dm+d

Unassigned

New Medicines

Danyelza High-risk neuroblastoma in children (aged ≥1 year) and adults

Information

Danyelza
New molecular entity
Y-mAbs Therapeutics
Y-mAbs Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Launched
Yes
Yes
Aug 21Has been launched in the US [9].
Nov 20Approved in US for use in combination with GM-CSF for the treatment of relapsed or refractory high-risk neuroblastoma. Accelerated approval of this orphan drug, was based on evidence from two studies; further data from one ongoing study (201) is required to verify and further characterise the clinical benefit [7].
Apr 20Y-mabs has completed the submission of its Biologics License Application under the FDA’s Rolling Review process for naxitamab after market close on March 31, 2020 [6].
Dec 19Y-mAbs plans to commercialise naxitamab in the US and Europe itself [5].
Nov 19Company has submitted to the FDA the first portions of its Biologics License Applicationfor naxitamab for the treatment of patients with relapsed/refractory high-risk neuroblastoma in a rolling review [4].
Apr 19Also has a rare paediatric disease status in the US for the treatment of neuroblastoma [3].
Oct 18Granted orphan drug status in EU for treatment of relapsed or refractory high-risk neuroblastoma. It also has orphan drug status in US for neuroblastoma and osteosarcoma [3].
Aug 18Granted breakthrough therapy status in US, in combination with GM-CSF, for the treatment of high risk neuroblastoma [3].

Category

Humanised anti-GD2 3F8 monoclonal antibody (IgG1). GD2 is a disialoganglioside expressed on almost all of neuroblastoma cancer cells regardless of disease stage and in almost all osteosarcomas, but a highly restricted expression on normal tissue.
Fewer than 100 children are diagnosed with neuroblastoma each year in the UK. However, neuroblastomas are the most common extracranial solid tumour of childhood [1].
High-risk neuroblastoma in children (aged ≥1 year) and adults
Intravenous infusion

Further information

Yes

Trial or other data

Oct 21PII study (201; NCT03363373) continues to recruit, Collection of primary outcome data is due to complete Nov 24 [10].
May 21PI/II study (12-230; NCT01757626) is no longer recruiting, after enrolling 186 participants. Collection of primary outcome data is due to complete Dec 23 [10].
May 20PI/II study (12-230; NCT01757626) is recruiting in the US. Collection of primary outcome data is due to complete Dec 21 [8].
Jan 20Pivotal PII 201 study (NCT03363373, no longer classed as a PIII study) is recruiting; collection of primary outcome data due to complete Nov 24. The recent US approval of Danyelza was based on data from two open-label, single arm trials in patients with high-risk neuroblastoma with refractory or relapsed disease in the bone or bone marrow, Study 201 and Study 12-230 [8].
Mar 19According to its latest annual report, Y-mAbs estimates there are approximately 700 children diagnosed with high‑risk NB in the US each year, and the European market is at least one and a half times the size of the US market with approximately 1,050 patients diagnosed with high‑risk NB in Europe each year. It believes the current addressable market for naxitamab consists of approximately 960 new front‑line high‑risk NB patients each year and 675 primary or second‑line eligible R/R NB pediatric patients each year, representing approximately 40% of all pediatric patients diagnosed with NB in the US and Europe, combined [5].
Mar 19PIII study (NCT03363373) is recruiting [2].
Feb 18PIII trial to evaluate the efficacy of intravenous hu3F8 plus granulocyte-macrophage colony stimulating factor, in high-risk neuroblastoma patients with primary or secondary refractory osteomedullary disease starts (NCT03363373). Evaluation of the overall objective response rate is the defined primary endpoint of the trial. 37 patients will be recruited in the US, Canada, Denmark, Hong Kong, Spain and UK. Collection of primary outcoem data is expected to complete Dec 20 [2].

Evidence based evaluations