RANKIN CO., Miss. (WLBT) – The U.S. Food and Drug Administration\u2019s denial of a life-changing medication pushed a Rankin County family to visit Washington, D.C., and prove its effectiveness. <\/p>\n
At the end of September, the medication for a teenager\u2019s rare disease would no longer have been available. <\/p>\n
However, the family\u2019s battle for access made a change.<\/p>\n
\u201cNow I can actually have a life where, before, I couldn\u2019t,\u201d Christopher Pena said.<\/p>\n
Pena has Barth syndrome, a rare genetic mitochondrial disease. <\/p>\n
The teen said he is now looking forward to a normal life – walking without tiring.<\/p>\n
Just days before the 17-year-old would not have access to Elamipretide, the FDA approved the medication. <\/p>\n
In August, his mother, Kristi Pena, and aunt, Amy Wilson, met FDA Commissioner Dr. Marty Makary. <\/p>\n
On Friday, they got the call that the drug was approved.<\/p>\n
\u201cIt\u2019s a medicine we\u2019ve been fighting for since I was six, seven years old,\u201d Christopher Pena said. \u201cKnowing we have it now is amazing.\u201d<\/p>\n
His mother was told the FDA had until February to decide if production would be permanently halted.<\/p>\n
\u201cWhat has transpired in the past month has ultimately changed the course of history for a lot of people,\u201d said Kristi Pena. \u201cThis is setting a precedent. This is paving the way for more rare disease developments. This highlighted and underscored a broken system, and our administration has acknowledged that.\u201d<\/p>\n
\u201cI\u2019m not worried about having to bury my son,\u201d Amy Wilson added.<\/p>\n
Amy Wilson\u2019s son, Jacob, also has Barth syndrome. He was recently granted emergency access and is no longer in kidney failure. <\/p>\n
\u201cHe has gained 10 plus pounds. His kidney function is getting better,\u201d Wilson explained. \u201cWe know that this drug works. We know that it\u2019s safe.\u201d<\/p>\n
Elamipretide, under the prescription Forzinty, is now available to around 160 people in the U.S. with certain rare mitochondrial diseases. <\/p>\n
Approximately 300 people around the world are diagnosed with Barth syndrome.<\/p>\n
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