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Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients

The rare genetic disorder affects around one in every 40,000 people in the UK


Sanofi has announced that its long-term enzyme replacement therapy will now be available on the NHS to patients with Pompe disease, following a recommendation from the National Institute for Health and Care Excellence (NICE) in August last year.

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, resulting in irreversible damage to the heart and skeletal muscles.

The disorder can present as both infantile-onset Pompe disease (IOPD), the most severe form of the disease with rapid onset in infancy, or as late-onset Pompe disease (LOPD).

If left untreated IOPD can lead to heart complications and death, with an average life expectancy of 14 months, while patients with LOPD may require ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses.

Sanofi's own alglucosidase alfa – sold as Myozyme in Europe and Lumizyme in the US – is the current standard of enzyme replacement therapy for patients with Pompe disease, which has been shown to extend overall survival and slow disease progression in Pompe patients.

However, there is a recognised need for treatment options that can offer a greater benefit and longer duration of response.

The company’s latest enzyme replacement therapy, Nexviadyme (avalglucosidase alfa), showed a 2.89% improvement from baseline, compared to 0.46% with Myozyme, in forced vital capacity percent-predicted, a key measure of respiratory function and the phase 3 COMET study’s primary endpoint.

Additionally, patients treated with Nexviadyme walked 32.2 metres further compared to baseline in the six-minute walk test, a key secondary endpoint, which was 30 metres further than the change with Myozyme.

“The availability of [Nexviadyme] in the UK marks another critical milestone in Sanofi’s 20-year commitment to the Pompe community,” said Rippon Ubhi, general manager UK and Ireland, Sanofi Specialty Care. “We are pleased to see this new treatment now be made available to people with Pompe disease.”

As well as its UK approval last year, which authorised the use of Nexviadyme across the full spectrum of Pompe disease, the therapy was also approved in the US for patients with LOPD aged one year and above.

Article by
Emily Kimber

8th February 2023

From: Regulatory, Healthcare



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