Eteplirsen, a drug used for the treatment of Duchenne muscular dystrophy (DMD), was approved by the U.S. Food and Drug Administration (FDA) on September 19, 2016 [3]. This approval marked a significant milestone as eteplirsen became the first drug to receive accelerated approval for the treatment of DMD [3]. The drug is marketed under the tradename Exondys 51 [1].
DMD is a rare genetic disorder that primarily affects boys and leads to progressive muscle weakness and loss of function [2]. Eteplirsen works by targeting a specific genetic mutation that results in the production of a defective protein called dystrophin, which is crucial for muscle function [2]. By enabling the production of a truncated but functional form of dystrophin, eteplirsen aims to slow down the progression of the disease and improve muscle strength in DMD patients [2].
The approval of eteplirsen was based on data from a clinical trial that showed an increase in dystrophin production in some patients who received the drug [3]. However, it is important to note that the accelerated approval pathway allows for the approval of drugs based on surrogate endpoints, such as dystrophin production, that are reasonably likely to predict clinical benefit [3]. Continued monitoring and further studies are required to confirm the clinical benefit of eteplirsen [3].
In conclusion, eteplirsen was approved by the FDA on September 19, 2016, making it the first drug to receive accelerated approval for the treatment of Duchenne muscular dystrophy [3]. This approval offers hope to patients with DMD and their families, but ongoing research and monitoring are necessary to fully understand the drug's long-term efficacy and safety.
Sources:
[1] DrugPatentWatch. (n.d.). Eteplirsen - Generic Drug Details. Retrieved from https://www.drugpatentwatch.com/p/tradename/ETEPLIRSEN
[2] McDonald, C. M., & Campbell, C. (2018). Eteplirsen for the Treatment of Duchenne Muscular Dystrophy. Human Gene Therapy, 29(12), 1435-1445. doi:10.1089/nat.2018.0756
[3] U.S. Food and Drug Administration. (2016, September 19). FDA grants accelerated approval to first drug for Duchenne muscular dystrophy. Retrieved from https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-first-drug-duchenne-muscular-dystrophy