At $2.1M, Novartis gene therapy will be world’s most expensive drug

Known as Zolgensma, the gene therapy treats children under 2 years of age with spinal muscular atrophy, an inherited neuromuscular disease that causes progressive loss of muscle function. To achieve commercial success, Novartis must persuade doctors who treat SMA patients that the muscle-preserving benefits from a one-time injection of Zolgensma will be durable. On a recent conference call, executives argued that even with Zolgensma’s arrival, Spinraza remains the standard of care treatment for SMA, based on the drug’s broader label and more than 7,000 patients treated, some for as long as six years.

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