New Spinal Muscular Atrophy Gene Therapy to Have Access Programs

According to a story from PR Newswire, the Novartis Company AveXis has issued an announcement detailing special access programs that will help patients access Zolgensma, the first ever single use gene therapy for spinal muscular atrophy which was recently approved by the US Food and Drug Administration (FDA). The company plans to work closely with US payers as well as implement patient programs that will improve access to the treatment, which is extremely expensive.

About Spinal Muscular Atrophy (SMA)

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. The most effective treatment currently available for the disease is a single use gene therapy called Zolgensma. To learn more about spinal muscular atrophy, click here.

Paying for Zolgensma

There is no doubt that Zolgensma is a major breakthrough for the treatment of spinal muscular atrophy, and over time the drug will become more cost effective when compared to Spinraza, which is the only other disease modifying therapy on the market for the disease. Unlike Zolgensma, Spinraza must be taken indefinitely. Novartis has priced the drug to be 50 percent less expensive than ten years of treatment with Spinraza. This gives Zolgensma a price tag of over $2 million.

The company is prepared to offer five year pay-over-time options and other outcome based agreements to payers. Such an agreement with self-insured employers, states, and small payers can help alleviate budgetary strains that such an expensive drug can bring. 


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