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FDA Approves Most Expensive Drug Ever - And It's Exclusively For Babies

The United States' Food And Drug Administration approved a new gene therapy last month to treat a genetic condition that impacts newborn babies. This novel treatment, called Zolgensma, is the most expensive drug to ever be produced, checking in at a whopping $2.125 million dollars per infusion! Considering that the average drug trial costs the manufacturer $19 million, Novartis may be pricing their product to recoup that research & development investment. Zolgensma is only the second treatment on the market for spinal muscular atrophy, or SMA. The first treatment, Spinraza, was released in 2016.

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Via MDA

What Is Spinal Muscular Atrophy?

SMA is the leading genetic cause of infant death. The disease manifests in several different forms: Types I, II, III, & IV. Type I is the most severe form of SMA; it presents within the first six months of life and is a terminal disease. Ninety percent of children with SMA Type I don't live to see their second birthday. About sixty percent of SMA cases are classified as Type I.

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Spinraza, the treatment approved in 2016, can treat any of the four types. However, Zolgensma is designed specifically to treat SMA Type I and is only available to patients two years of age or younger.

“Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases." - Acting FDA Commissioner Ned Sharpless, M.D.

Available SMA Treatments

Currently, the FDA has only approved two treatments for SMA - Spinraza and Zolgensma. Several pharmaceutical companies have SMA treatments in development, though only two therapies are in the final phase of the drug development lifecycle. Spinraza, a Biogen product, costs $125,000 per injection. The first year of treatment costs $750,000 and every year thereafter, $375,000. Since patients have to continue Spinraza throughout their life, the treatment could easily cost more than Zolgensma's $2,125,000.

Via Fox4

Zolgensma may have a shocking price tag, but Novartis has established a payment plan. The infusion can be paid over five years, at a cost of $450,000 per year. This payment plan puts Zolgensma in direct competition with Spinraza, especially for those ages two and under. Luckily, the Zolgensma treatment is being touted as a potential lifetime cure! The patients from the clinical trial have been asymptomatic since their treatment, about five years. Hopefully, Zolgensma will hold true and be the treatment that babies with SMA need to survive.

Why Does Zolgensma Matter?

My friends Gloria and Steven welcomed their first child, Isaac, on May 24th, 2014. Newborn Isaac was tiny and sweet and adorable. His toothless grin lit up the room! Unfortunately, Isaac was soon diagnosed with SMA Type I. Steven and Gloria decided they would try anything to help Isaac, even though they knew the disease was terminal. I got to meet Isaac when Gloria and her mom came to my city. They weren't vacationing; Isaac was part of a clinical trial for a treatment for his SMA.

Before Isaac was diagnosed, I had never heard of SMA. Seeing Gloria, Steven, & Isaac face this unfathomable medical tragedy was heartbreaking. Issac Nathaniel Kimmel passed away on July 31, 2015. He was just over fourteen months old. Isaac's participation in the clinical trial means that his future siblings, who might have SMA themselves, will have a fighting chance to live a long and healthy life.

Gloria & Steven went on to have a second child - their daughter Claire. They included a photo of their commemorative tattoos for Issac in Claire's newborn photo shoot. The photo went viral. (Photo Credit To Megan Miles Photography)

"This photo is our first family photo with all of us together. Our son is there in that photo with us, through our memorial tattoos. It is a beautiful reminder of his life and loss, and also one of hope and love. A testament to our past and our joy for the future. So very blessed to have this photo to remember him by and to welcome our daughter at the same time." - Gloria Kimmel

Issac is not the only child to have lost his life to SMA Type I. Hopefully, Zolgensma will make his death one of the last.

SOURCE: FDA Approves Innovative Gene Therapy

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