FRANCE – The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Sanofi’s avalglucosidase alfa,a long-term enzyme replacement therapy for the treatment of people with Pompe disease.

CHMP’s positive opinion and recommended indication reflect the robust data from avalglucosidase alfa’s clinical development program.

The positive opinion is based on data from the Phase 3 COMET study, which found that avalglucosidase alfa showed clinically meaningful improvements in respiratory function and movement endurance measures in people with late-onset Pompe disease.

Avalglucosidase alfa is an enzyme replacement therapy designed to target the mannose-6-phosphate (M6P) receptor, the underlying disease mechanism in Pompe disease.

With approximately 15-fold increase in M6P content compared to the current standard of care, avalglucosidase alfa aims to help improve cellular enzyme uptake and enhance glycogen clearance in target tissues.

Avalglucosidase alfa is also currently undergoing review in other countries including the U.S., U.K. and Japan.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy, Fast Track and Priority Review designations to avalglucosidase alfa and the target FDA action date under the Prescription Drug User Fee Act is anticipated to be August 18, 2021.

About Pompe disease

According to National organization for Rare Disorders (NORD), Pompe disease is classified as a rare disease continuum with variable rates of disease progression and different ages of onset.

First symptoms can occur at any age from birth to late adulthood. Earlier onset compared to later onset is usually associated with faster progression and greater disease severity.

At all ages, skeletal muscle weakness characterizes the disease-causing mobility problems and affecting the respiratory system.

The most severely affected infants usually present within the first 3 months after birth. They have characteristic heart problems (dysfunction due to heart enlargement) in addition to generalized skeletal muscle weakness and a life expectancy of less than 2 years, if untreated.

Less severe forms of Pompe disease with onset during childhood, adolescence, or adulthood, rarely manifest cardiac problems, but gradually lead to walking disability and reduced respiratory function.

Pompe disease is inherited in an autosomal recessive pattern. Recessive genetic disorders occur when an individual inherits a non-working gene from each parent.

If an individual receives one working gene and one non-working gene for the disease, the person will be a carrier for the disease.

Pompe disease occurs in various populations and ethnic groups around the world. Estimates vary, but its incidence is generally placed at approximately 1 in 40,000 births in the United States (and in the Netherlands).

However, a recent review of birth incidences in Missouri reported a much higher incidence of 1 in 5,463 in that state.

Sanofi boasts as one of the few pharma giants in the industry that venture into the rare diseases portfolio.

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