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FDA grants orphan drug designation to BPX-501 for GVHD, immunodeficiency after stem cell transplants
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The FDA granted orphan drug designation to BPX-501 for the treatment of patients who develop immunodeficiency or graft-versus-host disease following stem cell transplant, according to a press release from the agent’s manufacturer.
BPX-501 (Bellicum Pharmaceuticals) is an adjunct T-cell therapy of genetically modified donor T cells that incorporates a proprietary safety switch.
The product is designed to eliminate BPX-501 alloreactive T cells should severe GVHD occur. This allows clinicians to more safely perform haploidentical stem cell transplants by adding back BPX-501 genetically engineered T cells to accelerate immune reconstitution and provide control over viral infections, according to the press release.
The orphan drug designation is for the combination of BPX-501 genetically modified T cells and the activator agent rimiducid (Bellicum Pharmaceuticals).
“Expanding the availability of hematopoietic stem cell transplants to more patients with rare genetic diseases and blood cancers is a primary goal of our BPX-501 program,” Tom Farrell, president and CEO of Bellicum Pharmaceuticals, said in the press release. “The FDA’s approval of our request for orphan drug designation is an important milestone that supports our efforts to address key risks and improve outcomes in allogeneic transplant, which could make the procedure more viable for patients who lack a perfect match donor.”
BPX-501 is being evaluated in multiple phase 1 and 2 clinical trials in adult and pediatric patients with leukemia, lymphoma or genetic blood diseases.
Interim results from a study that included pediatric patients with a variety of genetic diseases — including beta thalassemia, Wiskott-Aldrich syndrome and severe combined immunodeficiency — demonstrated efficacy of haploidentical T cell-depleted hematopoietic stem cell transplant followed by an add-back of BPX-501 donor T cells. Results showed reduced infection rates, faster immune reconstitution, and reductions in time to hospital discharge and re-hospitalizations compared with historical controls.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — seven years of market exclusivity.