RARE Daily

FDA Grants Fast Track Designation to Three Passage Bio Gene Therapy Candidates for Rare CNS Disorders

March 8, 2021

Rare Daily Staff

The U.S. Food and Drug Administration granted Passage Bio Fast Track designation to the company’s three lead investigational gene therapies.

The FDA designation will facilitate development and expedited review of the company’s gene therapy product candidates for life-threatening diseases including PBGM01 for GM1 gangliosidosis (GM1), PBFT02 for frontotemporal dementia with granulin mutations, 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.

GM1 is a rare and often life-threatening CNS disorder. 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.

Fast Track designation is granted to therapies that demonstrate the 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.

“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, president and CEO 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.”

Photo: Bruce Goldsmith, president and CEO of Passage Bio

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