The FDA has granted Protalix BioTherapeutics Inc., an Israel-based company, fast track status for PRX-102, the company’s infusion treatment for Fabry disease.
PRX-102 (pegunigalsidase alfa) also has orphan drug status in the United States, which gives the company market protections during the early phases of the drug launch.
“We are pleased that the FDA has recognized the potential for pegunigalsidase alfa to fill an unmet need for Fabry patients,” Moshe Manor, the president and CEO of Protalix, said in a press release. “We believe that fast track designation will help facilitate our development program for pegunigalsidase alfa and may shorten the timelines to an anticipated approval, which will greatly benefit Fabry patients.”
The release noted, “The data generated in our clinical trials of pegunigalsidase alfa thus far, as well as nonclinical data, as presented to the FDA with Protalix’s application for fast track designation, demonstrate that pegunigalsidase alfa has the potential to address an unmet medical need for Fabry patients, such as the prevention of renal failure, improved survivability and a positive impact on quality of life.”
According to the release, pegunigalsidase alfa has demonstrated enhanced circulatory half-life, with higher enzyme activity in target organs affected by Fabry disease. The company is developing pegunigalsidase alfa in two dosing regimens to provide better efficacy and to lower the treatment burden of bi-weekly infusions by reducing the number of infusions by half for patients with Fabry disease and declining renal function.
Last September, the FDA gave Amicus Therapeutics orphan drug status and fast track designation for its oral drug migalastat to treat Fabry disease. The company submitted a new drug application for migalastat on Dec. 14.