Protalix and Chiesi Set to Seek Approval in Fabry Disease
Fabry disease is caused due to buildup of globotriaosylceramide in the cells of body.
Shares of Protalix Biotherapeutics skyrocketed Monday morning after the company announced pegunigalsidase alfa hit the mark in improving kidney function in Fabry disease patients.
Shares of the company are up more than 65% based on the data from the plant-based PEGylated enzyme replacement therapy it co-developed with Chiesi Global Rare Diseases. The topline results showed pegunigalsidase alfa (PRX-102) demonstrated a favorable tolerability and immunogenicity profile. The positive data from the Phase III BALANCE study is expected to support the resubmission of a Biologics License Application for PRX-102.
Last year, the U.S. Food and Drug Administration issued a Complete Response Letter to Protalix and Chiesi for their BLA for accelerated approval of PRX-102. Following a Type A meeting with the FDA, a path toward traditional approval of a BLA was approved. The full BLA approval called for the final data from the BALANCE study. With the results announced today, it appears that necessary data is in hand.
About the Studies Conducted on PRX-102 for Fabry Disease
In the BALANCE study, PRX-102 was administered every two weeks to patients and data was compared to treatment with Fabrazyme, one of two drugs approved in the United States for Fabry disease, a rare, X-linked inherited disease that results from abnormal deposits of a fatty substance in blood vessels. The other approved therapy is Amicus Therapeutics’ Galafold (migalastat). In the study, PRX-102 was shown to be non-inferior to Fabrazyme. Of the patients that completed the trial, 69 have opted to continue receiving PRX–102 every other week in a long-term open-label extension study.
“Based on results from our clinical program, we believe that PRX–102, as a PEGylated enzyme replacement therapy with potentially two different dosing regimens, may be a valuable new treatment option for individuals suffering from Fabry disease,” Dror Bashan, president and chief executive officer of Protalix said in a statement.
The Phase III BALANCE study data builds on previous data showing the efficacy of PRX-102 for Fabry disease. Two years ago, Protalix announced positive switch-over data for patients who switched to PRX-102 from Fabrazyme. In that study, following the switch to PRX-102, researchers noted a substantial improvement in patients’ renal function as measured by the mean annualized estimated Glomerular Filtration Rate.
Protalix Chief Development Officer Einat Brill Almon called the BALANCE data an important milestone for the Fabry community and the company. The study met the predefined criteria for non-inferiority in both the intent-to-treat population and per-protocol analysis.
“These topline results show that PRX–102 was comparable to agalsidase beta in controlling eGFR decline, which is a key measure of Fabry disease progression, and continue to demonstrate a favorable tolerability profile for PRX–102. Combined with previous Phase III results from our BRIGHT and BRIDGE studies, as well as the results from our Phase I/II study and its long-term extension, we believe we have a compelling and consistent dataset from both treatment–naïve and ERT–experienced patients,” Almon said in a statement.
Giacomo Chiesi, head of Chiesi Global Rare Diseases, called the BALANCE data “especially encouraging” based upon the other positive data the two companies have seen with PRX-102. Chiesi said the totality of the PRX-102 data “suggests a favorable benefit-risk profile for the treatment of adult patients with a confirmed diagnosis of Fabry disease.”