- The FDA has granted Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation to FBX-101 for treatment of patients with Krabbe disease - FBX-101 is a first-in-human gene therapy utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intraveniously to cells in the central nervous system (CNS) and peripheral organs
| COLUMBUS, Ohio, Feb. 16, 2021 /PRNewswire/ -- Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intravenious gene therapy program for patients with Krabbe disease and marks a major step forward in building out the company’s hybrid model as a gene therapy manufacturing and development engine. “FDA’s decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients,” said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. “Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease.” Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company’s Biologics License Application (BLA). The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. 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. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. “Infantile Krabbe is a progressive and devastating leukodystrophy,” said Jessie Barnum, M.D., Assistant Professor, Department of Pediatrics, Division of Blood and Marrow Transplantation and Cellular Therapies and Principal Investigator of the FBX-101 trial at UMPC. “FBX-101 is an AAV gene therapy that has shown promising preclinical efficacy in Krabbe animal models of disease by extending survival and improving neuromuscular function when administered early in the disease course.” “The FBX-101 preclinical data brings a new wave of hope to the Krabbe community,” said Anna Grantham, Director of Leukodystrophy Care Network Programs at Hunter’s Hope. “These FDA designations for FBX-101 underscore a beautiful and collective effort to accelerate the timelines of bringing this potential therapy to patients who urgently need them.” “To see a promising new treatment for Krabbe receive these designations so quickly brings us one step closer to what everyone in our disease community is ultimately working towards: an FDA-approved treatment for Krabbe disease to reach the beside of all patients impacted by this disease,” said Stacy Pike-Langenfeld, Director of Programs and Administration at The Legacy of Angels Foundation. “Our mission has always been to promote research to develop and enhance treatments for Krabbe disease, so it’s very encouraging to see that Forge and FBX-101 have made so much progress in such a short amount of time.” Patients and families can learn more about clinical trials for FBX-101 by visiting https://www.forgebiologics.com/science/#krabbe. About Krabbe disease About FBX-101 About Forge Biologics For more information, please visit https://www.forgebiologics.com. Patient, Pediatrician, Genetic Counselors & Family Inquiries Media Inquiries: Investor Relations and Business Development SOURCE Forge Biologics |
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