FDA Approves Aphexda for Use in Stem Cell Mobilization

The FDA has approved BioLineRx’s Aphexda (motixafortide) in stem cell mobilization for autologous transplantation in patients with multiple myeloma. Aphexda is administered by injection and is indicated to be used along with filgrastim (G-CSF). It will be available later this month.

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 success of the transplant depends on adequate mobilization of stem cells during the treatment process. 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.

Aphexdra will have a list price 5,900 per vial, according to a spokesperson. Dosing for Aphexdra is based on weight, and the company expects that based on the average weight of patients with multiple myeloma, most patients will require two vials.

“We believe that Aphexda can change the paradigm of stem cell mobilization in patients with multiple myeloma by providing improved treatment journeys for patients and more certainty for transplant centers,” the spokesperson said. “This price is based on extensive research and reflects the value proposition of Aphexda while appropriately considering the new generic landscape.”

Payers, the spokesperson said, have viewed the clinical data favorably. BioLineRx has met with payers covering more than 90% of commercially covered lives, which includes both commercial payers and CMS. A support program called, BioLineRx Connect, is designed to assist patients with insurance coverage, financial support, the payer process, and additional resources.

John F. DiPersio, M.D., Ph.D.

“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,” John F. DiPersio, M.D., Ph.D., 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, said in a press release.

The approval was based on results from the two-part phase 3 trial GENESIS, which compared Aphexda plus filgrastim with filgrastim plus placebo. Aphexdra plus filgrastim enabled 67.5% of patients to achieve the stem cell collection goal within two apheresis sessions, versus 9.5% for the placebo plus filgrastim regimen. Additionally, 92.5% of patients reached the stem cell collection goal in up to two apheresis sessions in the Aphexda arm and 21.4% in the placebo arm.

The safety data was evaluated in 92 patients with multiple myeloma. Serious adverse reactions occurred in 5.4% of patients receiving Aphexda. These reactions included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia and hypoxia. The most common adverse reactions occurring in GENESIS were injection site reactions (pain, erythema and pruritus), pruritus, flushing, and back pain.

BioLineRx is also studying motixafortide in a phase 2 trial as a first line treatment for patients with metastatic pancreatic cancer, as well as in combination with natalizumab for stem cell mobilization for gene therapies in sickle cell disease.