Sanofi’s enzyme replacement therapy now funded in rare neuromuscular disorder

Pharma News: Pompe disease is an autosomal recessive disorder caused by acid alpha glucosidase (GAA) enzyme deficiency. A deficiency leads to skeletal muscle destruction and progressive myopathy causing early death from respiratory or heart failure.

Sanofi’s Nexviazyme (avalglucosidase alfa), an IV monotherapy that was TGA registered in November 2021, is now funded through the Life Saving Drugs Program (LSDP) for Pompe disease.

Neurologist Associate Professor Robert Henderson from the Royal Brisbane and Women’s Hospital said the addition of Nexviazyme to Australia’s Life Saving Drugs Program was “a significant step forward for the Pompe community and represents the first new treatment for the condition in seven years”.

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“Clinicians treating Pompe disease are in a race against time to save muscle function so new treatments are urgently needed. The opportunity to deliver this therapy for the first time for some patients through home infusion services will also make a significant difference to people who have work, school or family commitments, or those who had to travel extended distances to hospital for treatment,” said A/Professor Henderson.

At the November 2021 meeting, the Pharmaceutical Benefits Advisory Committee (PBAC) considered Sanofi’s submission for listing of Nexviazyme on the Pharmaceutical Benefits Scheme (PBS). The PBAC did not recommend the requested Section 100 (Highly Specialised Drugs Program) listing of Nexviazyme for the treatment of IOPD (infantile-onset Pompe disease), JOPD (juvenile-onset) or AOPD (adult late-onset) as it considered that Nexviazyme was not cost-effective at the proposed price and the extent of benefit to AOPD provided in the submission was uncertain.

In order to be included in the LSDP, a medicine must be considered to meet each of the eight LSDP funding criteria, which Nexviazyme did. The LSDP panel also found that the consumer input was informative and contributed to its deliberations.

Nexviazyme’s phase 3 COMET trial compared the drug’s efficacy to that of its enzyme-replacement predecessor alglucosidase alfa. At 49-weeks, Nexviazyme patients scored 2.4 points higher on a standard lung function test used to measure respiratory muscle weakness than those on alglucosidase alfa. Patients on Nexviazyme were able to walk 30 meters farther than those in the alglucosidase alfa cohort at 49 weeks, according to a six-minute walk test used to measure functional endurance.

Sanofi ANZ Head of Medical, Specialty Care, Dr Kasia Siwek, said that the addition of Nexviazyme to the Life Saving Drugs Program is an “important moment for the rare disease community”.

“We are proud of our long-standing commitment to the rare disease community in Australia, and that we now supply six of the sixteen medicines on the Life Saving Drugs Program – a vital program that ensures those affected by rare diseases do not miss out on treatment, simply because their condition is rare,” Dr Siwek said.

In line with LSDP policy and to manage uncertainties, a review of Nexviazyme’s clinical data at 24 months after listing will be conducted to ensure use and performance of the medicine in Australia is in line with the expectations at the time of listing.

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