Novartis Therapy For Infant Muscle-Wasting Disease — Potentially Carrying A Record $2 Million Price Tag — Is About To Hit Market

Denise Roland, The Wall Street Journal

May 7, 2019

Official price has yet to be set for the gene therapy Zolgensma as insurers, drugmakers grapple with new payment models

Zolgensma is expected to go on sale soon, with an FDA decision due this month. The treatment tackles spinal muscular atrophy, whose sufferers lack a gene essential for muscle control. Without treatment, victims of the most severe form typically die before their second birthday, making SMA the most common genetic cause of infant death.

A new treatment for an infant muscle-wasting disease is about to go on sale at a potential cost of $2 million, a record price tag likely to fuel the continuing scrutiny of how companies price their drugs and how insurers pay for them.

Novartis AG NVS, -2.05% has yet to set a price for the gene therapy called Zolgensma, but executives say the drug’s potential to cure spinal muscular atrophy, an inherited disease that typically kills babies before they turn two, justifies a seven-figure price.

Gene therapies target diseases that result from a faulty gene by introducing a working version into the body. They are attracting interest, both for their ability to cure otherwise devastating illnesses in one treatment and also for their high cost. Luxturna, the only gene therapy on sale in the U.S. so far to treat a form of inherited sight loss, costs $850,000 a patient.

The issue is gaining in importance as more gene therapies go on sale. The Food and Drug Administration expects to approve 10 to 20 gene and cell therapies a year by 2025

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