Zolgensma Use Strong with About 100 Children Treated Since US Approval, Novartis Says

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by Mary Chapman |

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Zolgensma guidance

With about 100 spinal muscular atrophy (SMA) patients already treated with Zolgensma (onasemnogene abeparvovec-xioi) since the gene therapy’s May approval in the United States, and expected growth in newborn screening, the groundbreaking treatment that is also the world’s most expensive is off to a robust start, Novartis said.

More than half of the children treated with Zolgensma — the first gene therapy for SMA or any chronic neurologic disease — switched from Spinraza (nusinersen), the first disease-modifying treatment approved for SMA by the U.S. Food and Drug Administration, the company stated in an October investor report.

Exceeding analysts’ expectations, sales of this treatment helped boost third-quarter results for Novartis, the company that owns AveXis, which developed Zolgensma (formerly known as AVXS-101). The list price is $2.1 million for the potentially curative, one-time intravenous infusion treatment approved for children up to age 2.

“There has been strong and high interest in the product across the country,” Vas Narasimhan, Novartis’ CEO, was quoted as saying by Bloomberg. The company reported that sales of Zolgensma brought in $160 million last quarter (Q3 2019) alone.

Most SMA patients have been able to get Zolgensma through insurance. According to the investor report, current plans cover about 90 percent of commercially insured patients in the U.S. Roughly 30 percent of those enrolled in the Medicaid program are also covered, with more states, including Florida, New Jersey and Michigan, expected to provide coverage soon.

The company allows payments over time, a suggestion that hasn’t caught on, Narasimhan said. And while it offers to do so, Novartis has not needed to reimburse for ineffective treatment, he added. More than 50 U.S. sites administer Zolgensma, with even distribution across all age and disease types.

Novartis said newborn screening programs will help in identifying and treating more newly diagnosed children. Currently, 30% of newborns in the United States are screened for SMA, a figure the company expects to rise to 70% by year’s end. There also are stepped-up plans globally to screen for the disease.

About 100 children are diagnosed with the disease every three months, and Novartis expects to provide Zolgensma to 90 of them, on average, the company was reported to say.

The investor report also noted the promising results reported earlier in the ongoing STRONG Phase 1/2 clinical study (NCT03381729), which is evaluating Zolgensma’s safety, tolerability and efficacy in SMA type 2 children up to 5 years old treated by intrathecal (spinal canal) injection.

As for availability abroad, Novartis said delays in Japan and Europe are due to additional questions from regulators about manufacturing. By the first quarter of next year, a decision is expected from the Committee for European Medicinal Products for Human Use (CHMP), the European Medicines Agency branch that judges whether a medicine should be approved for the European Union. The company is expecting a decision in Japan by mid-year.

Early access programs are also in place in a number of countries, including France, Germany and Portugal.

Narasimhan said these delays are unrelated to a federal investigation into the company’s handling of data from early animal studies in Zolgensma. Novartis has been criticized for not disclosing data irregularities to the FDA prior to the medicine’s U.S. approval.