Sales of Novartis’ gene therapy Zolgensma declined in the third quarter as use of the medicine has mostly shifted to infants newly diagnosed with the neuromuscular disease it treats.
Between July and September, global sales totaled $319 million, down 16% from the previous quarter and 15% from the same period last year, according to financial numbers disclosed by Novartis Tuesday. Both the U.S. and non-U.S. markets now predominantly involve “incident” disease treatment, rather than of already diagnosed patients, Novartis said.
“Zolgensma has had a little bit of a challenging quarter,” said Novartis CEO Vas Narasimhan in a Tuesday call with analysts.
Approved in 45 countries, Zolgensma replaces the faulty gene responsible for causing spinal muscular atrophy, a condition that in its severe form robs infants of the ability to stand, sit and, eventually, breathe. Untreated, it’s typically fatal by a very young age.
Zolgensma can deliver dramatic benefits, keeping children alive and in some cases helping them achieve motor and developmental milestones they otherwise wouldn’t. In the U.S, its use is limited to babies under the age of two, where it’s seen as having the greatest benefit. Two other drugs, from Biogen and from Roche, are also approved for the disease, but can be used in older children and adults.
Over time, many of the already diagnosed patients eligible for Zolgensma have received treatment — a pattern that’s played out elsewhere, too.
“As we add markets, we initially penetrate a bolus of patients and then we move back to the incident patient population,” said Narasimhan, on Tuesday. “In the incident patient population, demand driven is by expanding newborn screening, particularly outside of the U.S.”
In Europe, for example, Novartis said newborn screening is less common for spinal muscular atrophy than it is in the U.S.
Negotiations over reimbursement of Zolgensma are also continuing in 10 of the 45 countries where the gene therapy is approved, including Brazil, India, Turkey and Saudi Arabia. While designed to be given just once, the treatment is very expensive. In the U.S., Novartis priced an infusion at $2.1 million, making it one of the priciest medicines on the market.
Zolgensma has become one of Novartis’ top-sellers, generating more than $1.3 billion in annual sales last year. The company expects it to eventually earn between $1.5 billion and $2 billion annually. To get beyond that, Narasimhan said Novartis will need to win approval of a formulation that’s injected into the spine and can be used for older patients. The company has hit roadblocks developing that version, however.
Novartis is also competing with Biogen and Roche, which sell Spinraza and Evrysdi for spinal muscular atrophy, respectively. Sales of Spinraza were $431 million in the third quarter, Biogen reported Tuesday, while Roche revenue from Evrysdi totaled 293 million Swiss francs, or about $294 million.