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Experimental drug request granted for sick boy

Posted March 11, 2014
Updated March 12, 2014

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— 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.


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  • smdrn Mar 12, 2014

    It wasn't the media storm that made this happen, it was the drug company itself. He is NOT get a compassionate dose. They created a new clinical trial to include him and had to get FDA approval for the new trial, so it wouldn't delay the approval process to bring the drug to the market. If the FDA said no, he wouldn't be getting the drug at all. I'm glad it worked out this way and hope the drug saves that little boy's life.

  • MindBomb Mar 12, 2014

    I am aware of the difference between gross and net profits, and while the business may be different from years ago, I am also aware that large profits are still being made.

  • Beth Pearce Mar 12, 2014
    user avatar

    I was so glad to hear this, yesterday evening! Great news. Hope it helps.

  • Naysayer Mar 12, 2014

    Just wanted to add big kudos for St Jude's Hospital. Fantastic institution that has helped so many children and their families!

  • LastSon1981 Mar 12, 2014

    View quoted thread

    Yeah if there is nothing nothing gets done and nothing happens right? Maybe there will be more nothing that will explode causing something and that can save the boy

  • dollibug Mar 12, 2014

    My husband got cancer and was treated with *experimental drugs*....he was the 10 patient at the time that had the rare form of cancer. The doctors gave him a very slim chance of surviving it. He had chemo and radiation. This was 21 years ago. He lost 95 lbs and looked like *death warmed over*. Thanks to the *experimental drugs* which allowed him to live. I hope and pray that this little one will also survive and be blessed with a wonderful and long life. It is sad that it took so much publicity to get the drugs that might save this child's life. THANK GOD someone helped him.

  • Olenc Native Mar 12, 2014
    user avatar

    View quoted thread

    Very good point. I wonder how long before this comment has to get pulled.

  • btneast Mar 12, 2014

    [bbut the drug companies are still reaping large rewards as well. Billions and millions certainly seems like a huge profit to me.][/b]

    Know the difference between "gross profit " and "net profit"? Yes, there are several companies that have hit a home run on a particular drug, but most of that money goes back into developing tomorrow's new miracle drug. The overwhelming majority of drugs in development never see the light of day. The cost to develop a new drug is astronomical. The success rate is very low. I have been involved with "big pharma" for over 20 years. The profits now vs then are vastly different. Why do you think there have been so many mergers and layoffs over the past few years?

  • MindBomb Mar 12, 2014

    LoveMyPirates- Again, I disagree. Yes, there are generics, but there are plenty of new drugs coming out based on the same medication, but with a "tweak" here and there to put it back on the market as a brand name medication. Consumers are becoming more aware, but also convinced they need a medication (with no generic equivalent) because their doctor said so. Yes, I am sure big energy is big money, but the drug companies are still reaping large rewards as well. Billions and millions certainly seems like a huge profit to me.

  • Kristin Byrne Mar 12, 2014
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    View quoted thread

    While I agree with most of your points, you've grossly underestimated a cost of bringing a new drug to market. Now the average is $5 billion from start to finish for a drug that's approved, and for every drug that's approved, there are many that aren't. The money lost from drugs that are never approved is in the BILLIONS of dollars.

    Kind of scary when you think about it. The cost of bringing a drug to market is a lot of the reason that these drugs are so expensive once they get there. It's ridiculous!