Experimental drug request granted for sick boy
Posted March 11, 2014
Updated March 12, 2014
Durham, N.C. — Chimerix, the Durham-based drug company working on experimental medicine that could save the life of a seven-year-old Virginia child, was granted approval by the U.S. Food and Drug Administration to administer the drug to the boy, the company announced Tuesday evening.
Seven-year-old Josh Hardy is in the intensive care unit at St. Jude Children’s Research Hospital in Memphis, Tenn., where relatives say he’s trying to fight off a virus contracted while undergoing cancer treatment.
Josh’s mother, Aimee, asked the company for a “compassionate dose” of brincidofovir, but the company originally denied the request because doing so could slow down the process of getting the drug to the market, they said.
"I know you have your reasons, but this is my child and I don't care what your reasons are," she said.
Brincidofovir has been in development for nearly 14 years and has "the potential to become the first broad-spectrum antiviral for the prevention and treatment of clinically significant infections and diseases caused by DNA viruses," the company said.
Chimerix President and CEO Kenneth Moch told WRAL it was "heartbreaking" to say no to the initial request.
"Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make," he said in a statement.
Josh's request became a national story in the media and online. A Facebook page dedicated to his request has over 21,000 likes.
"Thank you to every member of Josh’s Army," read a post Tuesday night. "The world has heard you and because of you Josh and many others will have the opportunity to receive CMX001 (Brincidofovir) the life saving antiviral drug made by Chimerix."
The post had more than 1,000 likes about 22 minutes after it was posted.
The drug was shipped to Memphis and Josh could receive his first dose as soon as Wednesday, said Moch, who added that the company has been working with the FDA all along to find a way to get the drug to Josh and others like him.
"Obviously the social media helped accelerate the discussion, but we were in that process already," Moch said.
The boy's mother and family expressed their thanks to everyone who took part in the social media campaign.
"It is completely unbelievable what has transpired since last Thursday," she said in a statement. "(It's) nothing short of a miracle. We are so thankful that Chimerix was able to release the medicine for Josh. The truth of the matter is he is still in the ICU, and we would like to focus on him making a full recovery."
Josh will be the first patient in a pilot trial of the medicine.
The FDA allows someone with an immediate life-threatening illness to ask for permission to use experimental drugs that have not yet received the agency’s approval. Last year, the FDA approved 974 such requests, according to CNN.