FDA allows expanded access of pegunigalsidase alfa to treat Fabry disease
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Chiesi Global Rare Diseases and Protalix BioTherapeutics Inc. today announced the launch of an expanded access program for a drug that may treat Fabry disease.
As Healio Nephrology previously reported, the FDA accepted a biologics license application and granted priority review to the drug, known as pegunigalsidase alfa (PRX-102), in August 2020.
Designed to slow the progression of end-organ failure, the expanded access program (EAP) will run concurrently with Protalix’s ongoing phase 3 clinical program, according to a press release.
“We are excited that a broader group of physicians and patients beyond those in our phase [3] program will have access to pegunigalsidase alfa, and that such support to Fabry patients in the U.S. is available prior to FDA’s final review,” Raul Chertkoff, MD, vice president and chief medical officer at Protalix, said in the release.
The release noted that patients in the EAP will receive infusions of pegunigalsidase alfa every 2 weeks at 1mg/kg body weight. Information related to adverse events will be collected from patients.
Jerry Walter, founder and president of the National Fabry Disease Foundation, also commented on the program.
“The National Fabry Disease Foundation and the Fabry community are excited about the launch of Chiesi’s expanded access program for pegunigalsidase alfa for the treatment of Fabry disease,” he said. “As the number of people diagnosed with Fabry disease continues to exceed predictions, access to treatment through expanded access programs can play an important role in helping as many eligible patients as possible access the treatment they need.”
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