FDA Approves $2.1M Gene Therapy Treatment For Rare Muscle Disease

The single-dose treatment is the most expensive medicine ever.
Posted at 6:42 PM, May 24, 2019

On Friday, The Food and Drug Administration approved the first drug to treat a rare, fatal genetic disease. Babies with spinal muscular atrophy, or SMA, experience extreme weakening of muscles — the worst cases result in death before age 2. The gene therapy, Zolgensma, is priced at $2.1 million, making it the most expensive medicine ever. 

Because of the huge price tag, critics say insurance companies and the government will have to help finance it. Novartis said it will provide patient support and payment programs to help patients get the treatment. 

In a statement, Acting FDA Commissioner Ned Sharpless said, "The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future."

According to Novartis, SMA affects approximately 1 in 10,000 "live births." It's caused by a mutation in the survival motor neuron 1 (SMN1) gene.  Zolgensma is designed to replace that defective or missing gene and stop the disease with a single dose.