Y-mAbs Therapeutics, a commercial-stage biopharmaceutical firm, has obtained the US Food and Drug Administration (FDA) approval for danyelza (naxitamab-gqgk) to treat Neuroblastoma.
The US regulatory agency indicated danyelza 40mg/10ml in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for the treatment of neuroblastoma in paediatric patients, aged one year and above.
Also, the combination indicated for treating relapsed or refractory high-risk neuroblastoma in the bone or bone marrow of adult patients, with partial response, minor response, or stable disease to previous treatment.
Y-mAbs said that the drug is approved under accelerated approval, based on overall response rate and duration of response. Continued approval for the indication is subject to validation of clinical benefits in a confirmatory trial.
Developed by researchers at Memorial Sloan Kettering Cancer Center (MSK), danyelza is a humanised, monoclonal antibody, exclusively licensed to Y-mAbs.
The drug is designed to target ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumours and sarcomas, and is administered three times in a week in an outpatient setting, repeated every four weeks.
Y-mAbs founder, chairman and president Thomas Gad said: “Today is an important day for children living with refractory/relapsed high-risk neuroblastoma.
“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.”
FDA approval of danyelza is based on clinical data from two clinical studies
The company has secured FDA Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations for the product.
The FDA approval of danyelza is supported by clinical evidence from two clinical studies in patients with high-risk neuroblastoma with refractory or relapsed disease.
Neuroblastoma is a solid tumour that arises in the nervous system, outside of the brain, with variable clinical behaviour, as some tumours are easily treatable, while the most being aggressive.
In the clinical trials, treatment using danyelza was well tolerated with limited treatment discontinuations and clinically manageable adverse events, said the company.
Also, the FDA requires a post-marketing clinical trial, the ongoing Study 201, to verify and further characterise the clinical benefit of the drug.
The trial that will enrol at least 80 patients and report overall response rate (ORR), which is the primary endpoint, and duration of response (DOR), progression free survival (PFS) and overall survival (OS), the secondary endpoints.
Y-mAbs chief executive officer Claus Moller said: “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 paediatric and adult medical needs.”
In August this year, Roche secured the European Commission conditional marketing authorisation for Rozlytrek (entrectinib) to treat a type of solid tumours.
