Sept 14 (Reuters) – Scholar Rock on Monday said the net annual cost of its newly approved spinal muscular atrophy treatment will be about $310,000 for a typical patient, after the drug became the first therapy approved to directly target muscle loss associated with the rare genetic disorder.
The cost will depend on a patient’s weight and insurance coverage, the company’s executives said, with a typical patient in the 35 kg to 45 kg range expected to receive approximately three vials every four weeks.
The therapy, branded as Isembyld, won approval for use in all adults and children over 2 years of age with spinal muscular atrophy who are currently receiving therapies that target SMN2, a gene for neurons that control movement.
Scholar Rock’s shares were about 5% higher before the bell on Monday.
The wholesale acquisition cost for a single-use vial is $11,659. The company has commenced the launch of Isembyld immediately upon Friday’s approval.
“In terms of the launch, we expect a steady and consistent pace of Isembyld patient starts as we work to serve a gradually increasing number of children and adults living with SMA,” Chief Operating Officer Keith Woods said.
The genetic disease results in irreversible loss of motor neurons and progressive muscle wasting, leading to motor function decline.
In a late-stage trial, the drug, when combined with an SMN2-targeting drug, helped patients’ motor skills to significantly improve compared to placebo.
Isembyld selectively blocks activation of myostatin, a protein that limits muscle growth, with the aim of increasing muscle mass and strength.
(Reporting by Sriparna Roy in Bengaluru; Editing by Maju Samuel)
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