A 7-year-old boy who has beaten cancer four times but whose weakened immune system is being threatened by a rare virus will start receiving an experimental treatment within 48 hours after the drug’s manufacturer reached an agreement with federal officials for a pilot trial to begin immediately.
The boy, Josh Hardy, will be the first patient to be enrolled Wednesday in the new study of the drug, brincidofovir, the Durham, N.C.-based company, Chimerix, announced late Tuesday.
Earlier the company had said it could not release the drug to Josh outside of clinical trials. The boy’s family had been pleading with the company to change its mind.
The U.S. Food and Drug Administration agreed to the pilot trial of brincidofovir for the treatment of adenovirus infections in immune-compromised patients, the company said in a statement late Tuesday. The FDA will work expeditiously with Chimerix to design a Phase 3 study that would be a continuation of the pilot trial, the company said.
“Josh Hardy’s story brought to public attention the often-devastating impact of adenovirus infection, and helped accelerate a discussion between the FDA and Chimerix regarding the need for additional clinical development to assess brincidofovir’s potential in adenovirus infection,” Kenneth I. Moch, Chimerix’s president and CEO, said in a statement.
Matthew Herper Forbes Staff
Pharma & Healthcare 3/11/2014 @ 9:12PM 4,486 views
Company Makes Drug Available To Ailing Boy Following Public Outcry
Chimerix, a small and unprofitable biotechnology company, will make an experimental drug available to a young Virginia boy who is suffering from an infection he contracted while being treated for cancer.
Chimerix originally said it could not ethically provide the drug to one patient without opening the floodgates to others, presenting it with a huge operational burden that might prevent the drug, brincidofovir, from ever reaching patients. The story of the boy, Joshua Hardy, was shared on CNN, USA Today, The Huffington Post, and generated a large volume of posts on the social networking site Twitter under the hashtag #savejosh. This evening, Chimerix, based in Durham, North Carolina, issued a press release saying that it was going to start a new, 20-patient clinical trial for the treatment of adenovirus (the virus brincidofovir treats) in immunocompromised patients. The first immunocompromised patient, dosed tomorrow morning, will be Josh Hardy.
“This 20-patient open-label study underscores Chimerix’s mission to develop innovative antiviral therapies in areas of high unmet need – for everyone,” said Chimerix Chief Executive Kenneth I. Moch in his company’s press release. “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.”