Sanofi presents positive results for Pompe disease enzyme replacement therapy

2024-02-14

临床结果上市批准临床3期

Sanofi presents positive results for Pompe disease enzyme replacement therapy

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来源: PMLiVE

Sanofi has shared positive results for its enzyme replacement therapy, avalglucosidase alfa, across a wide range of Pompe disease patient groups.

Affecting approximately one in every 40,000 people in the UK, Pompe disease is a rare genetic disorder caused by low levels of the enzyme acid alpha-glucosidase. This results in the build-up of glycogen in muscle cells throughout the body, leading to potentially irreversible damage to the heart and skeletal muscles.

The disorder can present as either infantile-onset Pompe disease (IOPD), the most severe form of the disease with rapid onset in infancy, or late-onset Pompe disease (LOPD), which progressively damages the muscles over time.

If left untreated, IOPD can lead to heart complications and death within the first year of life, while patients with LOPD may require ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses.

According to data from the mid-stage Mini-COMET study long-term extension, presented at this year’s WORLDSymposium, avalglucosidase alfa was associated with meaningful improvements in ptosis, or drooping eyelid, in paediatric patients with IOPD over nearly three years.

Ptosis impacts approximately 50% of IOPD patients and can cause vision loss and decreased quality of life, Sanofi outlined.

Positive safety results from the phase 3 Baby-COMET trial, which has been evaluating avalglucosidase alfa in treatment-naïve IOPD patients, were also presented at the WORLDSymposium, as well as key findings surrounding the therapy’s use in LOPD.

This included eight-year follow-up results from the phase 2/3 NEO-EXT study of avalglucosidase alfa in patients with LOPD and real-world findings from the international, voluntary Pompe Registry sponsored by Sanofi of those who switched to avalglucosidase alfa from prior treatment with a long-time standard of care, alglucosidase alfa.

Alaa Hamed, global head of medical affairs, rare diseases, Sanofi, said: “The totality of data presented at WORLDSymposium builds upon existing evidence that supports the value of avalglucosidase alfa in treating Pompe disease, showcasing the therapy’s long-term durability and favourable efficacy and safety profile across a wide range of patient types and clinical circumstances…”

Avalglucosidase alfa holds approvals to treat Pompe disease, with specific indications varying by country. In Europe, the therapy is marketed under the brand name Nexviadyme and approved for use in both LOPD and IOPD patients, while in the US, it is indicated for the treatment of LOPD in patients one year and older under the brand name Nexviazyme.

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