Bellicum Pharmaceuticals has just announced that its lead immuno-oncology product candidate BPX-501 and its activator agent rimiducid have received orphan drug designations by the European Commission (EC), after receiving the same designation by the U.S. Food and Drug Administration earlier this year.
The decision means Bellicum will pursue an early market authorization in Europe.
Orphan status provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or very serious conditions that affect very few people in the European Union (EU), and where no treatment is currently approved.
Early marketing authorization can be granted based on exceptional circumstances such as medicines targeting very rare diseases and, or when controlled studies are impractical or inconsistent with accepted principles of medical ethics.
Bellicum met with regulatory authorities to discuss the approval of T-cell therapy BPX-501 for the treatment in hematopoietic stem cell transplantation (HSCT) and of rimiducid for the treatment of graft versus host disease (GvHD) following a haploidentical HSCT in children with leukemias, lymphomas and rare inherited blood diseases who have not found a matched donor.
GvHD can occur after transplant when the transplanted cells regard the recipient’s body as foreign and then attack the recipient’s body.
To ground the Marketing Authorization Applications for the lead product candidates, the company will present data from the European arm of its BP-004 clinical trial, which includes a six-month follow-up time and expanded time to enroll additional patients.
BP-004 is a Phase 1/2 open-label, dose escalation trial in pediatric patients with malignant and non-malignant diseases evaluating the safety of administering BPX-501 T-cells from a haploidentical donor given after T-depleted HSCT. The trial also wants to verify if the treatment can enhance immune reconstitution.
BPX-501 is designed to provide a safety net to eliminate alloreactive BPX-501 T-cells (via administration of the activator agent rimiducid) should uncontrollable GvHD develop. The goal is to help physicians perform safer stem cell transplants by adding BPX-501 engineered T-cells to accelerate immune reconstitution and control infections while avoiding GvHD.
The trial is being conducted in Europe and the U.S. in specialized pediatric centers. Interim results were presented at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation, in April.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) agreed that the exceptional marketing approval might be suitable, recognizing that a randomized trial might be impractical in a pediatric setting. Instead of a randomized trial, Bellicum will collect data from a concurrent observational study of allogeneic HSCT outcomes in children.
“We are pleased with the progress we have made toward defining an expedient pathway to approval of BPX-501 and of rimiducid for pediatric transplant patients in Europe,” said Tom Farrel, Bellicum’s president and chief executive officer, in a press release. “We are now initiating discussions with the FDA, and expect to be able to provide additional guidance on the approval pathways during the fourth quarter.”