FDA Approves Aphexda to Aid in HST Mobilization in Patients With Multiple Myeloma

• Motixafortide plus filgrastim (G-CSF) is the first stem cell mobilization therapy to be granted FDA approval in multiple myeloma.¹

• This marks the first FDA-approved therapy for the developer, BioLineRx Ltd.

• The company plan to make the therapy available later in September.

The FDA has granted approval to motixafortide in combination with filgrastim (G-CSF), a compound therapy intended to mobilize hematopoietic stem cells (HSC) to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma (MM).¹

Approval was granted based on findings from the phase 3 GENSIS study (NCT03246529) in which 122 patients were enrolled to receive motixafortide plus G-CSF, In the double-blind, placebo-controlled, multicenter study, patients were randomized 2:1. Results by central laboratory assessment showed that 67.5% of patients in the motixafortide plus G-CSF arm achieved the stem cell collection goal of ≥ 6 × 10⁶ CD34+ cells/kg within 2 sessions compared with 9.5% of the placebo plus G-CSF arm. Further, 92.5% of patients in the motixafortide/G-CSF reached the stem cell collection goal in up to 2 apheresis sessions vs 21.4% in the placebo arm.^1,2

Safety results from 94 patients in the motixafortide/G-CSF showed that 5.4% of patients experienced serious adverse events (AEs). The serious AEs observed included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia, and hypoxia. The most common AEs occurring in 20% of patients or more were injection site reactions like pain, erythema, and pruritus, pruritus, flushing, and back pain.

“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,” said John DiPersio, MD, PhD, primary investigator for the GENESIS trial and 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, in a press release.¹ “Innovation in this area of medicine has been needed, and today’s approval of APHEXDA addresses the demand for new therapies that can meet today’s challenges by delivering more reliability in stem cell mobilization, versus filgrastim alone, with fewer days of apheresis sessions and fewer doses of filgrastim for people living with this cancer.”

The primary end point of the GENESIS study was the percentage of participants mobilizing ≥ 6.0 x 10⁶ CD34+ cells/kg with up to 2 apheresis sessions. Secondary end points included the rate of patients who collect ≥2.0 x 10⁶ CD34+ cells/kg in 1 apheresis session, the rate of patients who collect ≥6.0 x 10⁶ CD34+ cells/kg in 1 apheresis session, time to neutrophil engraftment, time to platelet engraftment, and graft durability at 100 days post engraftment.

Image Credit: © PepeGallardo - www.stock.adobe.com

To participate, patients must have histologically confirmed multiple myeloma, at least 4 weeks from last induction cycle of chemotherapy, eligible for hematopoietic stem cell transplantation (HSCT), in their first or second complete remission, an ECOG performance status of 0 or 1, adequate organ function at baseline, and using effective contraception. Patients with a previous history of autologous or allogeneic-HCT failed previous HSC collections or collection attempts, or who have received more than 6 cycles of lifetime exposure to lenalidomide are not eligible to participate.

“FDA approval of Aphexda, the company’s first approved therapeutic, is a tremendously exciting and important moment in our history and validates our drug development programs,” said Ella Sorani, PhD, chief development officer of BioLineRx Ltd, in the press release. “We would like to thank all of the patients and families who have contributed to the research and development of Aphexda.”

_REFERENCES:

_{1. 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. September 11, 2023. Accessed September 11, 2023. https://tinyurl.com/mryekzu7}

_{2 .BioLineRx announces positive top-line results from GENESIS phase 3 trial of motixafortide in stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients. News release. BioLineRx. May 4, 2021, Accessed September 11, 2021. https://tinyurl.com/ywcec8hy}