Gonvick residents heading to Washington D.C. to advocate for Duchenne muscular dystrophy treatment
Tue, 09/26/2017 - 9:41am
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Gonvick residents Cheri and John Gunvalson and their son Jacob will travel to Washington, D.C. to attend a Federal Drug Administration, FDA public forum on September 28th that will help decide if a treatment their son is on will be approved in the U.S. Their son was diagnosed with Duchenne muscular dystrophy (DMD) and is currently enrolled in a clinical trial for a drug called Translarna.
A FDA advisory committee will listen to and review advice and insights from outside experts including doctors and patients who have had experience with Translarna. The committee’s end vote could be used as a deciding guideline when the FDA decides the drug’s fate on October 24th. Without treatment and an approval, the family fears Jacob’s condition may worsen and lead to death.
Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.
What the Gunvalsons will be giving is testimony for getting the drug approved in the United States. Jacob has been on the test drug for the past 8 years. There is no question that the benefits of this drug far outweigh the risks. Every 90 days for the past eight years Jacob has gone to the University of Minnesota for treatment. He’s currently enrolled in a clinical trial for ataluren, which he takes three times a day.
“Independence is my life; without independence, I’m not living my life,” said Jacob.
He has had many successes in his life. He graduated from the University of North
Dakota as a licensed social worker. He has interned and worked at the Minnesota Governor’s office, the State Senate and at the institute for mental health.
Jacob went into social work as his true passion is to help others. He does not want his disease to limit his happiness. His goals are to work, make money and live a life as independent as possible. He will be 26 on his birthday October 5. He will celebrate his birthday by going to a Viking game which he hopes they will win.
DMD is a rare form of muscular dystrophy that causes muscle weakness. Patients eventually lose the ability to care for themselves. The typical life expectancy for someone with DMD is mid-20s. Jacob lost the ability to walk around 2007 and is now permanently in an electric wheelchair.
Although Jacob is wheelchair bound, he doesn’t take for granted performing tasks such as using his cell phone, something his peers with the disease are not able to do.
Congressman Peterson’s speech to the FDA public forum
Thank you for the opportunity to speak on behalf of Minnesota’s 7th District regarding ataluren’s application for approval. The timely delivery of this treatment is of the utmost importance to the Duchenne community.
One of my constituents, Jacob Gunvalson, is in the audience with us today to share his experience with Duchenne. Jacob was not expected to live past his teenage years, but access to ataluren has allowed him to live and thrive well into his 20s with no side effects. Jacob recently completed a successful internship in Governor Dayton’s office in Minnesota and enjoyed a trip to Europe with some of his friends.
During my time in Congress, I have consistently supported several measures to ensure that my constituents can benefit from life-saving therapies the way that Jacob has.
The 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) enhanced the FDA’s ability to speed patient access to safe and effective products. In particular, the legislation helped develop and implement accelerated approval programs to provide therapies to patients with rare, debilitating, and 100% fatal diseases. Under FDASIA, treatments that benefit Duchenne patients warrant consideration for accelerated approval.
More recently, Congress passed the 21st Century Cures Act which recognized the essential role that patient advocates play in the development of drugs and medical devices. It is my hope that, in keeping with this legislation, the FDA will enhance its efforts to incorporate patient experience into its regulatory evaluations and decision-making.
As there are no alternative therapies for this particular form of Duchenne eligible for purchase or approval in the United States, patients are left unable to mitigate the effects of the deadly disease. The full consideration of ataluren not only fulfills the Congressional intent of FDASIA and the 21st Century Cures Act, but also has the potential to save lives across the nation.
Treatment for Duchenne Muscular Dystrophy
SOUTH PLAINFIELD, N.J., PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the U.S. Food and Drug Administration (FDA) has acknowledged the filing over protest of PTC's New Drug Application (NDA) for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The Company is seeking approval to market the drug for the treatment of nmDMD patients in the United States. Translarna received marketing authorization for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.
The FDA has granted standard review and assigned a Prescription Drug User Fee Act (PDUFA) date of October 24, 2017. The PDUFA date is the target date for the FDA to complete its review of the NDA.
"We look forward to working closely with the FDA and the DMD community to bring this much-needed therapy to patients," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We believe that the totality of clinical data in our NDA, which includes the results of two of the largest placebo-controlled DMD clinical trials ever conducted, demonstrates Translarna's benefits to patients and merits a full and fair review by the FDA, including an advisory committee meeting."
Primarily affecting males, Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD lose the ability to walk in their early teens and experience life-threatening lung and heart complications in their late teens and twenties. It is estimated that nonsense mutations account for approximately 13% of DMD cases.
PTC used the FDA's file over protest regulations to file the NDA. These regulations allow a company to have its NDA filed and reviewed following receipt of a refuse to file determination.