Novartis' gene therapy Zolgensma costs $2.1 million per patient. But that record price tag hasn't impeded the Swiss drugmaker from recording a strong commercial launch for the one-time treatment in the U.S.
Sales numbers released Wednesday show Zolgensma, which was approved last year for the fatal neuromuscular disease spinal muscular atrophy, earned $186 million in the fourth quarter. Another 100 infants received the therapy in the three months from October through December, bringing the total number treated in 2019 to about 200.
Zolgensma's commercial performance to date should offer some reassurance to investors that Novartis can successfully sell the gene therapy, which poses unique challenges to a system built around drugs for chronic diseases. Not only is Zolgensma's cost dramatically higher than typical, its value is tied to its potential to be a genetic cure for a disease that typically leads to death before the age of two.
So far, however, insurers are willing to cover Zolgensma's cost. According to Novartis, nearly all eligible patients with spinal muscular atrophy are covered for treatment with the therapy. Roughly half of infants who are insured through Medicaid are covered, an improvement from the 30% or so Novartis reported at the end of September.
Payers have reimbursed treatment for all eligible infants who have received Zolgensma but one, according to comments by company CEO Vas Narasimhan at the recent J.P. Morgan Healthcare Conference.
Newborn screening for spinal muscular atrophy has also improved, which AveXis President David Lennon told BioPharma Dive earlier this month would be a key priority for expanding access to Zolgensma.
Novartis expects to treat about 100 infants per quarter until Zolgensma receives approval in Europe, which would further expand demand, said Narasimhan on a conference call Wednesday. A recommendation from the European Medicines Agency on market authorization is expected this quarter.
Novartis is also working to resolve a clinical hold placed by the Food and Drug Administration on testing of a spinal injection formulation of Zolgensma, which is designed for patients older than those who can receive the currently approved infusion version.
Narasimhan told investors Wednesday the company plans to submit data to the FDA in the coming weeks that it hopes will address the regulator's concerns and allow for a submission of the intrathecal dosing this year.
Zolgensma's launch is also looked to as a barometer for the gene therapy field as a whole, which has impressed with compelling benefits in diseases like hemophilia and muscular dystrophy but remains largely untested commercially.
Roche, Pfizer and Astellas have also made major bets that gene therapies can become profitable businesses, as have biotech companies like Sarepta Therapeutics, BioMarin Pharmaceutical and UniQure. Sales of Zolgensma, which totaled $361 million in 2019, suggest those bets might not be misplaced.
Novartis' results could have implications for Biogen, which sells the competing spinal muscular atrophy drug Spinraza, and for Roche, which aims to soon win approval for an oral drug for the condition. Both companies will deliver business updates on Thursday.
While Wednesday's results should ease worries about Zolgensma's sales potential, Novartis is still awaiting the judgment of the FDA on whether penalties will be levied for the company submitting manipulated preclinical testing data to the agency.