Zolgensma, Novartis' gene therapy drug for spinal muscular atrophy, recently demonstrated significant therapeutic benefits for patients in various extended clinical trials, reports Pharmaceutical Technology.
The new data highlighted advantages for patients treated presymptomatically and showed sustained durability in patients up to five years post-dosing.
The trial results are expected to boost Zolgensma's sales and grow its patient pool.
Zolgensma is anticipated to lead the SMA market and reach $2.5 billion in sales by 2025, according to GlobalData.
Priced at $2.1 million, Zolgensma offers a one-time treatment for SMA. The drug secured $186 million in fourth quarter sales for Novartis, with full-year 2019 sales totalling $361 million.
Novartis is pursuing regulatory approval in more than 30 countries.
The company recently secured approval for the drug in Japan and expects decisions to be made by Swiss, Canadian and Australian authorities in late 2020 or early 2021.