Nonprofit Organization Predicts Gene Therapy to be Most Cost Effective Treatment for Spinal Muscular Atrophy

According to a story from, a recent report from the Institute for Clinical and Economic Review (ICER) suggests that, pending more data about overall price and longterm success rates, an investigational gene therapy from Novartis AG could be a more cost effective treatment for spinal muscular atrophy in comparison to Spinraza. Spinraza is the only currently available therapy for the rare disorder.

About Spinal Muscular Atrophy

Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. There are a variety of management strategies for spinal muscular atrophy, but it is still usually fatal in its most severe forms. To learn more about spinal muscular atrophy, click here.

Still Not The Final Word

ICER’s assessments appear to have some significant sway in determining how new drugs get priced. The organization utilized a measure known as quality-adjusted life year in its calculations which compared Spinraza and the gene therapy. Regardless, these findings are not final, as upcoming announcements from drug makers, more clinical trial results, and feed back from patients and insurers could result in revisions to the report, the final version of which will be released late next month. 

Both Spinraza and the experimental gene therapy have displayed meaningful effectiveness in treating spinal muscular atrophy. Spinraza is an extremely expensive drug, costing $750,000 for the first year of treatment. The new gene therapy could cost even more once it gets approved, but it is also intended as a curative, one time treatment.

A representative from Biogen, the company that developed and manufactures Spinraza, pushed back on the claims made in the report, saying that that calculations downplay Spinraza’s overall impact and that it is misleading to compare the results of small scale clinical trials to the real-world impact that Spinraza has had on thousands of patients.

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