U.S. Food and Drug Administration Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates Targeting Rare CNS Disorders

By Globe Newswireabout 1 month ago


PHILADELPHIA, March 08, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company's three lead investigational gene therapies: PBGM01 for the treatment of GM1 gangliosidosis (GM1), PBFT02 for frontotemporal dementia with granulin mutations (FTD-GRN), and PBKR03 for Krabbe disease. Passage Bio plans to initiate clinical trials in GM1 in the first quarter of 2021 and in FTD-GRN and Krabbe disease in the first half of 2021.

“At Passage Bio, we are working to address some of the world's rarest and most devastating neurological diseases that affect infants and adults,“ said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “The FDA's decision to grant Fast Track designation to each of our lead gene therapy candidates highlights the urgent need for new treatments in these diseases and represents an important step towards achieving our objective of getting potentially transformative therapies to patients as quickly as possible.“

Fast Track designation facilitates the expedited development and review of a new drug that demonstrates potential to address unmet medical needs and treats a serious or life-threatening disease. Benefits of this designation include more frequent interactions with the FDA to discuss the drug's development plan, as well as eligibility for other regulatory mechanisms intended to expedite development and review, such as priority review.

“In the last year, we have laid the groundwork for Passage Bio to boldly execute on our strategy,“ said Dr. Goldsmith. “These regulatory designations are intended to accelerate the timelines in our efforts to make a meaningful difference in patients' lives.“

PBGM01: Addressing the urgent need in infantile GM1

PBGM01 is being studied for the treatment of GM1, a rare and often life-threatening CNS disorder that impacts patients worldwide. Passage Bio is targeting the infantile form of the disease, which is the most severe, with a rapid disease course and no current treatment options beyond support care. PBGM01 has received Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation from the European Medicines Agency (EMA). Passage Bio has activated its first clinical trial site in the United States for the global Imagine-1 study evaluating PBGM01 ...

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