FDA Greenlights Orchard’s Rare Disease Gene Therapy, Following EU’s Stamp of Approval

Hope on the Horizon: Orchard Therapeutics’ Breakthrough Gene Therapy for Rare Disease Gains FDA Priority Review

In a game-changing move, the FDA has given the green light to Orchard Therapeutics’ Biologics License Application (BLA) for OTL-200, a groundbreaking gene therapy designed to combat the rare and devastating metachromatic leukodystrophy (MLD). This development comes on the heels of OTL-200’s European approval in 2020, marking a significant milestone for patients battling this rare disease.

The FDA’s stamp of approval doesn’t stop here—it’s a Priority Review designation, which means the regulatory review timeline has been trimmed down to a mere six months, rather than the usual 10. The verdict? Expected on March 18, 2024.

Orchard’s CEO, Bobby Gaspar, hailed this BLA acceptance as a monumental triumph for MLD patients and their families. For far too long, they’ve endured the heart-wrenching journey of navigating the diagnostic labyrinth, hearing grim prospects of no treatment beyond supportive care, and witnessing their loved ones slip away.

While the FDA’s review is in full swing, Orchard is wasting no time. They’re working in tandem to potentially launch OTL-200 in 2024, ensuring that this life-changing treatment reaches patients at the earliest possible moment.

MLD, affecting roughly one in every 100,000 live births, is a rare and hereditary disorder that wreaks havoc on the metabolic system. It’s triggered by a faulty mutation in the arylsulfatase-A (ARSA) gene, responsible for breaking down sulfatides—a type of fatty substance.

This genetic glitch leads to the accumulation of sulfatides in vital organs like the brain, kidneys, and spleen, resulting in a cascade of motor, cognitive, and behavioral issues, along with seizures and spasticity. As the disease mercilessly progresses, MLD patients lose their ability to move, speak, eat, and see, ultimately succumbing to its devastating grip.

OTL-200, also known as atidarsagene autotemcel, goes straight to the root of the problem. It delivers functional copies of the ARSA gene using genetically modified hematopoietic stem cells—a game-changing approach.

Orchard has presented compelling data from two prospective non-randomized studies, featuring 39 children with early-onset MLD. The results are nothing short of remarkable, showing that this investigational gene therapy has the potential to preserve motor function and cognitive development in these young patients.

With more than 250 cumulative patient-years of follow-up, OTL-200 has demonstrated exceptional tolerance, with no treatment-related serious adverse events or fatalities reported.

This groundbreaking mechanism, coupled with an impressive clinical profile, has already earned OTL-200 regulatory approval in the European Union, where it goes by the name Libmeldy, and has been embraced by at least six countries. The FDA has also granted the therapy its coveted Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations, underscoring its potential to transform the lives of those affected by MLD.

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