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news.Scholar Rock has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA) for muscle-targeted therapy, apitegromab, aimed at enhancing motor function in individuals with spinal muscular atrophy (SMA) who are already on SMN-targeted therapies.
The therapy is aimed at enhancing motor function in individuals with spinal muscular atrophy. Credit: CHUTTERSNAP on Unsplash.
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Additionally, the company is on track to submit a marketing authorisation application (MAA) to the European Medicines Agency (EMA) in the first quarter of this year.
The Division of Neurology Products will review the submission at the agency’s Center for Drug Evaluation and Research.
The data from the Phase II TOPAZ trial and Phase III SAPPHIRE trial backed the FDA submission.
Last year, the company revealed positive topline outcomes from the SAPPHIRE trial.
A priority review was also requested by the company, and if the request is granted, it would decrease the review timeline of the FDA to six months from the date of accepting the filing.
The therapy has already received FDA’s orphan drug, rare paediatric disease, and fast track designations.
It gained priority medicines (PRIME) and orphan medicinal product designations from the European Medicines Agency (EMA) for SMA treatment.
Plans are underway to investigate the therapy in the Phase II trial, OPAL, for SMA patients who are below the age of two, with the commencement of the trial anticipated in the middle of this year.
An investigational fully human monoclonal antibody, apitegromab works by blocking myostatin activation, done by selectively binding to both the pro- and latent myostatin forms in skeletal muscle.
Scholar Rock chief medical officer Jing Marantz said: “We are gratified that in patients already on a SMN-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.”
