Leuven, Belgium, ONCURIOUS NV, an emerging oncology company focused on the development of innovative orphan drugs for the treatment of pediatric tumors, today announces that the European Commission has confirmed the orphan drug designation for TB-403 for medulloblastoma. Today's decision by the EC follows the earlier positive opinion issued by the European Medicine Agency (EMA).
TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF). PlGF is expressed in several types of cancer, including medulloblastoma. In medulloblastoma patients, high expression of the PlGF receptor neuropilin 1 has been shown to correlate with poor overall patient survival.
An orphan drug designation is for "medicines to be developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious".
Today's confirmation by the EC follows an earlier in-depth review and positive opinion on the drug candidate by the EMA Committee for Orphan Medicinal Products (COMP). The COMP gathers a group of experts chosen on the strength of their qualifications and a number of patient organization representatives. Altogether, the COMP assesses the potential incremental benefits of a drug candidate versus existing treatments.
TB-403 is currently being evaluated in a Phase I/IIa clinical trial for treatment of medulloblastoma, a rare, life-threatening pediatric cancer.
The clinical trial is being conducted in partnership with the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC). The Consortium is a collaboration of 25 US academic medical centers, teaching hospitals and other entities, with the purpose of facilitating and conducting collaborative research activities and investigations of new treatments for neuroblastoma, medulloblastoma and other pediatric cancers. NMTRC is headquartered in Grand Rapids, MI, USA.
ONCURIOUS nv is working with BioInvent International AB as a development partner for this project.
Patrik De Haes, MD, Executive Chairman of ONCURIOUS nv comments
"We are very happy with today's news. Gaining this orphan-drug designation is an important validation of the TB-403's scientific rationale and preclinical data. It also offers very valuable potential benefits including protocol assistance and fee waivers, as well as 10-year market exclusivity once the medicine is on the market in Europe."