Although it remains to be seen which medications will be approved to treat Duchenne muscular dystrophy, lawmakers have tried to make the path easier.
DUCHENNE MUSCULAR DYSTROPHY is a genetic disorder characterized by progressive muscle degeneration and weakness.
It impacts fewer than 200,000 annually in the United States and is 1 of 9 types of muscular dystrophy that occurs primarily in boys. The cause “is a mutation in a gene called the DMD gene, which encodes the muscle protein dystrophin. Boys with Duchenne muscular dystrophy (DMD) do not make the dystrophin protein in their muscles. DMD is inherited in an X-Linked recessive fashion; however, it may also occur in families without a known family history of the condition.”1
As this disease progresses, children can lose the ability to walk by their early teens. DMD, which can cause patients to lose respiratory function and can cause severe cardiac issues, can be fatal by the time a person enters their 20s or early 30s. French neurologist Guillaume Benjamin Amand Duchenne first described the disease in the 1860s.
Until the 1980s, however, information was limited surrounding the cause of any form of muscular dystrophy. Recent advances in cardiac and respiratory care have improved life expectancy to allow individuals to live beyond their early teens to enter young adulthood, with some cases of men living into their 40s and 50s.2
While there are no FDA-approved therapies for Duchenne, recently the path to approval has grown increasingly rocky. BioMarin Pharmaceutical was the first to have the FDA reject its treatment called drisapersen. Sarepta Therapeutics received a critical initial review from the FDA, but Sarepta’s drug eteplirsen will receive a final adjudication by the FDA no later than this coming May.
PTC Therapeutics’ ataluren has become the latest drug whose application was rejected by the FDA. However, Tarix Orphan, Catabsis Pharmaceutical, and Marathon Pharmaceuticals are also working on drugs with a potential solution to DMD. While it remains to be seen which medications will be approved to treat DMD, lawmakers have tried to make the path easier.
Landmark legislation affecting Duchenne was enacted in December 2001. Public Law 107-84 titled, the Muscular Dystrophy Community Assistance, Research and Education Amendments of 2001, was the first Congressional bill that targeted muscular dystrophy.
The highlights of the law are listed below:
This act was later amended via the enacted Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education Amendments of 2014. Public Law 113-166 established the following:
While these laws have advanced the cause, the question is what other types of legislation are currently in Congress that can help?
So what’s next? While there is still much work to do on the issue of curing DMD, it seems encouraging that rare diseases such as this are being discussed more openly in order to find a cure. As our science continues to improve, I am optimistic that a cure will eventually be found. The only question is once there is a cure, will the payer and manufacturer communities come together on the appropriate price to charge that balances the need for patient access. SPT
RON LANTON III, ESQ is president of True North Political Solutions, LLC. He has over 20 combined years of government affairs and legal experience. This includes activities on the municipal, state, and federal levels of government. Most recently, he worked for a pharmaceutical wholesaler where he created and oversaw the company’s government affairs department, served as their exclusive lobbyist, and advocated for the company’s various health care customers. Prior to that, Ron worked at a government affairs consulting firm in Arlington, Virginia, where he focused on health care, energy, commerce, and transportation issues. He has also clerked for a federal magistrate, was appointed as a municipal commissioner on environmental issues, and has served as consultant to Wall Street firms on financial issues. He has been a featured industry speaker on issues such as pharmaceutical safety and health care cost containment. Ron earned his juris doctor from The Ohio State University Moritz College of Law and a bachelor of arts from Miami University of Ohio. He is also a “40 Under 40” award recipient. He is admitted to practice law in New York, Illinois, and the District of Columbia.