FDA approves Elfabrio as treatment for Fabry disease
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Key takeaways:
- Elfabrio received FDA approval.
- Elfabrio treats Fabry disease.
The FDA has granted approval to Elfabrio, manufactured by Chiesi Global Rare Diseases and Protalix BioTherapeutics Inc. The drug is intended to treat Fabry disease in adults, according to the press release.
“This approval is a testament to the dedication of the Protalix and Chiesi teams to deliver this much-needed new therapeutic option to patients in need. The totality of clinical data suggests that Elfabrio has the potential to be a long-lasting therapy,” Dror Bashan, president and CEO of Protalix said in the release. “Together with Chiesi, we are grateful to all of the patients and investigators and their staff members who participated in our clinical trial programs and remain committed to bringing Elfabrio to patients with Fabry disease.”
Expressed in a plant-cell culture, Elfabrio (pegunigalsidase alfa-iwxj) is a recombinant human alpha-Galactosidase–A enzyme.
This approval follows a comprehensive clinical development program in which researchers examined the safety, tolerability and efficacy of the treatment in more than 140 patients. Another study revealed the drug has noninferior efficacy compared with agalsidase beta when controlling eGFR decline.
According to the release, the treatment has an initial half-life of 78.9 plus or minus 10.3 hours.
“While much progress has been made in the treatment of Fabry disease, there is still a need for new treatment options,” Giacomo Chiesi, head of global rare diseases of the company, said in the release. “We established Chiesi Global Rare Diseases to deliver innovative therapies and solutions for people affected by rare diseases. With the FDA approval of Elfabrio, we can now offer people living with Fabry disease an alternative treatment option.”
Editor’s note: The article was updated on May 23, 2023, to change the URL listed as the source.
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