Bellicum Pharmaceuticals has announced that two of its lead product candidates, BPX-501 and rimiducid, have been granted orphan drug designations by the European Commission’s (EC) drug regulatory agency. Bellicum says it plans to pursue EU approval for BPX-501 and rimiducid therapy based on the ongoing BP-004 clinical trial under the European regulator’s exceptional circumstances provision.
The clinical stage biopharmaceutical company, whose main research focus is on discovering and developing cellular immunotherapies for treating cancers and inherited blood disorders, met with the European Medicines Agency (EMA) to discuss the potential approval pathway for the two products.
Bellicum’s BPX-501 is an additional T-cell therapy designed to improve results after haploidentical hematopoietic stem cell transplantation (HSCT) and to broaden eligibility for the procedure to many more pediatric patients with leukemias, lymphomas and rare inherited blood diseases who do not have a family matched donor. Rimiducid is a proprietary activator agent for treating graft-versus-host disease (GVHD), which can occur after stem cell or bone marrow transplant when newly transplanted donor cells attack the recipient’s body.
The company says its therapy consists of powerful “molecular switches” designed to eliminate, reduce or activate therapeutic cells, to potentially provide greater results and safety compared to current cell therapies, using genetically modified donor T cells with Bellicum’s proprietary CaspaCIDe “safety switch” technology. As a “safety net” it could eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. BPX-501 engineered T cells could speed immune reconstitution and provide more control over viral infections. Bellicum reports that this approach has yielded “highly encouraging early results” noting that children with hematological cancers and life-long genetic blood diseases have been able to leave the hospital either in remission or disease-free.
Earlier this year, the U.S. Food and Drug Administration (FDA) granted both BPX-501 and rimiducid orphan drug status as a combination therapy.
Based on Bellicum’s discussions with the European regulators, the company believes data from the European arm of its BP-004 trial, a phase 1/2 open-label dose escalation trial in pediatric patients with malignant and nonmalignant diseases, could form a basis for EMA Marketing Authorization Applications for BPX-501 and rimiducid. The agency’s Committee for Medicinal Products for Human Use (CHMP) has agreed that review and approval of these drugs “under exceptional circumstances” may be appropriate because a randomized trial may not be practical in a pediatric setting. As an alternative to a randomized trial, Bellicum says it intends to collect data from a concurrent observational study of allogeneic HSCT outcomes in the pediatric setting.
“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 Farrell, 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.”
The EU – EMA orphan drug designation from the EC puts in place regulatory and financial incentives for companies to pursue development of therapies for treating treat life-threatening or very serious conditions affecting no more than five in 10,000 European Union (residents, and for which no treatment is currently approved. Orphan drugs are granted a 10-year marketing exclusivity period in the EU upon subsequent product approval, as well as providing fee waivers, protocol assistance, and marketing authorization under a centralized procedure for granting approval in all 27 EU countries.
More information can be found at http://www.bellicum.com