August 12, 2020
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FDA grants priority review to drug that may stabilize kidney function in Fabry disease

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The FDA has accepted a biologics license application and granted priority review to a therapy shown to stabilize kidney function in patients with Fabry disease, according to a press release from Protalix BioTherapeutics.

The release further stated that the drug, known as pegunigalsidase alfa (PRX-102), was designed to stop the accumulation of globotriaosylceramide (Gb3) and slow the progression of end-organ failure. While kidneys are the most commonly affected organ by the disease, the heart and the cerebrovascular system can also be impacted and patients may experience pain and impaired peripheral sensation, according to the release.

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Protalix BioTherapeutics, along with its commercialization partner Chiesi Global Rare Diseases, submitted the BLA following results from clinical trials which yielded positive results for this patient population.

“PRX-102 represents an important advance in research with the potential to deliver significant advantages to patients with Fabry disease," Giacomo Chiesi, head of Global Rare Diseases, said in the release. “We are very encouraged by the strong interest in this therapy among both patients and clinicians and we look forward to the prospect of making it available to patients around the world who can benefit from treatment.”

Dror Bashan, president and CEO of Protalix, also weighed in on the action by the FDA.

“The FDA’s acceptance of the BLA and grant of priority review for PRX-102 are significant achievements for Protalix and Chiesi, and represent a crucial step forward as we look to establish a new treatment option to the Fabry patient community,” he said. “Based on the encouraging results for PRX-102 we have seen to date, we are eager to continue discussions with the FDA and to continue our other development efforts for PRX-102, as marketing approval of PRX-102 is our top priority.”

According to the release, the FDA has set an action date of Jan. 27, 2021 under the Prescription Drug User Fee Act, though it is not currently planning to hold an advisory committee meeting to discuss the application.