Protalix BioTherapeutics Inc. (PLX) and Chiesi Global Rare Diseases said, in an update on pegunigalsidase alfa or PRX-102 for the treatment of Fabry Disease, that they are encouraged by productive discussion with the U.S. Food and Drug Administration, which the companies believe provides a pathway to resubmit the PRX-102 Biologics License Application.
Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal a-Galactosidase-A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body.
The Type A meeting was held with the FDA on September 9, 2021. As part of the meeting minutes provided by the FDA, which included the preliminary comments and meeting discussion, the FDA agreed that the data package proposed to the FDA for the BLA resubmission has the potential to support a traditional approval of PRX-102 for the treatment of Fabry disease.
The planned data package for the BLA resubmission, given the changed regulatory landscape in the United States, will include the final two-year analyses of the phase III BALANCE clinical trial.
Protalix and Chiesi also announced that a meeting was held with the Rapporteur and Co-Rapporteur of the European Medicines Agency (EMA) on October 8, 2021 regarding PRX-102.
At the meeting, Chiesi and Protalix discussed the scope of the anticipated Marketing Authorization Application (MAA) for the European Union, and the Rapporteur and Co-Rapporteur were generally supportive of a planned MAA submission for PRX-102. This is an important step in the necessary pre-submission activities leading up to a planned MAA submission.
Chiesi and Protalix expect to submit an MAA for PRX-102 during the first quarter of 2022.
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