FDA: Approve Vyondys 53 (Golodirsen) for the treatment of Duchenne Muscular Dystrophy

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On Monday, August 19th, 2019 the United States Food & Drug Administration (FDA) denied Vyondys 53 (Golodirsen) for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal disease (most patients die in early adulthood). Golodirsen is essentially the same as Eteplirsen (approved in 2016 under Accelerated Approval) but skips exon 53 rather than exon 51.

As noted by Sarepta Therapeutics (2019), the FDA’s response “…cites two concerns: the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of golodirsen and observed following administration of other antisense oligonucleotides.” (para. 2)

The issue of risk of infections with IV infusions is unwarranted as countless IV medications have been approved for many conditions. With any IV infusion, there is an infection risk, but the drug itself does not cause the infection.

The issue raised about renal (kidney) toxicity is questionable as this wasn’t seen in the Phase I/II Study of SRP-4053 (Vyondys 53) in DMD Patients. The renal toxicity was only seen in mice with a dose ten times higher than what a human would take.

I am urging the FDA to reverse the denial and approve Vyondys 53 as soon a possible to give people with DMD a chance to live a long and fulfilling life. Many patients need the drug now and cannot wait for years or even months. No one deserves to die at a young age from a fatal condition. This denial violates the 21st Century Cures Act, which ensures that the patient's voice is incorporated into the drug development process. According to the Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA must "...solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions." (GPO, 2012, p. 132) Despite these regulations, the FDA refused to have an advisory committee meeting for those with DMD to provide their perspectives which they allowed with Eteplirsen.

 

According to the NIH Genetic and Rare Diseases Information Center (2017), DMD is: “…a progressive form of muscular dystrophy that occurs primarily in males. DMD causes progressive weakness and loss (atrophy) of skeletal and heart muscles. Heart and respiratory muscle problems begin in the teen years and lead to serious, life-threatening complications.” (para. 1)

About me: My name is Jacob Hill, and I’m 20 years old. I am currently a student pursuing a bachelor’s degree in Operations & Supply Chain Management. I hope to graduate by 2021 and then pursue a career as a Procurement Manager or Supply Chain Manager at a large corporation such as Amazon. I hope to become a Chief Operating Officer (COO) eventually. I choose not to let DMD get in the way of my goals. I have a twin brother who also has DMD.

References:

Government Publishing Office (GPO). (2012, January 3). Food and Drug Administration Safety and Innovation Act. Retrieved from https://www.govinfo.gov/content/pkg/BILLS-112s3187enr/pdf/BILLS-112s3187enr.pdf

NIH Genetic and Rare Diseases Information Center. (2018, September 28). Duchenne muscular dystrophy. Retrieved August 21, 2019, from https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy#ref_13902

Sarepta Therapeutics. (2019, August 19). Sarepta Therapeutics Receives Complete Response Letter from the US Food and Drug Administration for Golodirsen New Drug Application. Retrieved August 21, 2019, from https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-receives-complete-response-letter-us-food