Reena Sharma, MD, Adult Metabolic Consultant at Salford Royal Hospital and University of Manchester, discusses plans for a phase 3 clinical trial evaluating FLT201 in patients with Gaucher disease.
Gaucher disease refers to a rare group of inherited metabolic diseases in which harmful amounts of the lipid glucocerebroside accumulates in various cells and organs. Signs and symptoms vary widely among affected individuals and may include skeletal disorders, hepatosplenomegaly, liver malfunction, anemia, low platelet counts, bone problems, and neurological problems. There are different types of Gaucher disease classified according to specific features and severity. Gaucher disease type 1 is the most common form. It is caused by genetic changes in the GBA gene.
FLT201 is an investigational adeno-associated virus (AAV) gene therapy in clinical trials for a potential one-time treatment for Gaucher disease type 1. The treatment uses AAVS3 capsid to deliver GCase85, a longer-acting version of the deficient enzyme causing this disease.
The completed phase 1/2 GALILEO-1 clinical trial showed improvements in key biomarkers and clinical assessments such as reductions in the toxic buildup of substrate and a favorable safety and efficacy profile. Additionally, durable reductions were observed in glucosylsphingosine (lyso-Gb1), maintenance of normal hemoglobin levels, and sustained improvements or maintenance of platelet counts, spleen, and liver volume.
A phase 3 trial is supported by positive feedback from the U.S. Food and Drug Administration (FDA) in the end-of-phase 2 meeting with Spur Therapeutics. Based on that meeting, the clinical trial design of the phase 3 study may allow FLT201 the opportunity for accelerated approval based on the reductions in lyso-Gb1 with full approval coming later if the primary endpoint of the study is met (maintenance or improvement in hemoglobin at one year). The pivotal phase 3 study is expected to start in the second half of 2025.
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To learn about Gaucher disease and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
