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Novartis’ Zolgensma starts strong as about 100 infants are treated with gene therapy

Sales of Novartis’ gene therapy for spinal muscular atrophy exceeded early forecasts, with about 100 infants treated in the drug’s first full quarter of commercial availability in the U.S. Zolgensma earned the Swiss pharma $160 million between July and September, a “big surprise” given Wall Street predictions of just over $100 million, Evercore ISI analyst Umer Raffat said Tuesday. The gene therapy has been closely watched since winning Food and Drug Administration approval in May to treat infants with the deadly inherited disease. While Zolgensma’s strong start boosts Novartis, the company has drawn criticism for pricing the therapy at $2.1 million, making it the most expensive drug ever launched. Novartis is also under fire for how its executives handled a data scandal after the FDA revealed the pharma submitted manipulated preclinical results in its regulatory application for Zolgensma.

Zolgensma (onasemnogene abeparvovec) and Spark Therapeutics’ Luxturna (voretigene neparvovec) are the first gene therapies commercialized in the U.S., the leading edge of what the FDA predicts will soon be a wave of cell and gene therapy approvals.

While the clinical science behind both is compelling, how well such expensive, one-time treatments sell remains a major question overhanging the broader gene therapy field.

Sales of Luxturna amounted to about $21 million over the first six months of 2019, while genetically engineered cell therapies from Gilead and Novartis have struggled commercially.

Zolgensma’s start should offer those in the field some measure of confidence, with Novartis CEO Vas Narasimhan saying the therapy has launched “with strong momentum.” The company’s focus is now on expanding newborn screening for SMA, and continuing to improve payer coverage — an early sticking point.

About 90% of commercial patients and 30% of Medicaid patients are currently covered, and Narasimhan said he expects public coverage to grow.

The FDA approved Zolgensma with a broad indication that covered all SMA patients less than two years old who had mutations in the SMN1 gene. So far, sales are evenly spread out between SMA types as well as by age.

Novartis aims to expand Zolgensma’s approval with clinical data in two- to five-year-olds. Interim results were presented earlier this month at the World Muscle Society’s annual congress, and showed a statistically significant increase in function that was nearly double the clinically meaningful threshold.

Narasimhan said data for the first two doses used in that study have been submitted to the FDA.

Zolgensma is not yet approved outside the U.S. Novartis anticipates regulatory decisions in Europe and Japan in the first three months of 2020 and the first half of next year, respectively.

While the vast majority of Zolgensma’s sales came from the U.S. market, Novartis has also worked with Germany, Portugal and France to create programs or approve early access for the gene therapy.