Scholar Rock SMA Drug Approved by FDA
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 enabled Friday's approval.
- Leerink Partners analyst Basma Radwan called the prescribing warning about bone fractures "unexpected," as fractures had not previously been disclosed. Still, side effects affected only patients with pre-existing risk factors and did not halt treatment.
- On Monday, Scholar Rock set the list price at $11,659 per vial, with typical annual costs around $310,000 for the 10,000 to 25,000 SMA patients in the U.S. impacted by the disease.
- 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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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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