Protalix BioTherapeutics (PLX) Granted FDA Fast Track Designation for PRX-102 (Pegunigalsidase Alfa)
Protalix BioTherapeutics, Inc. (NYSE: PLX) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to pegunigalsidase alfa, or PRX-102, the Company’s plant cell-expressed recombinant, pegylated, cross-linked a-galactosidase-A candidate for the treatment of Fabry disease. The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs and vaccines for serious conditions that fill an unmet medical need.
“We are very pleased that the FDA has recognized the potential for pegunigalsidase alfa to fill an unmet need for Fabry patients,” said Moshe Manor, Protalix’s President and Chief Executive Officer. 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. “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.”
According to the FDA, Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need, the purpose being to make important new drugs available to patients earlier. A drug that receives Fast Track designation from the FDA is eligible for some or all of the following:
More frequent meetings with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval;
More frequent written communication from the FDA about such things as the design of the proposed clinical trials;
Eligibility for the FDA’s Accelerated Approval and Priority Review, if relevant criteria are met; and
Eligibility for Rolling Review, which allows a drug company to submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by the FDA, rather than waiting until every section of the BLA or NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA.
Fabry disease is a serious, life-threatening condition. It is a disease or condition associated with morbidity that has a substantial impact on survival, day-to-day function, and the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one. Fabry disease is an X-linked multisystem lysosomal storage disorder caused by the absence or reduction of a-galactosidase-A (a-Gal-A) activity, which is a lysosomal enzyme that catalyzes the hydrolysis of globotriaosylceramide (Gb3) from oligosaccharides, glycoproteins and glycolipids. The absence or reduction of this enzymatic activity leads to the progressive accumulation of glycolipids, especially Gb3, in capillary endothelial cells, podocytes, tubular cells, glomerular endothelial cells, mesangial cells, interstitial cells, cardiomyocytes, fibroblasts, and neurons. The accumulation of glycosphingolipids (e.g., Gb3) leads to chronic pain, skin lesions, cardiac, deficiencies, and, in particular, renal involvement. End-stage renal failure and cardiomyopathy often lead to early death in Fabry patients. Fabry disease causes substantial reduction in life-expectancy, by an average of 15 years in female patients and 20 years in male patients, compared to the general population.
Pegunigalsidase alfa is currently being studied globally in three phase III clinical trials. Enrollment in each of the trials continues to progress and estimated timelines for top-line data announcements will be announced upon completion of enrollment for each individual trial.
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