(CercleFinance.com) – Theranexus climbed more than 22% on Wednesday on the Paris Stock Exchange after obtaining the positive opinion of the European Medicines Agency (EMA) concerning the design of its phase 3 study in Batten disease .
The biopharmaceutical company and its partner in the trial, the Beyond Batten Disease Foundation (BBDF), emphasize that the EMA’s response is in line with the terms of the agreement already obtained from the FDA American in May.
In this case, the same efficacy criteria were retained by the EMA, namely visual acuity as the main criterion for phase 3 and the evaluation of cognitive and motor functions as secondary criteria.
The juvenile form of Batten disease, a rare, genetic disorder of the nervous system for which there is no cure, is manifested by progressive loss of vision, which appears between the ages of four and six.
Behavioral disorders and learning difficulties follow, followed by seizures. Eventually, patients move around in a wheelchair, or even remain bedridden, before dying prematurely.
Patient recruitment should start by the end of 2023 in several centers in the United States and Europe, with the objective of achieving first sales in 2027.