zolgensma, drug, expensive drug, spinal atrophy

The US pharmaceutical giant Novartis just got the world’s most expensive drug approved by FDA. Zolgensma, An effective drug to treat a type of spinal atrophy in infants is priced at a whopping $2.125 million.

The company will offer an annualized cost of $425,000 per year for five years. Novartis justified the price by saying the drug is really effective and will cut down the overall cost spent on medication of SMA.

The spinal atrophy is a hereditary neuromuscular disease. It is a progressive disease and the patient will loose muscle movements over time. Every year 400 children are born with this disease and those inflicted with the most severe type, do not live past 2 years or have to rely permanently on life support.

The disease is caused by a defect in a gene that makes SMN, a necessary protein for motor neurons. Zolgensma helps replace the defective gene with a functional copy to resume the production of SMN protein.

There are three types of spinal atrophy and the Zolgensma has been found to be effective for all. In the clinical trials, children under 2 years of age, who were injected with Zolgensma once, showed great improvement. The majority of young children diagnosed with muscle atrophy, lived after being treated with Zolgensma. They were able to breathe on their own, and showed continued improvement in motor movements, like sitting-up without support.

However, Biogene the producer of widely used spinal atrophy treatment Spinzara, doubt the effectiness of the Zolgensma. In their statement, they focused more on the fact that Spinzara has been used effectively for thousands of patients.

The patients being treated with Spinzara, require yearly spinal infussions, which cost $750,000 the first time and $375,000 thereafter.

In a short survey, 30% doctors currently treating SMA patients showed interest in using the world’s most expensive drug for their patient after a year of its launch. Doctors also showed interest in using the Zolgensma in combination of Spinzara.

Another pharmaceutical company Roche is also working on a daily pill to treat muscle atrophy that would reach market in 2020.

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