By Kelly Cloonan
Novartis has received approval from the European Commission for a treatment for certain patients with spinal muscular atrophy.
The pharmaceutical company said Thursday it got approval for Itvisma--a one-time, fixed-dose treatment--to treat those aged two years and older with 5q SMA with a bi-allelic mutation in the survival motor neuron 1 gene.
The approval makes Itvisma the first and only gene replacement therapy currently approved for that SMA population in the European Union, the company said.
Itvisma aims to address the genetic root cause of SMA and improve motor function. It is a new treatment option for this population, given it is a one-time fixed dose that doesn't need to be adjusted for age or body weight, while other available therapies tend to include ongoing dosing, the company said.
Write to Kelly Cloonan at kelly.cloonan@wsj.com