In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the Japanese Ministry of Health, Labour and Welfare, allowing patients in Japan the same access to this novel treatment.
AveXis is working to get this therapy approved in 3 dozen nations across the world. This year, they expect decisions from Canada and Switzerland. They expect Australia’s decision to come soon after.
In the United States, 400 patients under 2 years old who have SMA with mutations in the SMN1 gene have been treated with Zolgensma. 60% of all those with SMA have the Type 1 form of the disease. Zolgensma is a gene therapy which works to replace the malfunctioning SMN1 gene. All it requires is a singular, one-time IV infusion.
Approval in Japan
In Japan, Zolgensma has been approved for all SMA patients under 2, even those who are still asymptomatic. To qualify for the therapy, patients must not have elevated levels of anti-AAV9 antibodies.
It is estimated that 15 to 20 patients will be eligible to receive the therapy each year in Japan. As SMA, when untreated, is the leading genetic cause of death in infants, this treatment could be revolutionary.
Approval came about as a result of 4 clinical trials all evaluating the safety and efficacy of the therapy. Survival rates for participants were unseen in the history of SMA. Zolgensma was also found to improve rapid motor function. Patients were able to sit by themselves, something never before seen in patients who aren’t provided this therapy.
Researchers emphasize the importance of diagnosing SMA early, and beginning treatment as soon as possible. This therapy can stop motor neuron loss, which is irreversible, and stop the disease from progressing. Hopefully, we will continue to see this therapy become more and more widespread, supporting patients throughout the globe.
You can read more about this therapy here.