SAT-3247 for DMD wins FDA rare pediatric disease designation
Satellos oral therapy aims to improve muscle repair, slow progression
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The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition.
The FDA awards this status to investigational medicines that are designed to treat serious, rare diseases that mainly affect children. The designation provides certain perks to SAT-3247’s developer Satellos Bioscience: Most notably, if the therapy ultimately is approved, then Satellos may be eligible to receive a voucher that it can use to have the FDA speed up its review of applications for another medicine. The voucher may also be sold to another company.
The new designation comes just a few months after the FDA granted SAT-3247 orphan drug status, which is given to experimental treatments designed for rare disorders (irrespective of what age group they affect). Among the benefits of orphan drug designation is a guarantee that, if SAT-3247 is approved, Satellos would get seven years of market exclusivity that would allow the company to sell the therapy without any competition.
Both rare pediatric disease and orphan drug designations are designed to provide economic incentives for companies that are investing in the development of treatments for rare diseases, which by definition have a small market that can make it difficult to recoup investment losses and turn a profit on the therapy’s development.
“Obtaining the rare pediatric disease and orphan drug designations for SAT-3247 from the FDA are important milestones for Satellos as we continue to progressively build value in our DMD program,” Frank Gleeson, CEO and co-founder of Satellos, said in a company press release.
Rare pediatric disease designation follows award of orphan drug status
According to Gleeson, the FDA’s decision to grant the therapy rare pediatric disease designation “highlights the continued need for new disease-modifying therapeutic options for pediatric patients with Duchenne, a need which we believe SAT-3247 has the potential to address.”
[The award of FDA rare pediatric disease designation] highlights the continued need for new disease-modifying therapeutic options for pediatric patients with Duchenne, a need which we believe SAT-3247 has the potential to address.
SAT-3247 is an oral small molecule that’s designed to boost the regenerative activity of muscle stem cells. Normally when muscles are damaged, stem cells can kick into gear to help fix the damage. However, evidence suggests that this process is defective in DMD, contributing to the progressive muscle damage that characterizes the disease. By activating muscle stem cells, SAT-3247 aims to improve muscle repair to slow disease progression.
Satellos now is working to launch a Phase 1 trial to test the safety of SAT-3247 in healthy volunteers. The company said the trial is expected to start in the next month or two.
SAT-3247 is Satellos’ lead treatment candidate for DMD. The therapy was awarded orphan drug status by the FDA earlier this year, according to the company.
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