Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger. According to a news release from June 14, 2021, biotechnology company Jasper Therapeutics, Inc. (“Jasper”)’s therapy JSP191 recently received this designation. Altogether, JSP191 conditions patients with severe combined immunodeficiency (SCID) for hematopoietic stem cell transplants.
JSP191
According to Jasper, JSP191 is:
a targeted, first-in-class humanized monoclonal antibody [mAB] in clinical development as a conditioning agent to clear hematopoietic stem cells [HSCs] from bone marrow prior to transplant. A unique compound, JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells.
Normally, SCF and CD117 work in tandem to ensure the survival of stem cells. By preventing CD117 from binding to SCF, JSP191 causes stem cell death, allowing for a space to engraft healthy, donor, or genetically corrected transplanted HSCs. Thus far, JSP191 has been tested in over 90 healthy volunteers. In addition to SCID, Jasper is evaluating JSP191 as a potential therapy for patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).
Rare Pediatric Disease Designation
For SCID, JSP191 received Rare Pediatric Disease designation as a conditioning treatment. In short, a conditioning treatment is one which prepares patients for stem cell transplants. Currently, an ongoing clinical trial is evaluating JSP191 for SCID. Thus far, research shows that JSP191 is relatively safe and well-tolerated in patients from ages 3 months to 38 years. Additionally, the treatment is relatively effective and shows successful HSC engraftment. Through receiving this designation, Jasper also earned a Priority Review Voucher.
Orphan Drug Designation
Outside of the Rare Pediatric Disease designation, JSP191 also received an Orphan Drug designation in a broader sense: as a conditioning treatment for HSC transplantation. Orphan Drug designation is granted to drugs or biologics meant to treat rare illnesses that affect under 200,000 Americans. Alongside this status, Jasper received benefits such as tax credits, fee waivers, regulatory assistance, and 7 years of market exclusivity (once approved).
Severe Combined Immunodeficiency (SCID)
Severe combined immunodeficiency (SCID) is a group of rare genetic disorders which causes extreme immune difficulties and problems fighting infection. At least 13 gene mutations have been associated with SCID. Typically, these genes affect T and B cells (immune cells). As a result, infants are unable to fight off diseases or infections. In many cases, SCID is fatal during infancy (before age 2) unless a child is given immune-restoring treatments. In general, SCID is more common in those of Apache, Navajo, or Turkish descent.
Symptoms include:
- Failure to thrive
- Oral thrush
- Chronic diarrhea
- Skin rashes
- Serious and treatment-averse infections, such as:
- Pneumonia
- Recurrent ear infections
- Hepatitis
- Yeast infections
- Blood infections
- Meningitis
