World's most expensive drugs may be a stunning breakthrough
Zolgensma. That's the name of a drug on the forefront of a new wave of gene therapy drugs that don't address symptoms — they cure the problem by rewriting the very genetic code of a patient.
Zolgensma is believed to be a one-treatment cure for a deadly genetic disease that kills by causing motor neurons to die in the spinal cord. Spinal Muscular Atrophy takes away the ability to breathe, swallow, eat or walk.
About 1 in 11,000 babies are born with SMA and it is caused by a mutation in one single motor neuron called SMN1.
It turns out that the simplicity of the disease has made it possible to cure through the fledgling area of medicine called gene therapy.
The cure is new — and it is horrifically dear. A single IV dose of Zolgensma costs $2.125 million — the most expensive drug ever created.
Scientists wanted to put a healthy SMN1 gene into the motor neurons of SMA patients. To do that, they created a virus that would house the healthy SMN1 gene, and infect the patient's nervous system with the healthy gene. The treatment is administered through IV.
The first trials showed extraordinary results and led to FDA approval. Every one of the first 15 infants who received the treatment were alive and symptom-free at age 20 months. Compare that to children who have historically had SMA where the survival rates during a similar period were 8 percent.
What remains to be seen is how the children fare over time, whether they will be able to reach adulthood and how long their lives will be extended, according to the American Council on Science and Health.
A second drug called Spinraza — also a gene therapy — costs $750,000 for the first year injection and $375,000 every year after that. Over 10 years, there could be some savings with Zolgensma, which is thought to be needed only once.