Noetica Pharma has received the orphan drug designation for its drug candidate BRT002 from the European Medicines Agency. BRT002 offers hope for the treatment of neonatal hypoxic-ischemic encephalopathy, a severe neurological condition that can lead to long-term severe neurological deficits.
The startup relies on several patents derived from research conducted at the Laboratory for the Study of the Neurovascular Unit and Therapeutic Innovation (LENIT, SPI/DMTS). It was created at the end of 2025 by CEA, Technofounders, and four researchers, two of whom are from CEA-Joliot.
Questions
to Aloïse Mabondzo, director of the LENIT laboratory and who led the work
leading to the creation of the startup.
How
is this EMA designation a recognition of your work on neonatal hypoxic-ischemic
encephalopathy for more than ten years?
A.M. :
"For a drug to be designated as an orphan drug, on the one hand, the targeted pathology must be an orphan disease (with epidemiological data) and, on the other hand, the data obtained in vivo must show a potential therapeutic interest of the product. All the work carried out by the team for more than ten years now was sufficiently solid and supported to convince the authorities of the potential of our drug candidate, BRT002. A true recognition of our work."
Specifically, what will this designation bring to Nœtica Pharma?
A.M. :
"In the short term, it is a very positive message for potential investors: this designation constitutes a first recognition of our product by the authorities, it brings 'value' to the project. It also allows us to benefit from specific support.
In the long term, once we have obtained the marketing authorization for our drug, the orphan drug designation will allow us to benefit from a ten-year commercial exclusivity: during this period, the European Union and the member states will refrain from granting a marketing authorization or an extension of an existing marketing authorization for a similar drug. In other words, the designation prevents the marketing of another directly competing drug."
This text has been translated with the help of Meta AI.
Contact at the Frédéric-Joliot Institute for Life sciences: