Target therapy for high-risk neuroblastoma treatment: integration of regulatory and scientific tools is needed.

ABSTRACT:

Introduction

Several new active substances (ASs) targeting neuroblastoma (NBL) are under study. We aim to describe the developmental and regulatory status of a sample of ASs targeting NBL to underline the existing regulatory gaps in product development and to discuss possible improvements.

Methods

The developmental and regulatory statuses of the identified ASs targeting NBL were investigated by searching for preclinical studies, clinical trials (CTs), marketing authorizations, pediatric investigation plans (PIPs), waivers, orphan designations, and other regulatory procedures.

Results

A total of 188 ASs were identified. Of these, 55 were considered 'not under development' without preclinical or clinical studies. Preclinical studies were found for 115 ASs, of which 54 w

SUBMITTER: Ceci A 

PROVIDER: S-EPMC10377668 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature