EC decision due in May on enzyme replacement therapy PRX-102
FDA is also expected to decide in May about whether to approve treatment
The European Commission (EC) is expected to decide whether to approve PRX-102 (pegunigalsidase alfa) for adults with Fabry disease in early May.
The therapy’s co-developers, Protalix BioTherapeutics and Chiesi Global Rare Diseases announced that PRX-102 received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).
CHMP is part of the European Medicines Agency (EMA). The EC is not bound to abide by CHMP’s recommendation, but historically most experimental therapies favored by the committee have been approved by the commission.
“We are pleased to be another step closer to approval in Europe with the CHMP’s positive opinion recommending marketing authorization for PRX–102 for adult patients with Fabry Disease,” Dror Bashan, president and CEO of Protalix, said in a company press release. “We believe that this recommendation further recognizes the strength of the positive dataset from our robust clinical trial program and underscores the potential for PRX-102 to provide a new treatment option for patients with Fabry disease.”
PRX-102 is also being reviewed for approval by the U.S. Food and Drug Administration. A decision is expected May 9.
“We look forward to advancing towards approval and launch in Europe and will continue our mission to deliver this potential new treatment option to people living with Fabry disease around the world,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases.
How PRX-102 compares to other enzyme replacement therapies
In Fabry disease, genetic mutations disrupt the activity of the enzyme alpha-galactosidase A (Gal A), which breaks down certain fatty molecules. Without the enzyme, fatty molecules build up to toxic levels in body tissues, leading to the typical symptoms of Fabry.
PRX-102 is an enzyme replacement therapy that would deliver a functional version of Gal A to cells. Two other enzyme replacement therapies are available in much of the European Union — Fabrazyme (agalsidase beta), sold by Sanofi Genzyme, and Replagal (agalsidase alpha) by Takeda.
The enzyme in PRX-102 is made using plant cells. It’s chemically modified to last longer in the body to allow for less frequent dosing than current therapies that are given every two weeks.
Protalix and Chiesi applied for PRX-102’s approval in Europe last year. The application was supported by findings from several clinical trials, including the Phase 3 studies BALANCE (NCT02795676), BRIDGE (NCT03018730), and BRIGHT (NCT03180840).
The BALANCE study compared PRX-102 against Fabrazyme, each given every two weeks, in 77 adults with Fabry. Results indicated the two therapies had a similar safety profile and were comparably able to slow kidney function decline.
In the BRIDGE and BRIGHT studies, patients previously on Fabrazyme or Replagal switched to PRX-102, given every two weeks in BRIDGE, or every four weeks in BRIGHT. The results suggested PRX-102 was effective at maintaining kidney function and was generally well tolerated.
“Data from our clinical program indicates that PRX–102 has the potential to be a long lasting therapy with a favorable tolerability and immunogenicity profile,” Bashan said.
“Our deepest gratitude to all the individuals with Fabry disease who have participated in clinical trials. Thanks to them, PRX–102 has been extensively studied during the clinical development program, providing the data for the CHMP’s evaluation and positive opinion regarding a positive benefit-risk profile for PRX–102,” Chiesi said.