PRX-102’s Approval Application Resubmitted to FDA
The experimental enzyme replacement therapy targets adult Fabry disease patients
An application seeking approval for PRX-102 (pegunigalsidase alfa), an experimental enzyme replacement therapy for adults with Fabry disease, has been resubmitted to the U.S. Food and Drug Administration (FDA).
Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases expect the review of their request, sent in the form of a Biologics License Application (BLA), to be completed within six months.
“The resubmission of the BLA represents a significant milestone for Protalix and we believe it has meaningful potential for patients and families affected by Fabry disease,” Dror Bashan, Protalix’s president and CEO, said in a press release.
“Together with Chiesi,” he said, “we are committed to continuing to work with the FDA toward our goal of achieving regulatory approval and making PRX-102 available to patients with this rare disease in the United States.”
The FDA rejected a previous application citing issues with the manufacturing process and facility inspections, but nothing related to the therapy’s safety or effectiveness. The companies also have applied for PRX-102’s approval in Europe.
BLA resubmission included a comprehensive package of manufacturing and clinical data assembled from studies that tested the therapy in more than 140 adults with Fabry with up to five years of follow-up.
The clinical data set was compiled from three now-complete Phase 3 studies: BALANCE (NCT02795676), BRIDGE (NCT03018730), and BRIGHT (NCT03180840), as well as a Phase 1/2 trial (NCT01678898) with data from the related extension study. Safety data also were included from the ongoing extension studies associated with the Phase 3 clinical trials.
Topline data from the BALANCE study reported earlier this year demonstrated two years of PRX-102 prevented kidney decline and showed safety and efficacy similar to the approved enzyme replacement therapy Fabrazyme by Sanofi Genzyme.
“Together with Protalix, we thank the investigators and study participants who have made reaching this milestone possible and have supported our joint commitment to bringing this new treatment option to the Fabry patient community,” said Giacomo Chiesi, head of Chiesi.
Fabry disease is marked by a lack of alpha-galactosidase A (Gal A), an enzyme that breaks down a fatty molecule called globotriaosylceramide (Gb3). Gal A deficiency results in the toxic buildup of Gb3, causing damage to the kidneys, heart, and blood vessels in the brain (cerebrovascular).
“Fabry disease is a serious, life-threatening, rare genetic disorder, the ultimate consequences of which range from episodes of pain and impaired peripheral sensation to end-organ failure, particularly of the kidneys, but also of the heart and the cerebrovascular system,” Bashan said.
ProCellEx platform
PRX-102 is a lab-made version of Gal A, delivered directly into patients’ bloodstream, developed using Protalix’s proprietary ProCellEx technology platform, which uses plant cells to generate the therapeutic enzyme. In PRX-102, however, Gal A has been modified to last longer, meaning fewer infusions compared to other enzyme replacement therapies.
“Many people who are living with Fabry disease still have existing unmet needs, including access to treatment and the burden of regular infusions, and we believe it is important to deliver a potential new treatment option,” Â Chiesi added.
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