Y-mAbs Therapeutics, Inc. a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that the U.S. Food and Drug Administration has approved DANYELZA (naxitamab-gqgk) 40mg/10ml.
DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA is a humanized, monoclonal antibody that targets the ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumors and sarcomas. DANYELZA is administered to patients three times in a week in an outpatient setting and the treatment is repeated every four weeks. The product has received Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations from the FDA.
“Today is an important day for children living with refractory/relapsed high-risk neuroblastoma,” said Thomas Gad, founder, Chairman and President. “It’s very exciting to see this treatment go from being an experimental therapy used at my daughter’s bedside to now being FDA approved. On behalf of Y-mAbs, I want to thank all the patients and physicians who took part in our clinical trials and our scientific partner, Memorial Sloan Kettering, for helping us achieve this goal.”
“We believe that DANYELZA in combination with GM-CSF is a much-needed treatment for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow who have historically not had approved treatments available. This approval of Y-mAbs’ first BLA represents a key step in working towards our mission of becoming a world leader in developing better and safer antibody-based oncology products addressing unmet pediatric and adult medical needs,” said Claus Moller, Chief Executive Officer.
The FDA approval of DANYELZA is supported by clinical evidence from two pivotal studies in patients with high-risk neuroblastoma with refractory or relapsed disease. DANYELZA appears to be well tolerated with few discontinuations of treatment in the clinical trials and adverse events were clinically manageable. See below for information related to adverse reactions.
The FDA granted approval under the accelerated approval regulation. The postmarketing clinical trial required by the FDA to verify and to further characterize the clinical benefit is the ongoing Study 201, which will enroll a minimum of 80 patients and report overall response rate (“ORR”), duration of response (“DOR”), progression free survival (“PFS”) and overall survival (“OS”). The ORR is the primary endpoint for the study, DOR is the secondary endpoint, PFS and OS are secondary endpoints in long-term follow up.
