The second gene therapy to win approval in the U.S. saw strong sales in the third quarter of this year, the manufacturer said Tuesday.
Basel, Switzerland-based Novartis said in its third-quarter 2019 earnings that Zolgensma (onasemnogene abeparvovec-xioi) had sales of $160 million. The gene therapy was approved in May for treating spinal muscular atrophy in infants, being the second gene therapy to win Food and Drug Administration approval, after Spark Therapeutics’ Luxturna (voretigene neparvovec), which Novartis commercialized in the European Union and European Economic Area.
The gene therapy was launched with a list price of $2.1 million, the highest list price of any drug in the world. However, pricing watchdogs have said the price is cost-effective, given the greater costs of treatment over a patient’s lifetime, whereas Zolgensma is meant as a one-time therapy. A competing therapy is Biogen’s Spinraza (nusinersen), which has a list price of $750,000 for the first year and $375,000 every subsequent year, but must be taken for life, at $125,000 per injection.
Novartis said in its earnings statement that to date, plans are in place that pay for the therapy for around 90 percent of patients covered under commercial plans and about 30 percent of those covered by Medicaid.
Spinal muscular atrophy, or SMA, is a group of genetic diseases that cause progressive muscle weakness, usually starting in infancy, which can eventually lead to respiratory failure. The incidence of the disease is approximately 1-in-10,000 live births, according to the National Organization for Rare Disorders.
Other new therapies from the company had a strong showing as well. Another drug that received FDA approval in May, Piqray (alpelisib), saw sales of $43 million. The drug was approved as the first treatment for patients with a PIK3CA mutation in HR-positive/HER2-negative advanced breast cancer. Kymriah (tisagenlecleucel), which won FDA approval in August 2017 as the first CAR-T cell therapy, had sales of $79 million. The company reported continued strong demand, with ongoing uptake in the U.S. and Europe driving an increase in sales for the therapy, which is used to treat acute lymphoblastic leukemia in children and young adults as well as diffuse large B-cell lymphoma in adults.
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