Aphexda (motixafortide) from BioLineRx has been given U.S. approval in conjunction with filgrastim (G-CSF) to facilitate autologous hematopoietic stem cell transplant (AHSCT) for multiple myeloma patients.
Effective bloodstream mobilisation of healthy blood stem cells for collection and subsequent transplant is essential to the success of AHSCT. The combination was demonstrated to aid in mobilising blood stem cells in the Phase 3 GENESIS clinical trial (NCT03246529) in fewer sessions and with lower drug doses.
“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 Aphexda will play a critical role in addressing unmet needs and introduce a new treatment paradigm for this challenging cancer,” Philip Serlin, BioLineRx’s CEO, said in a company press release.
Later this month, according to BioLineRx, Aphexda should become accessible in the US.
“The company is working relentlessly to make this important innovation in stem cell mobilization available to appropriate patients, their physicians and transplant teams,” Serlin added.
The FDA’s clearance marks “the first innovation in stem cell mobilisation for multiple myeloma to be approved in the U.S. in a decade,” according to BioLineRx, and comes approximately 10 months after the organisation agreed to consider the regulatory application.
A blood cancer called multiple myeloma develops from aberrant plasma cells, a type of white blood cell produced in the bone marrow. The development of blood stem cells, which give rise to all other types of blood cells, is hampered by these aberrant plasma cells’ uncontrollable ability to multiply in the bone marrow.
AHSCT is a typical myeloma treatment that entails removing all of the patient’s remaining blood cells from the body with chemotherapy and replacing them with their own healthy blood stem cells. The body is then repopulated with fresh, healthy blood cells after the stem cells have been transplanted.
The quantity of blood stem cells that can be extracted from the bone marrow will determine how successful the treatment is. Filgrastim is typically used daily before apheresis to promote stem cell mobilisation in this delayed process.
3 million to 5 million stem cells per kilogramme (kg) of body weight is the suggested collecting objective. After one apheresis session, however, up to 47% of myeloma patients have trouble reaching collection target numbers, the business reported.
“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, the GENESIS trial’s principal investigator at Washington University School of Medicine in St. Louis.
By interacting with and inhibiting the protein CXCR4, which normally retains stem cells in the bone marrow, aphexda is anticipated to aid in the movement of stem cells from the bone marrow into the bloodstream.
In the second phase of the multicenter GENESIS trial, which included 122 adult myeloma patients, researchers compared the safety and effectiveness of Aphexda plus filgrastim and filgrastim plus a placebo for mobilising stem cells for AHSCT.
The trial’s participants, who had a median age of 63 and were receiving Revlimid (lenalidomide) in roughly 70% of both groups, were typical of the contemporary patient population.
The major objective was to collect at least six million stem cells per kg in up to two apheresis sessions by injecting 1.25 mg of aphexda under the skin.
According to measurements made by nearby laboratories, the collection objective was met in the majority of patients (92.5%) in the Aphexda plus filgrastim group as opposed to 21.4% of those receiving filgrastim with a placebo.
When measurements were made by central laboratories, the Aphexda combination outperformed filgrastim alone: more than two-thirds (67.5%) of patients met the objective compared to 9.5% of those on the filgrastim plus placebo group.
Additionally, compared to patients receiving filgrastim alone, individuals receiving Aphexda with filgrastim required fewer filgrastim injections (5.26 vs. 8.12) and fewer apheresis operations (1.23 vs. 3.24) to collect adequate stem cells.
The most frequent adverse event was transient injection site responses, but the combination therapy was typically safe and well tolerated.
5.4% of the 92 patients who received the Aphexda combination had serious adverse effects. Vomiting, adverse reactions at the injection site, allergic reactions, low potassium, and low oxygen levels were some of these side effects.
“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,” DiPersio said.