SQY51 had already obtained Orphan Drug Designation from the US Food and Drug Administration (FDA) in Duchenne Muscular Dystrophy in May 2024.
SQY51 is a palmitoyl-conjugated tc-DNA antisense oligonucleotide designed to restore semi-functional dystrophin by skipping exon-51 during mRNA splicing of the DMD gene in a subpopulation of boys affected by Duchenne Muscular Dystrophy.
Read More: Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
“We’re pleased to receive Orphan Drug Designation from the EMA, following on from the designation we have already received from the FDA,” said Luis Garcia, Special Scientific Advisor of SQY Therapeutics. “These designations reflect the significant unmet need in Duchenne Muscular Dystrophy and reinforce our belief in the potential of SQY51 to make a meaningful difference for patients and families affected by this devastating disease and concerned by the exon-51 skipping. » SQY51: A Promising Drug for Duchenne Muscular Dystrophy
SQY51 is being evaluated in the Phase 1/2a AVANCE-1 clinical trial in individuals with DMD who are amenable to exon-51 skipping. The company has completed enrollment of 12 patients in the Phase 1/2a, with data expected in 2026. Phase 1 is completed and Phase 2a is underway. SQY Therapeutics is also planning an extension phase for participants who would like to continue to benefit from the treatment.
Learn More: Next Generation Exon Skipping Therapies Developed for the Treatment of Duchenne Muscular Dystrophy
