Theranexus climbs over 22% on the Paris Bourse on Wednesday, after receiving a positive opinion from the European Medicines Agency (EMA) on 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 US FDA in May.

In this case, the same efficacy criteria were retained by the EMA, namely visual acuity as the primary endpoint in phase 3, and assessment of cognitive and motor functions as secondary endpoints.

The juvenile form of Batten disease, a rare genetic disorder of the nervous system for which there is no treatment, manifests itself through progressive loss of vision, which appears between the ages of four and six.

This is followed by behavioral disorders and learning difficulties, then convulsions. Eventually, patients become wheelchair-bound, or even bedridden, before dying prematurely.

Patient recruitment should begin by the end of 2023 in several centers in the USA and Europe, with the aim of achieving first sales as early as 2027.

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