Novartis | Reduce $2​.​1M price of Life-Saving Drug, Zolgensma, for Spinal Muscular Atrophy!

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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 drug price of $2.1 million for 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! According to ICER, "Zolgensma’s price for Type I patients would need to be between $710,000-$1.5 million per treatment." Once the price is reasonably reassessed, more countries will cover the treatment of Zolgensma ending ultimately the pain of afflicted families.

Expand Global Managed Access Program (MAP) beyond 100 free-of-charge doses per year to make Zolgensma available to eligible patients in countries where there is lack of such treatment. Include more countries in the program. Every month, there are 30 babies born with SMA in the U.S. Extrapolate that across the globe! SMA affects babies equally despite gender and race worldwide. There is a huge need for expanding the program especially in regions where treatment is out of reach.

SMA - BLOG (Part I)

SMA - BLOG (Part II)