Change.org - Most expensive treatment ever?

Spinal Muscular Atrophy (SMA) is a rare neuromuscular disorder. Sadly, babies born with SMA Type 1 often don’t live past 2 years old. But there’s hope - a one-time infusion called Zolgensma. It costs over $2 million, making it the most expensive single treatment. Ever. Petition starter Ewelina is fighting for the prices to be reduced so that “families around the world can afford this drug for their babies.” Sign now to help save babies with this fatal disease.  Novartis | Reduce $2​.​1M price of Life-Saving Drug, Zolgensma, for Spinal Muscular Atrophy! 6,221 have signed Ewelina Salaga’s petition. Let’s get to 7,500!

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Everyone should stand up for affordable access to lifesaving drugs!!! In May 2019, the Food and Drug Administration (F.D.A.) approved a gene therapy, Zolgensma, for Spinal Muscular Atrophy (SMA). SMA is a rare genetic disease that progresses rapidly in babies. If left untreated, even for days, SMA causes a permanent damage to babies. Zolgensma was developed by AveXis, Inc. and is sold by the Swiss drugmaker, Novartis. The drug is intended for babies under the age of two.Priced at $2.1 million per patient, Zolgensma is one of the most expensive drugs on the market. But given that the F.D.A. provided an accelerated approval process through Fast Track, Breakthrough Therapy, and Priority Review designations that saved hundreds of millions for Novartis, the drug price should have reflected that reality. Type 1, also known as the Werdnig-Hoffman disease, is the most common and aggressive form of SMA, affecting babies’ nerves and muscles. Babies with Type 1 do not produce enough protein because they are missing the SMN1 gene. They gradually die suffering grievously by being deprived of basic life functions like crawling, swallowing, and eventually breathing. Many newborns die within months, most other children do not live past age two. Therefore, the sooner the drug is administered, the better the results to maximize the outcomes of receiving the drug as Zolgensma does not reverse any damage already done. Zolgensma is simply out of reach for many suffering babies. Delayed accurate diagnosis and treatment leave parents with little time to find ample financial funds. While insurance companies may take the financial burden off parents in the U.S., at the end, we all pay this astronomical price for this drug. On the other hand, if parents are forced to raise money through crowdfunding organizations, out of the lack of available options, the treatment for babies is largely delayed. Even worse, some babies are left entirely out of the treatment! As of May 2021, Zolgensma will enter the third year of being sold at the $2.1 million price tag, unlike any other sophisticated drugs for rare diseases. Novartis AG, reduce the universal price of Zolgensma, by tying the safety and effectiveness of the drug to its pricing. Consider all generous incentives received by the F.D.A., not its fast-producing profit. Not only will that shorten the time of raising sufficient financial funds and assess the price of Zolgensma properly but will also save more babies!! SMA - BLOG (Part I) SMA - BLOG (Part II)   PLEASE SIGN, SHARE & DONATE.

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