Zolgensma, a gene therapy drug for spinal muscular atrophy, has been on the market since June 2019 and is widely reported to be the most expensive drug in the world.
10 things to know:
1. SMA is a rare genetic disease that leads to progressive muscle weakness and, in some cases, paralysis. The FDA approved the drug for infants under the age of 2.
2. Zolgensma was developed by AveXis, a biotechnology company acquired by Novartis for $8.7 billion in 2018.
3. Priced at $2.1 million, Zolgensma provides a one-time treatment for the rare neurological disease.
4. Zolgensma replaces the defective or missing SMN1 gene to halt disease progression with a single one-time infusion, administered to patients over one hour.
5. The drug brought in $186 million in fourth quarter sales for Novartis. Total sales for 2019 were $361 million.
6. The sales suggest that Zolgensma's high cost isn't preventing patients from accessing it. However, it is ultimately paid for through insurance premiums and by taxpayers.
7. Reimbursement agreements have been established with payers to cover 97 percent of patients with commercial plans and 50 percent of patients covered by Medicaid, according to Novartis.
8. About 200 patients have been treated with Zolgensma since its launch. Novartis said there are about 700 patients eligible for the treatment and approximately 30 babies born with SMA each month in the U.S.
9. In January, New Jersey Democrat Gov. Phil Murphy signed legislation requiring newborns to be screened for SMA. More states are anticipated to follow suit, with a similar bill introduced in Nebraska in January.
10. Currently, the only other treatment for SMA is Spinraza, which is developed by Ionis and marketed by Biogen. The FDA approved Spinraza in 2016, which costs $750,000 for the first year and $350,000 each year afterwards.