dm+d

Unassigned

New Medicines

Neuroblastoma in children

Information

New molecular entity
Y-mAbs Therapeutics
Y-mAbs Therapeutics

Development and Regulatory status

None
Pre-registration (Filed)
Phase III Clinical Trials
Yes
Yes
Apr 21MAA filed in EU for use in paediatric patients with central nervous system/leptomeningeal metastasis from neuroblastoma. Y-mAbs plan to submit a NDA to FDA Q2 or Q3 2021 [11].
Dec 20Y-mAbs plans to resubmit its omburtamab BLA for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma to the FDA in the beginning of 2021 [10].
Jul 20Y-mabs has announced initiation of its Biologics License Application (“BLA”) for omburtamab under the U.S. Food and Drug Administration’s (“FDA”) Rolling Review process [7].
Dec 19According to its latest annual report, Y-mAbs is in pivotal stage development for I‑omburtamab for the treatment of paediatric CNS/leptomeningeal metastases from neuroblastoma. It will advance the drug through an expedited regulatory pathway and expects it to be eligible for priority review/breakthrough designations in the US. It expects to submit a BLA in 2019, with a goal of receiving approval by the FDA in 2020. It plans to commercialise the drug in the US as soon as possible after obtaining FDA approval, if such approval occurs [4].
Dec 19Has orphan drug status in EU & US, plus rare paediatric disease status in US [3].
Dec 19Y-mAbs Therapeutics intends to submit a MAA to the EMA in H2 20 [6].
Jul 19US FDA grants Breakthrough Therapy designation to omburtamab for the treatment of paediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis [3].

Category

Theranostic monoclonal antibody 8H9, conjugated to to 131 iodine. The antibody 8H9 acts by targeting B7-H3 or CD276 receptors present on cancer cells, causing radiolabelled iodine to kill cancerous tumours.
Neuroblastoma represents about 10% of solid tumors in infants and children under the age of 15, with an annual incidence of about 1/70,000 in children in this class of age [1].
Neuroblastoma in children
Intracerebral

Further information

Yes
To be confirmed

Trial or other data

Dec 20Y-mAbs Therapeutics announces preliminary efficacy results from its pivotal PII/III 101 trial in patients with CNS/leptomeningeal metastasis from neuroblastoma. Overall survival was 75% after a period of 18 months. An additional independent radiographic evaluation of the tumour responses showed that for ten evaluable patients with measureable disease, a total of 40% of the patients responded to omburtamab, 20% with complete response (CR) and 20% with partial response (PR), and another five patients had stable disease (SD). All nine patients with response or SD maintained these at six months follow up. The study results were supportive of the conclusion from an earlier Study 03-133 [9].
Sep 20PII/III trial (101; NCT03275402) continues to recruit and collection of primary outcome data now expected Dec 20 [8].
Aug 19PII/III trial (NCT03275402) is recruiting; timescales unchanged [5].
Mar 19Y-mABs believes that the European market is at least one and a half times the size of the US market and that there are approximately 1,050 patients diagnosed with NB in Europe each year. The current addressable market for omburtamab, consists of approximately 200 new patients each year with CNS/LM from NB in the US and Europe, combined [4].
Oct 18Oct 18: PII/III trial to evaluate the efficacy and safety of intracerebroventricular 131I-omburtamab in patients with neuroblastoma of age up to 18 years starts (101; NCT03275402). Evaluation of the overall survival rate is the defined primary endpoint of the trial. The open label trial is presently enrolling approximately 32 patients in the US, Denmark and Spain. Collection of primary outcome data is expected to complete Dec 19 [2].

Evidence based evaluations