FDA Accepts NDA of Aphexda for Use in Stem Cell Mobilization

The FDA has accepted for review BioLineRx’s new drug application (NDA) for Aphexda (motixafortide) in stem cell mobilization for autologous transplantation in patients with multiple myeloma. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of Sept. 9, 2023.

In the United States, nearly 15,000 of these transplants are performed each year with the majority in patients with multiple myeloma, a cancer of the while blood cells. It is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the United States.

Autologous stem cell transplantation is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma. The current standard of care includes the administration of five to eight daily doses of granulocyte colony stimulating factor (G-CSF), with or without one to four doses of Sanofi’s Mozobil (plerixafor), an immunostimulant used to mobilize hematopoietic stem cells in cancer patients into the bloodstream.

The application is supported by the results from the GENESIS phase 3 trial of motixafortide in combination with granulocyte colony-stimulating factor (G-CSF) treatment (versus placebo on top of G-CSF) in stem cell mobilization for autologous transplantation in multiple myeloma patients. The study met all primary and secondary endpoints with a very high degree of statistical significance. In the study, about 90% of patients in went directly to transplantation after mobilizing the optimal number of stem cells following only one administration of motixafortide with G-CSF, compared with less than 10% of those receiving G-CSF alone.

The combination was also found to be safe and well tolerated.

BioLineRx submitted the application in September 2022.

A cost-effectiveness study conducted by the Global Health Economics and Outcomes Research (HEOR) team of IQVIA will be presented at the 64th American Society of Hematology (ASH) Annual Meeting held from Dec. 10 to 13, 2022, in New Orleans. The study is an indirect comparison of efficacy data for both motixafortide from GENESIS and plerixafor (aggregate data from literature), estimating plerixafor efficacy as if it had been one arm of the GENESIS trial (Bucher method), and implementing the results in the cost-effectiveness model.

BioLineRx is also developing Aphexda to treat patients with pancreatic ductal adenocarcinoma. The company has teamed up with GenFleet Therapeutics to conduct a study in China of motixafortide as a first-line treatment. BioLineRx is continuing its collaboration with Colombia University in an on-going phase 2 investigator-initiated study to evaluate motixafortide in combination with Regeneron’s anti-PD-1 Libtayo (cemiplimab) and standard-of-care chemotherapy (gemcitabine and nab-paclitaxel) in first-line pancreatic ductal adenocarcinoma patients.