FDA Grants Fast Track Designation to Daretabart for High-Risk Neuroblastoma

The FDA has granted fast track designation to daretabart (hu1418K322A) for the treatment of patients with high-risk neuroblastoma, according to a news release from the developer, Renaissance Pharma Limited.¹

Concurrent with the designation, the FDA also cleared an investigational new drug (IND) application for the agent, permitting the initiation of the phase 2/3 SHINE clinical trial in the US for patients with relapsed or refractory high-risk neuroblastoma. It was also noted that the first commercial-scale good manufacturing practice batch of daretabart was successfully manufactured for use in the SHINE trial. The agent is being developed by Renaissance Pharma under an exclusive license agreement with St. Jude Children’s Research Hospital.

Daretabart targets GD2, a cell surface antigen highly expressed on neuroblastoma cells, to enhance immune-mediated tumor cell killing. Novel structural modifications are also incorporated to improve the tolerability profile.

The clinical development of daretabart is supported by findings from a phase 2 trial (NCT01857934), which evaluated the humanized anti-GD2 monoclonal antibody as part of a frontline multimodality treatment regimen and in the post-consolidation setting for patients with high-risk neuroblastoma.

What efficacy and safety findings were observed with daretabart in high-risk neuroblastoma in the phase 2 trial?

Data published in the Journal of Clinical Oncology demonstrated that the integration of daretabart into induction chemotherapy significantly improved long-term outcomes for newly diagnosed patients.² Among those who received daretabart with induction chemotherapy, the 3-year event-free survival rate was 73.7% (95% CI, 60.0%-83.4%), and the 3-year overall survival rate reached 86.0% (95% CI, 73.8%-92.8%).² Partial responses or better after the first 2 chemoimmunotherapy cycles were noted in 66.7% of patients (95% CI, 55.0%-78.3%). The regimen was also well tolerated with continuous infusion narcotics.

How was the phase 2 trial designed?

This was an open-label, single-arm trial that enrolled children younger than 19 years with newly diagnosed high-risk neuroblastoma. Treatment included 4 daily doses of 40 mg/m² of daretabart on days 2 to 5, administered over 4 hours, given at the same time as each cycle of induction chemotherapy.

The primary objective of the trial was to compare response rates of the first 2 cycles of chemoimmunotherapy—cyclophosphamide, topotecan, and daretabart, followed by granulocyte-macrophage colony-stimulating factor and interleukin-2—with the response rates of cyclophosphamide and topotecan alone, as was reported in a prior study.

“Daretabart has the potential to make a real difference for children with high-risk neuroblastoma, a disease where outcomes remain deeply inadequate despite intensive treatment. FDA fast track designation is an important external validation of that potential, and together with IND clearance and our ability to manufacture at commercial scale, reflects the strength and maturity of this program,” said Simon Ball, interim chief executive officer of Essential Pharma and director of Renaissance Pharma Limited, in the press release.¹ “Having worked exceptionally hard behind the scenes for a number of months, it is with great excitement that we announce this update today. We are executing at pace and look forward to sharing data from the SHINE trial as it progresses. Today’s news brings us another step closer to delivering daretabart as a meaningful new treatment option for children [with] this aggressive cancer.”

How is the SHINE phase 2/3 trial for daretabart designed?

The upcoming phase 2/3 SHINE study will evaluate the efficacy and safety of daretabart specifically in pediatric patients with relapsed or refractory high-risk neuroblastoma.

References

1. Renaissance Pharma Limited, an Essential Pharma company, secures FDA fast track designation and IND clearance for daretabart (hu1418K322A) in high-risk neuroblastoma. News release. Renaissance Pharma Limited. April 14, 2026. Accessed April 14, 2026. https://tinyurl.com/mtyf3d4m
2. Furman WL, McCarville B, Shulkin BL, et al. Improved outcome in children with newly diagnosed high-risk neuroblastoma treated with chemoimmunotherapy: updated results of a phase II study using hu14.18K322A. J Clin Oncol. 2022;40(4):335-344. doi:10.1200/JCO.21.01375