10,000 to 25,000 people in the U.S. impacted by SMA.
- Prescribing information includes a warning about potential bone fractures, which Leerink Partners analyst Basma Radwan called "unexpected." The side effects affected only patients with pre-existing risk factors and did not halt treatment.
- Analysts at Leerink Partners project Isembyld could generate around $2 billion in peak annual sales. Scholar Rock also received a priority review voucher that can accelerate future drug evaluations.
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14 Articles
First Therapy to Target Muscle Loss in Spinal Muscular Atrophy Gets FDA Approval
(MedPage Today) -- The FDA approved apitegromab (Isembyld) injection to treat spinal muscular atrophy (SMA) in adults and pediatric patients ages 2 years and older who currently are receiving an SMN2-targeted treatment, the agency announced Friday...
Scholar Rock prices first drug to target SMA muscle loss at $11,659 per vial - Regional Media News
Sept 14 (Reuters) - Scholar Rock on Monday set a list price of $11,659 per single-use vial for its newly approved spinal muscular atrophy drug, the first therapy that directly targets muscle loss in patients with the rare genetic disorder. The net annual cost for [...]
Scholar Rock prices first drug to target SMA muscle loss at $11,659 per vial
Sept 14 (Reuters) - Scholar Rock on Monday set a list price of $11,659 per single-use vial for its newly approved spinal muscular atrophy drug, the first therapy that directly targets muscle loss in patients with the rare genetic disorder. T...
The Dallas Express News
wbap.comFDA Approves Isembyld, First SMA Treatment To Target Muscle Loss
A newly approved treatment for spinal muscular atrophy is moving toward U.S. availability, giving eligible children and adults an additional option to address muscle weakness. Scholar Rock announced September 11 that the Food and Drug Administration approved Isembyld, also known as apitegromab-mstn. The company said shipments would begin in the coming days as its commercial […]
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