Posted by Michael Wonder on 14 Aug 2019
This drug will save children’s lives. It costs $2 million.
13 August 2019 - Safety, innovation and affordability need not be mutually exclusive goals for cutting-edge treatments.
Spinal muscular atrophy is a condition often fatal by age 2. Babies with the disorder have a rare genetic mutation that prevents the nervous system from controlling certain muscles. As they grow to be toddlers, these children struggle to move their limbs, to swallow food, to talk.
The Food and Drug Administration recently approved a single-dose gene therapy, Zolgensma, that has the potential to cure spinal muscular atrophy. That was cause for hope for the hundreds of patients suffering from it, along with their families and physicians — until its manufacturer, Novartis, announced the treatment’s price tag: about $2.1 million per patient.
