Novartis gene therapy brings in $160 million, beating expectations – STAT

Zolgensma, a lifesaving treatment for infants and the worlds most expensive drug, has been used to treat 100 patients since its launch and brought in $160 million for its maker Novartis (NVS) last quarter, beating analysts expectations.

The gene therapy is a treatment for spinal muscular atrophy, a rare and devastating neurological disease. It carries a record price tag of $2.1 million, or an annualized cost of $425,000 per year for five years.

Novartis said Tuesday that roughly 99% of SMA patients who qualified for Zolgensma got coverage of the one-time therapy, although some had to go through an approval process to receive the drug. The company also indicated that it made progress in striking deals with health plans to cover the drug, saying that agreements are in place covering roughly 90% of patients insured commercially and roughly 30% of patients covered by Medicaid.

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I think the overall uptake that were seeing very early in the launch speaks to the promise of the product, the physician excitement, and the parent and patient desire to get kids on gene therapy, said Dave Lennon, president of AveXis, Novartiss gene therapy business.

The strong sales numbers came over a summer that saw a controversy involving manipulation of data used to support Zolgensmas approval by the Food and Drug Administration. In an unusual rebuke, the agency said in August that AveXis knew that preclinical data had been falsified before the drug was approved in May, but did not inform the Food and Drug Administration until later. The agency said that the drug should stay on the market, but the scandal sparked anger from lawmakers and a pledge from Novartiss CEO, Vas Narasimhan, to move more quickly on disclosing issues around data integrity.

Lennon said Novartis worked quickly to reassure patients and physicians of Zolgensmas quality, and the data manipulation scandal had no real impact on the treatments commercial performance, Lennon said.

Zolgensmas early success could be a worrying sign for Biogen, whose Spinraza has been the treatment of choice for SMA since its approval in 2016. More than 50% of patients treated with Zolgensma had switched over from Spinraza, according to Novartis, preferring a one-time therapy over Biogens every-four-months treatment.

Zolgensmas sales outstripped what analysts had forecasted. One average of analysts projections, from Bloomberg, had pegged the drugs sales for the quarter at $86 million. Another compiler of financial estimates, FOA, had gauged analysts consensus at $106 million.

Novartis expects revenue to grow. The company is counting on approvals for Zolgensma in Europe and Japan next year, and it recently presented data demonstrating the gene therapys effects on older SMA patients. Novartis is also counting on an expansion of newborn testing for SMA, which is currently done in 30% of U.S. states but could rise to 70% by 2020, according to the company.

Time is neurons for these kids, and we really want to make sure they get gene therapy as soon as possible, Lennon said.

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Novartis gene therapy brings in $160 million, beating expectations - STAT