- Swiss drugmaker Novartis has won a conditional approval from the European Commission to sell Zolgensma, its pricey gene therapy for spinal muscular atrophy, in Europe.
- The approval covers most infants and children with SMA who weigh up to 46 pounds, a label that would include almost all patients under the age of five, Novartis in a May 19 release. By contrast, Zolgensma is only approved for SMA patients aged two or younger in the U.S.
- Between 550 and 600 babies are born with SMA every year in Europe, Novartis said. The rare, inherited disease results in the loss of critical motor neurons — severely affecting movement, breathing and swallowing — and is the leading genetic cause of infant death.
Novartis is counting on launches in Europe and Japan to drive the "next sales ramp" for Zolgensma, CEO Vasant Narasimhan told investors on an April 28 conference call. The one-time treatment, priced in the U.S. at $2.1 million, brought in sales of $170 million for Novartis in the first quarter, slightly lower than the $186 million posted in the fourth quarter.
The next step for Novartis will be negotiating reimbursement with EU governments and agencies. While decisions on pricing and reimbursement will be made at the local level, Novartis said it plans to offer the treatment under its "Day One" access program, which allows for retroactive rebates, deferred payments and outcomes-based rebates after treatment.
Patients in France can get immediate access to Zolgensma through a temporary authorization program, and patients in Germany should soon be able to get the therapy as well, Novartis said.
So far, Novartis has had success in winning over insurers. In the U.S., where Zolgensma is approved for children under the age of two, almost all patients who fall within the label's guidelines have won approval from payers for access, Novartis said.
The Swiss drugmaker argues that the hefty price tag for the medicine provides value for insurers and health-care systems. In Europe, for instance, Novartis claims the cost of care per patient can exceed $4 million over the first 10 years of life, if infants survive what is typically a deadly disease in the most severe form.
Zolgensma is meant to halt the progression of the disease by delivering a functional copy of the SMN1 gene, which is defective in SMA patients.
As Novartis gets ready to treat new pools of patients, it's also working to ramp up manufacturing capacity. It's seeking regulatory approval for two new plants to produce Zolgensma, adding to a facility in Libertyville, Illinois, that can make some 700 to 800 doses a year.
The latest clearance includes all 27 European Union nations, plus Iceland, Norway, Liechtenstein and the U.K. Zolgensma won approval in Japan in March.
With the European approval, more families with infants diagnosed with SMA should soon be able to access Zolgensma through normal channels.
After the Food and Drug Administration cleared Zolgensma last year, Novartis had stirred up controversy with a global access program for SMA patients in countries in which the therapy was not yet approved. Set up like a lottery scheme, the program randomly allocates doses of Zolgensma for participating patients under the age of two with genetically confirmed spinal muscular atrophy. Patients would then receive the therapy free of charge.