The Japanese Ministry of Health, Labour and Welfare recently approved Zolgensma, Novartis' gene therapy drug for the treatment of spinal muscular atrophy, for infants under the age of 2, according to Medical Dialogues.
Five things to know:
1. Zolgensma was developed by AveXis, a biotechnology company acquired by Novartis for $8.7 billion in 2018.
2. The drugs is priced at $2.1 million and offers a one-time treatment for the rare neurological disease.
3. About 400 patients have been treated with Zolgensma so far.
4. Fifteen to 20 SMA patients in Japan are expected to be eligible for treatment annually.
5. Novartis is pursuing registration in more than 30 countries, with regulatory decisions anticipated in Switzerland, Canada and Australia in late 2020 or early 2021.