FDA Grants Jasper Fast Track Designation for Therapy to Treat SCID Patients Undergoing Stem Cell Transplant
September 15, 2022
The U.S. Food and Drug Administration granted Fast Track designation to Jasper Therapeutics for JSP191, an anti-CD117 monoclonal antibody, for the treatment of patients with severe combined immunodeficiency (SCID) undergoing allogeneic hematopoietic stem cell transplant.
Severe combined immunodeficiency (SCID) is a group of rare disorders caused by mutations in different genes involved in the development and function of infection-fighting immune cells. Infants with SCID appear healthy at birth but are highly susceptible to severe infections.
JSP191 is a humanized monoclonal antibody that blocks stem cell factor receptor signaling leading to the clearance of hematopoietic stem and progenitor cells from the bone marrow. JSP191 is in clinical development as a stem cell transplant conditioning agent where it helps create an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high-dose myeloablative conditioning is associated with severe toxicities and standard low-dose conditioning has limited efficacy.
To date, JSP191 has been evaluated in more than 110 healthy volunteers and patients. Four clinical trials for myelodysplastic syndromes (MDS)/ acute myeloid leukemia (AML), severe combined immunodeficiency (SCID), Fanconi anemia (FA) and Sickle Cell Disease undergoing allogeneic transplant are currently ongoing. JSP191 is also planned to enter clinical development as a second-line therapeutic in transfusion-dependent, lower-risk MDS patients to preferentially drive recovery of healthy hematopoietic stem cells in order to help restore normal hematopoiesis. JSP191 has been studied in 14 SCID patients in an ongoing multicenter clinical trial with clinical outcome data presented at academic medical conferences.
“Patients born with SCID have a severely compromised immune system and need to rely on an allogeneic hematopoietic stem cell transplant to create the immune cells needed to fight infection,” said Ronald Martell, president and CEO of Jasper Therapeutics. “Unfortunately, many patients are too fragile to tolerate the toxic chemotherapy doses typically used in transplant and may suffer severe side effects or fail transplant. Along with the FDA’s previous designations of Orphan and Rare Pediatric Disease for JSP191, this new Fast Track designation recognizes the potential role of JSP191 in improving clinical outcomes for these patients and will allow us to more closely work with the FDA in the upcoming months to determine a path toward a Biologics License Application (BLA) submission.”
The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill unmet medical needs. The purpose is to accelerate the development of important new drugs for patients. Drugs granted Fast Track designation are eligible for more frequent meetings with the FDA to discuss the drug’s development plan and ensure the collection of appropriate data needed to support approval, as well as eligibility for Accelerated Approval, Priority Review and Rolling Review if relevant criteria are met.
Author: Rare Daily Staff
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