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European Commission approves potential new treatment for Pompe disease

Nexviadyme is the first and only newly approved medicine for Pompe disease in Europe in over 15 years


The European Commission (EC) has issued a marketing authorisation for Sanofi Genzyme’s therapy Nexviadyme (avalglucosidase alfa) for the long-term treatment of both late-onset and infantile-onset Pompe disease.

Pompe disease is a rare, progressive and debilitating muscle disorder which can present as infantile-onset Pompe disease (IOPD), the most severe form of the disease with rapid onset in infancy, or as late-onset Pompe disease (LOPD), which results in progressive muscle damage.

People living with Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA), which results in a build-up of glycogen, leading to irreversible damage to skeletal and cardiac muscles.

Nexviadyme is an enzyme replacement therapy (ERT) and is currently the first and only newly approved medicine for Pompe disease in Europe in over 15 years.

Nexviadyme is specifically designed to target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of ERT and transport to the lysosome, and has an average 15-fold higher level of M6P moieties as compared to alglucosidase alfa. The treatment aims to help improve uptake and enhance glycogen clearance in target tissues as compared to alglucosidase alfa, which was used as the comparator arm in the pivotal phase 3 COMET study.

Results taken from the Mini-COMET study evaluating Nexviadyme in IOPD patients showed improvement or stabilisation at six months in efficacy outcomes, the trial’s secondary objective, of gross motor function measure (GMFM-88), quick motor function test (QMFT), paediatric evaluation of disability index (Pompe-PEDI), left ventricular mass z-score (LVMZ), and eyelid position measurements in patients previously declining or insufficiently controlled with alglucosidase alfa.

A pooled safety analysis from four clinical studies found serious adverse reactions reported in patients treated with Nexviadyme included chills (1.4%), headache, dyspnoea, respiratory distress, nausea, skin discolouration, chest discomfort, pyrexia, blood pressure increased, body temperature increased, heart rate increased and oxygen saturation decreased (0.7% each).

Additionally, hypersensitivity reactions (43.5%), anaphylaxis (1.4%) and infusion-associated reactions (26.1%) were reported. The most frequently reported adverse drug reactions (ADRs) (>5%) were pruritus (9.4%), rash (8%), headache (7.2%), urticaria (6.5%), fatigue (6.5%), nausea (5.8%) and chills (5.1%).

In November 2021, Sanofi announced that as part of the European Medicines Agency’s (EMA) review of Nexviadyme, the Committee for Medicinal Products for Human Use (CHMP) issued an opinion that the therapy does not qualify as a New Active Substance (NAS). The Committee for Orphan Medicinal Product (COMP) also recommended Nexviadyme be removed from the Community Register of Orphan Medicinal Products (OMP) in April 2022.

In a press release, Sanofi said: “[The company] stands by the totality of data in support of Nexviadyme as a potential new standard of care and is concerned that such narrow interpretation will undermine rare disease incentive mechanisms in Europe. We believe withholding these distinct designations could negatively impact patient health in Europe by restricting access to innovative advancements in care."

Article by
Fleur Jeffries

28th June 2022

From: Research, Regulatory



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