The FDA has granted orphan drug status to P-BCMA-101, a cell-based immunotherapy for the treatment of relapsed or refractory multiple myeloma.
P-BCMA-101 (Poseida Therapeutics) is an autologous chimeric antigen receptor T-cell therapy comprising a high percentage of stem cell memory T cells that target cancer cells expressing the B-cell maturation antigen.
“P-BCMA-101 has demonstrated outstanding potency, with strikingly low rates of toxicity in our phase 1 clinical trial,” Eric Ostertag, MD, PhD, CEO of Poseida Therapeutics, said in a press release. “In fact, the FDA has approved fully outpatient dosing in our phase 2 trial starting in the second quarter of 2019.”
Poseida Therapeutics previously reported preliminary data from its phase 1 trial of P-BCMA-101 for patients with multiple myeloma. Eleven patients were participating in the study as of September 2018, with 70% having at least a partial response to treatment. One patient had suspected cytokine release syndrome and no patients experienced neurotoxicity; both are common toxicities associated with CAR T-cell therapy.
P-BCMA-101 was developed using Poseida’s proprietary piggyBac technology — a DNA modification system that “results in CAR-T product candidates with a high percentage of stem cell memory T cells, the only T cell that is self-renewing and long-lived, leading to CAR-T products with improved efficacy, lower toxicity and potentially greater durability than earlier-generation CAR-T therapies,” according to the release.
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 — 7 years of market exclusivity.