Gene Therapy May Have Its First Blockbuster

May 24, 2019

(MIT Technology Review) – A newborn. A fatal diagnosis. And soon, a one-time gene replacement cure in the first weeks of life. The cost? You don’t want to know. Gene therapy is about to achieve a milestone. As soon as tomorrow, drug giant Novartis expects to win approval to launch what it says will be the first “blockbuster” gene-replacement treatment. A blockbuster is any drug with more than $1 billion in sales each year. The treatment, called Zolgensma, is able to save infants born with spinal muscular atrophy (SMA) type 1, a degenerative disease that usually kills within two years. But its expected cost is shocking, too: between $1.5 and $2 million, which would make it the most expensive one-time medicine ever sold.

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