Protalix BioTherapeutics and Chiesi Farmaceutici announced the start of a pediatric study plan (iPSP) to test PRX-102 (pegunigalsidase alfa) in children with Fabry disease, in agreement with the U.S. Food and Drug Administration (FDA).
Fabry is characterized by the lack of an enzyme called alpha-galactosidase A, which prevents the body from breaking down a type of fat molecule called globotriaosylceramide (Gb3 or GL-3). This results in damage to cells and several tissues.
PRX-102 is an investigational enzyme replacing therapy (ERT) that aims to compensate for the missing alpha-galactosidase A enzyme in Fabry patients. It was developed through Protalix’s ProCellEx platform that uses plant cells to produce therapeutic proteins, rather than the more common mammalian cells that can pose safety issues.
PRX-102 has been tested in multiple clinical trials with adults with Fabry disease (Phase 1/2 NCT01678898 and NCT01769001; Phase 3 NCT03018730 and NCT02795676). However, initial symptoms often appear in childhood, and there is a need for better treatments in this patient population.
Regulation around testing new therapies in children is tightly controlled. Prior to pediatric tests, companies are required to submit an iPSP that includes an outline of the planned study or studies. The FDA then decides whether it agrees or disagrees with what is planned.
“Proceeding with the pediatric study plan for PRX-102 marks an important milestone in our goal of bringing an alternative to the Fabry patient community which is in need of better treatment options other than those currently available,” Dror Bashan, Protalix BioTherapeutics’ president and chief executive officer, said in a press release.
“Together with our partner Chiesi, we value this pediatric study as a new opportunity to potentially bring a new treatment option to the entire Fabry community,” added Raul Chertkoff, MD, Protalix BioTherapeutics’ vice president, medical affairs.
Protalix and Chiesi earlier announced an intent to submit a Biologics License Application (BLA) to the FDA, asking that its treatment be approved in the U.S., at least for adults, under the accelerated approval pathway.
The FDA has agreed that enough data to support the BLA exist in previous nonclinical, clinical, safety, and manufacturing studies. The application is expected to be filed by the end of April.
The FDA has granted fast track designation to PRX-102 as a potential treatment for Fabry disease, which works to speed its clinical development and agency review.