New Fabry Disease Treatment Enters Lucrative Market


A wholesale acquisition cost (WAC) for Elfabrio will be announced once it is available in a few weeks.

FDA Approved

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FDA’s approval of Chiesi Global Rare Diseases and Protalix Bio Therapeutics’ Elfabrio (pegunigalsidase alfa-iwxj) for Fabry disease signals another enzyme replacement therapy (ERT) in the lucrative market for treating the disease.

"While much progress has been made in the treatment of Fabry disease, there is still a need for new treatment options," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "With the FDA approval of Elfabrio, we can now offer people living with Fabry disease an alternative treatment option.”

Chiesi told Formulary Watch that the company will provide a wholesale acquisition cost (WAC) for Elfabrio when it is available in a few weeks.

“As a family-run business, we are committed to ensuring access to Elfabrio to as many people living with Fabry disease as possible. It is important to deliver this new treatment option to reduce the burden of this chronic disease on patients, their families, and the healthcare system,” he said.

U.S. payers have been “very receptive” to having another ERT option for healthcare professionals and patients, Chiesi noted. As such, “we don’t anticipate any coverage hurdles with payers at this time.”

The new treatment joins Sanofi’s Fabrazyme (agilsadase beta) and Amicus Therapeutics’ Galafold (migalastat) in a lucrative market expected to be valued at more than $3.5 billion globally in 2023.

Drivers for growth include rising cases of the rare disease across the globe and advancements in Fabry disease therapies, according to Global Market Insights. At the same time, the high treatment costs of ERTs at $200,000 or more annually may restrain future development in the market.

Elfabrio is, a PEGylated ERT, is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.

The safety, tolerability, and efficacy of Elfabrio has been studied in a comprehensive clinical development program in more than 140 patients with up to 7.5 years of follow up treatment, Chiesi and Protalix said.

It has been studied in both ERT-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint with Ealfabrio demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline. Additionally, Elfabrio was generally well-tolerated with the majority of adverse events being mild or moderate in severity.

“The totality of clinical data suggests that Elfabrio has the potential to be a long-lasting therapy.” said Dror Bashan, president and CEO of Protalix.

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