Protalix Biotherapeutics Inc (NYSE:PLX) received CRL (complete response letter) from the US FDA for its BLA (Biologics License Application) for PRX-102 (pegunigalsidase alfa) on April 28, 2021, to cure adults with Fabry disease. The FDA did not mention any efficacy or safety concerns in CRL for PRX-102.
The FDA inspected the manufacturing facility of Protalix based in Carmiel, Israel. Further assessments related to the formulation are required to approve the BLA.
The FDA cannot carry out inspections during the review cycle because of travel restrictions in that location. It will monitor the travel restrictions and public health to schedule inspections. The fill and finish processes of PRX-102 are conducted at a third-party facility in Europe.
Reviews records under section 704(a)(4)
FDA reviewed the records of this drug under Section 704(a)(4) of the central FCDA (Food, Drug, and Cosmetic Act) instead of PLI (pre-licensing inspection). It will inform the company to resolve any pending issues at the facility in due course.
Protalix will closely work with the US FDA to find an expeditious pathway to get approval for its PRX-102. It is confident of resolving pending issues with the regulator and provides an alternative and innovative treatment to Fabry patients.
CEO of Protalix, Dror Bashan, said the company is disappointed with receipt of CRL from the FDA in April 2021. However, the regulator has not mentioned any adverse efficacy or safety concerns in its CRL for PRX-102. The company is working with FDA to complete subsequent assessment and inspection soon after lifting the travel restrictions.
The company is advancing its early-stage formulations and expects to achieve progress throughout this year.
Reports positive topline results of PRX–102 treatment
Protalix and Chiesi reported positive topline results of phase III clinical trial that examines the efficacy and safety of PRX-102 on February 23, 2021. The trial is conducted on 30 Fabry patients treated earlier with Fabrazyme. The patients have well tolerated the drug and showed stable clinical presentation.
Protalix plans to secure capital to further develop PRX-102 following the receipt of CRL of the US FDA. The company signed an agreement with Chiesi on May 13, 2021, to provide capital by amending the supply and license agreements for PRX-102.
The company reported revenues of $4.5 million (down 10% YoY) from the sale of goods in Q1 2021. It also posted revenues of $6.8 million from R&D services and licenses.
