Motixafortide/Filgrastim OKed by FDA for Transplantation in Multiple Myeloma

The News

The FDA has approved motixafortide (Aphexda) combined with filgrastim (G-CSF) to aid in mobilizing hematopoietic stem cells in the peripheral blood for collection, as well as succeeding autologous stem cell transplantation in a population with multiple myeloma, according to a press release from BioLineRx.¹

Study Efficacy

In total, 67.5% of patients reached the stem cell collection goal of 6x10⁶ CD 34-positive cells/kg or more in 2 apheresis sessions by a central laboratory. The corresponding metric in the placebo/filgrastim arm was 9.5%. Moreover, 92.5% and 21.4% of patients reached the goal with up to 2 apheresis sessions.

GENESIS Study Design

The injectable agent’s approval was supported by data from part 2 of the double-blind, placebo-controlled phase 3 GENESIS study (NCT03246529) in which the potential of motixafortide and filgrastim in mobilizing stem cells for transplantation was assessed vs placebo. The first part of the study focused on determining dosage and included 12 patients with multiple myeloma. Additionally, the second part used a 2:1 randomization and included 122 patients.

The injectable agent’s approval was supported by data from the double-blind, placebo controlled phase 3 GENESIS study (NCT03246529) in which the potential of motixafortide and filgrastim in mobilizing stem cells for transplantation was assessed vs placebo.

Pharma Perspective

“Greater numbers of patients with multiple myeloma are candidates for autologous stem cell therapy; however, achieving target collection goals can be difficult in some patients given modern barriers, including the treatment of older patients and use of contemporary induction regimens,” primary investigator John DiPersio, MD, PhD, a professor of Medicine, Pathology, and Immunology and director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine in St. Louis, said in the press release. “Innovation in this area of medicine has been needed, and today’s approval of [motixafortide] addresses the demand for new therapies that can meet today’s challenges by delivering more reliability in stem cell mobilization, [vs] filgrastim alone, with fewer days of apheresis sessions and fewer doses of filgrastim for people living with this cancer.”

Philip Serlin, chief executive officer at BioLineRx also shared perspective on the approval.

“Given the strong efficacy data shown in the GENESIS trial, which included patients who are representative of the current multiple myeloma patient population, we believe [motixafortide] will play a critical role in addressing unmet needs and introduce a new treatment paradigm for this challenging cancer,” Serlin stated.

Safety

In the study’s safety population, the experimental arm received a 1.25 mg/kg subcutaneous dose of motixafortide plus filgrastim (n = 92) and the control arm received placebo and filgrastim (n = 42). In total, 5.4% of patients in the experimental arm had serious adverse effects (AEs); this included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia, and hypoxia. Frequent AEs included injection site reactions, pruritus, flushing, and back pain.

Reference

BioLineRx announces FDA approval of APHEXDA™ (motixafortide) in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma. News release. BioLineRx. September 11, 2023. Accessed September 11, 2023. https://bit.ly/45RWbms