Passage Bio’s PBKR03 Receives Orphan Drug and Rare Pediatric Disease Designations from FDA for Treatment of Krabbe Disease

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[{"type":"text","content":"PHILADELPHIA, Oct. 28, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. Krabbe disease is a rare and often life-threatening lysosomal storage disease that presents early in the patient’s life, resulting in progressive damage to both the brain and peripheral nervous system.\n “Receiving both Orphan Drug and Rare Pediatric Disease designations for PBKR03 underscore the urgent unmet medical need for children with Krabbe disease, for which there are no approved treatments,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “We are encouraged about the potential of PBKR03 as a life-altering therapy for this underserved patient population, and we look forward to working with the FDA as we solidify our plans to advance PBKR03 into clinical testing in 2021.” The FDA grants Orphan Drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug designation affords Passage Bio the potential for certain benefits, including up to seven years of market exclusivity, assistance in the drug development process, tax credits for clinical development, and exemptions from certain FDA fees. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. Under the RPD program, and subject to FDA approval of PBKR03 for the treatment of Krabbe disease, Passage Bio would be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for any subsequent marketing application, or sold or transferred to other companies for their programs. About Krabbe Disease Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which ...

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