Protalix BioTherapeutics, Inc. announced that it recently held an End-of-Phase II meeting with the U.S. Food and Drug Administration (FDA) to discuss the Company's proposed BLA plan for PRX-102 for the treatment of Fabry disease. Official FDA meeting minutes indicate the FDA's acceptance of the Company's path forward for a phase III clinical trial to support a full BLA approval.
The phase III clinical trial will be a randomized, multi-center, placebo-controlled, safety and efficacy study in treatment-naïve Fabry patients evaluating the 1 mg/kg dose of PRX-102. The Company anticipates a small sample size of patients will be needed to achieve statistical significance with a study duration of approximately six months. The primary endpoint will be Gastrointestinal Symptoms, with key secondary endpoints including renal function.
In the official FDA meeting minutes, the FDA noted that the Company reported interim analysis results from its phase I/II clinical trial of PRX-102 that preliminarily show a favorable trend in the severity and frequency of abdominal pain and frequency of diarrhea after six months of treatment with PRX-102. According to the FDA, during a recent ERT (enzyme replacement therapy) shortage, patients who reduced or discontinued ERT dosing developed worsening of GI signs and symptoms within a few weeks to months.
In addition to the phase III clinical trial described above, the Company and the FDA also agreed to a phase III head-to-head superiority trial comparing PRX-102 versus Fabrazyme, which the Company plans to commence in early 2016. The primary endpoint for this head-to-head trial will be an improvement in eGFR. The trial will enroll patients who are currently treated with Fabrazyme; such patients will be treated with 1mg/kg of PRX-102 for a two-year period. Interim results from this head-to-head trial will also provide supportive safety data for the BLA submission.
"We are very pleased with the outcome of the FDA meeting and appreciate the valuable guidance the agency has provided us regarding the phase III clinical program required to support a full BLA approval," said Moshe Manor, Protalix's President and Chief Executive Officer. "We are in a strong financial position and believe we are well capitalized to run both phase III trials through an anticipated BLA filling."
The Company plans to submit a request for a Special Protocol Assessment (SPA) to the FDA later this year, and to commence both Phase III trials in early 2016.
