UPDATE: Company says ailing 7-year-old will get drug as part of study

(CNN) — The Chimerix pharmaceutical company says it will give ailing 7-year-old Josh Hardy a drug that could help him survive as part of a pilot trial.

The company said in a statement Tuesday that it had reached an agreement with the Food and Drug Administration to begin a pilot trial with the drug, brincidofovir, and provide it to Hardy as part of that study.

The study is expected to start on Wednesday, the statement said.

The company had previously denied calls from the 7-year-old’s family to give him the drug, arguing that spending the time to help Josh and others like him would slow down efforts to get the drug on the market.

The child’s family and supporters, in online and media campaigns, had been pushing the company to change its mind.

CNN told Josh’s story Sunday night, describing his parents’ desperate pleas for help from Chimerix.

Josh’s journey began when he was diagnosed with a rare form of kidney cancer at 9 months old. Over the years, cancer turned up in his thymus, lung, and bone marrow, and each time Josh beat it.

But a bone marrow transplant left Josh without much of an immune system, and in February doctors diagnosed him with an adenovirus that spread through his body.

Now he’s in critical condition in an intensive care unit as the virus ravages his body.

His doctors at St. Jude’s Children’s Research Hospital in Memphis had also been pushing for the company to provide the drug.

In a statement Tuesday, the hospital said it expected to receive the medication within 48 hours, but noted that its safety and effectiveness has not yet been established for use in children.

“It is also important to understand that this remains a critical and complex medical situation,” the hospital said.

“St. Jude will continue to pursue state-of-the-art treatment for Josh and all of our patients. We are grateful for the efforts of Chimerix, the FDA and many others who worked to achieve this outcome. We ask that you continue to keep Josh and his family in your thoughts.”

An FDA policy known as “compassionate use” allows someone with a serious or life-threatening disease to ask a drug company for an experimental drug.

Though it’s called compassionate use, sometimes it feels anything but compassionate.

Companies often say yes: The FDA approved 974 compassionate use arrangements in fiscal year 2013.

But pharmaceutical companies often say no, as Chimerix did at first to Josh Hardy.

Chimerix President Kenneth Moch previously told CNN he wouldn’t back down from his decision not to give Josh the drug.

In the company’s statement Tuesday, he said he hopes the new study will help other patients as well.

“This 20-patient open-label study underscores Chimerix’s mission to develop innovative antiviral therapies in areas of high unmet need — for everyone,” he said. “Being unable to fulfill requests for compassionate use is excruciating, and not a decision any one of us ever wants to have to make. It is essential that each individual in a health crisis be treated with equal gravity and value, a principle we have upheld by pursuing further clinical study of brincidofovir that will inform its use in adenovirus and other serious DNA viral infections.”

2 comments

  • Thomas Offal

    The company is not denying Josh the drug because it’s evil or because of money – it’s denying because providing the drug would likely delay FDA approval by months or years. The federal government Chimirex $72M to run a CLINICAL TRIAL — which does NOT require it (or any other pharmaceutical company) to distribute an unapproved drug on a “compassionate use” basis.

    Do you know WHY there are stringent laws/regulations governing clinical trials? Because 1) it ensures that drugs are safe before doctors are allowed to prescribe them and 2) stops horrors like the infamous Tuskegee Experiment from EVER happening again. The laws/regs are NEEDED.

    Last but not least, there are multiple stages to a clinical trial – in this particular case, the FDA has deemed it safe (Phase 1), effective relative to placebo (Phase 2) and is in the process of confirming it to be safe/effective (Phase 3). The drug was NOT designed to fight the type of infection little Josh is battling, so if he got the drug and had an adverse reaction for any reason (e.g. so sick that he died anyways, reacts badly with the other drugs he’s taken, etc), Chimirex is required to report it to the FDA, which will delay FDA approval by months or years while the reason for the adverse effect is investigated.

    My heart goes out to Josh and his family – but the drug company is making the RIGHT call by refusing to give him the drug.

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