Rare Daily Staff

The U.S. Food and Drug Administration granted rare pediatric disease designation to Jasper Therapeutics JSP191 as a conditioning treatment for patients with severe combined immunodeficiency, a life-threatening genetic illness.

The agency also granted orphan drug designation to JSP191.

Severe combined immunodeficiency (SCID) is a rare, pediatric disorder affecting an estimated one in 58,000 births in the general population. The condition results in the absence or lack of function of key immune cells, resulting in a severely compromised immune system. It is typically fatal within two years without hematopoietic cell transplantation.

JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients.

Jasper is currently conducting clinical studies of JSP191 as a conditioning agent prior to hematopoietic stem cell transplant in patients with SCID and separately in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).

The Rare Pediatric Disease designation is open to drug development programs targeting serious, life-threatening diseases that primarily affect less than 200,000 Americans aged 18 or younger. Once a new drug application is approved under this program, the company is eligible to receive a priority review voucher for any related marketing application.

The voucher, which reduces the standard review time for a new drug application by four months, is potentially lucrative because it is transferable. Most recently, Rhythm Pharmaceuticals sold a priority review voucher to Alexion for $100 million.

“The FDA’s decision to grant orphan drug designation to JSP191, and rare pediatric disease designation in SCID, underscores the critical need for innovative conditioning agents for patients undergoing hematopoietic cell transplant, in an indication where currently there is no FDA approved or consensus guidelines recommended conditioning agent,” said William Lis, executive chairman and CEO of Jasper Therapeutics. “Hematopoietic cell transplant is a potentially life-saving cure for multiple diseases such as SCID, acute myeloid leukemia, myelodysplastic syndromes, sickle cell disease, autoimmune disease and others. However, the genotoxic mechanism of action of current conditioning agents limits the use of curative transplants due to severe adverse safety events or lack of efficacy due to the use of a reduced dose or no conditioning prior to transplant.”

Photo: William Lis, executive chairman and CEO of Jasper Therapeutics

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