FDA Approved $2.1 Million Medication to Treat a Genetic Disease

The Food and Drug Administration (FDA) has recently approved a new drug called Zolgensma, which can be used for treating spinal muscular atrophy in kids. It costs $2.1 million to purchase this medication, making it the most expensive option on the market.

Though many doctors and experts claim that this amazing drug is too expensive, parents of kids with spinal muscular atrophy might consider the new option as a miracle.

In kids with this disease, nerve cells in their spinal cords and brain stem that are responsible for controlling the muscles for swallowing, breathing, walking, and speaking are damaged. When the condition progresses, it will weaken muscles.

Patients will eventually lose the ability to do some normal tasks such as eat, walk, or breathe. This occurs quickly in infants born with the most serious kinds of the condition. Spinal muscular atrophy is affecting around 1 out of 11,000 kids.

Zolgensma is created by Novartis. Vas Narasimhan, the chief executive officer of the company said the price tag is justified.

“Critics don’t think about how the healthcare system is actually working,” he said. “We expend between $3 million and $5 million each patient for transplants, which is much less efficacy than this drug. We are attempting to price it at 50% of the current expense of care over 10 years for patients.”

Vas Narasimhan claimed that Zolgensma has been effective for every child who received this drug. “All 15 babies who participated in our studies are still alive and doing well.” said the CEO.

According to an epidemiologist, there is no incentive for pharmaceutical firms creating drugs for rare conditions to reduce the prices simply because they don’t have any competitors. These companies tend to charge as much as possible.

People will have no choice but to go for the drug. However, the government and insurance firms will eventually help them fund the huge expenses of treatments.

“I think that the firm is going to attempt and lobby to require insurance companies to pay for those medications. The advocacy community which is usually financed by the drug firms will push legislators to ask insurers for these treatments,” said an expert.

Vas Narasimhan also said that he hoped that insurance firms could cover the expense of treatment. But no matter how high the cost would be, most parents are willing to seek new options for their kids.

Source:

https://www.washingtonpost.com/business/economy/the-fda-approves-a-gene-therapy-that-is-the-most-expensive-drug-in-the-world/2019/05/24/57c66500-7e4a-11e9-8ede-f4abf521ef17_story.html?utm_term=.f9403c4c53e8

Below are other articles that you may be interested in:

Causes of Spinal Muscular Atrophy

Spinal Muscular Atrophy Symptoms

Spinal Muscular Atrophy – Diagnosis & Treatments

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