FDA Grants Rare Pediatric Disease Designation to SAT-3247 to Treat Duchenne Muscular Dystrophy

The FDA has recently granted rare pediatric disease designation to SAT-3247 (Satellos Bioscience Inc) for the treatment of duchenne muscular dystrophy (DMD) among children aged 18 years and younger. The designation follows a previous orphan drug designation that was granted earlier this year.¹

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“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 news release. “The rare pediatric disease designation for SAT-3247 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.”¹

DMD is one of the most severe forms of genetic muscular dystrophies, occurring in all race or ethnic groups, according to study authors. DMD occurs as mutations in the dystrophin gene cause ongoing muscle fiber degeneration and weakness. This results in a decrease of daily activities and wheelchair use among individuals impacted. However, the study authors noted more severe difficulties including cardiac and orthopedic complications commonly occur. Individuals diagnosed with DMD are associated with a poor prognosis, as individuals are often in need of a wheelchair by 12 years old and death could occur in their adolescent years or early 20s. Study authors noted that there is no cure for DMD, and available treatment options included glucocorticoid therapy, prevention of contractures, and medical care of cardiomyopathy and respiratory compromise.²

Researchers from Satellos created SAT-3247 to repair muscles and regeneration by regulating a dystrophin-independent pathway to increase muscle function. The drug is an oral, small molecule inhibitor of adapter associated kinase 1 that could target the main cause of muscles weakness caused by DMD, according to study authors.^1,3

In July of this year, Satellos announced a submission of regulatory filing to initiate the phase 1 clinical trial to assess the use of SAT-3247, under their Therapeutic Goods Administration’s Clinical Trial Notification scheme.⁴

"We are pleased with the results from our preclinical studies and look forward to initiating clinical development of SAT-3247,” Phil Lambert, PhD, chief scientific officer of Satellos, said in a news release. “Prior to submitting our regulatory documentation, we conducted our preclinical and toxicology studies to the standards of relevant global regulatory bodies. Thus, we expect to be able to leverage these results for additional Phase 1 and subsequent clinical trials in Australia and further jurisdictions including the United States and Canada, where we plan to advance into trials with DMD patients."⁴

The study authors noted that the commencement of the phase 1 clinical trial of SAT-3247 is expected in Q3 of 2024.^1,4

References

1. Satellos Receives Rare Pediatric Disease Designation from the U.S. FDA for SAT-3247 for the Treatment of Duchenne Muscular Dystrophy. Satellos. News release. August 8, 2024. Accessed August 9, 2024. https://www.businesswire.com/news/home/20240808320005/en/Satellos-Receives-Rare-Pediatric-Disease-Designation-from-the-U.S.-FDA-for-SAT-3247-for-the-Treatment-of-Duchenne-Muscular-Dystrophy

2. Duchenne Muscular Dystrophy. National Library of Medicine. News release. July 10, 2023. Accessed August 9, 2024. https://www.ncbi.nlm.nih.gov/books/NBK482346/#:~:text=Duchenne%20muscular%20dystrophy%20(DMD)%20is%20one%20of%20the%20most%20severe,muscle%20fiber%20degeneration%20and%20weakness.

3. Lambert P. LB: SAT-3247: An Oral Small Molecule Inhibitor Targeting AAK1, a Critical Effector Of Skeletal Muscle Regeneration. MDA Clinical & Scientific Conferece. March 2024. Accessed August 9, 2024. https://www.mdaconference.org/abstract-library/sat-3247-an-oral-small-molecule-inhibitor-targeting-aak1-a-critical-effector-of-skeletal-muscle-regeneration/

4. Satellos Announces Submission of Regulatory Filing to Commence a Phase 1 Clinical Trial with SAT-3247. Satellos. News release. July 11, 2024. Accessed August 9, 2024. https://ir.satellos.com/news/news-details/2024/Satellos-Announces-Submission-of-Regulatory-Filing-to-Commence-a-Phase-1-Clinical-Trial-with-SAT-3247/default.aspx