The first UK patient has been enrolled in Sanofi Genzyme's pivotal Phase III COMET clinical trial of neoGAA to treat Pompe Disease Pipeline Drugs Market and dosed.

NeoGAA is an investigational second-generation alglucosidase alfa enzyme replacement therapy with a greater affinity for muscle cell M6P receptor targeting and enzyme uptake.

It aims to enhance alglucosidase alfa's clinical efficacy while also increasing glycogen clearance.

A genetic defect or malfunction of the lysosomal enzyme acid alpha-glucosidase (GAA) is the cause of Pompe disease, a progressive, debilitating, and frequently fatal neuromuscular condition.

Professor Volker Straub, principal investigator for the Newcastle Hospitals NHS Foundation Trust and Newcastle University John Walton Muscular Dystrophy Research Centre, stated, " A serious, progressive, and crippling condition, Pompe disease

"Doing the first patient in the UK is an exciting and significant step in our endeavor to improve the treatment options available to patients with this disease."
"Doing the first patient in the UK is an exciting and significant step in our endeavor to improve the treatment options available to patients with this disease."

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In order to compare the safety and efficacy of repeated biweekly infusions of neoGAA and alglucosidase alfa in 96 treatment-nave patients with late-onset Pompe disease, the Phase III, multi-centre, multinational, double-blinded COMET study is being conducted.

The primary objective of the study is to ascertain how neoGAA affects respiratory muscle strength as expressed as a percentage of predicted forced vital capacity in the upright position.

The six-minute walk test, muscle strength, motor function, health-related quality of life, and patient-reported outcomes will also be evaluated.