After much negative publicity, a North Carolina pharmaceutical drug company has reversed their stance of not giving an ailing 7-year-old boy an experimental drug and will now provide the anti-viral medicine. Josh Hardy has been battling cancer since he was first diagnosed as a baby. Since then, his parents report that he has seen the cancer go into remission three times. A diagnosis in November 2013 revealed myelodysplastic syndrome which is a bone marrow disorder. He had been cancer-free for four years until November.
After undergoing chemotherapy and a bone marrow transplant, the transplanted cells began to attack Josh’s body. After a number of complications, he was admitted to the ICU in January 2014 for heart failure. Eventually improving, Josh was seemingly on the road to recovery. It was at this point that he came down with an adenovirus. Doctors placed him on an antiviral that began to damage his kidneys. Medical professionals were unable to allow Hardy’s immune system to fight the virus as it could reinitiate the disease from the transplant. Given his medical background, it was at this point that doctors recommended the experimental drug brincidofovir.
Brincidofovir is manufactured by a North Carolina pharmaceutical drug company, Chimerix. It is an antiviral drug that is still under trial and considered experimental. Originally, the pharmaceutical company told the family that they will not give authorization for compassionate use of the experimental drug to their 7-year-old son. The CEO of Chimerix, Kevin Moch, said that the inability to provide compassionate usage of experimental drugs is excruciating. He followed up this comment by explaining that being faced with this decision is not something anyone ever should have to do. While many are indicating that the reason behind the initial denial from Chimerix is monetary based, Moch says it is not. He told reporters that the decision was based on ethics.
The company has had brincidofovir in development for the last 14 years and is currently attempting to gain approval from the U.S. Food and Drug Administration. The approval from the FDA would allow the drug to be available to everyone. In cases such as this, when a pharmaceutical drug which is still considered experimental, compassionate care use can conceivably affect the ability of the drug to get the needed approval. In the past, the company provided compassionate care use of brincidofovir to other patients in conjunction with ongoing tests. Testing procedures are not currently underway. Allowing the use of the anti-viral outside of clinical trials could potentially slow, or altogether disrupt, the possibility of getting drug approval. The allowance of non trial compassionate care usage by Chimerix could result in placing the whole program development in jeopardy.
The company anticipated the conflicts between those compassionate use requests and the larger patient population who would be served by having a swifter FDA approval of the drug. Given these parameters, the company denied the request. The pharmaceutical drug company had previously set up a dedicated email address to handle requests for experimental drugs before their approval. Additionally, the company refers to a paper released by the Biotechnology Industry Organization which outlines historical compassionate care use and the effects on FDA approval. The April 2010 study indicates that the use could create a conflict between the singular patient currently in need and the larger population which could benefit from having the experimental drug quickly approved.
However, Chimerix announced yesterday that they have forged an agreement with the FDA so they can launch an immediate new open-label trial of brincidofovir and allow Josh Hardy to become the first trial patient. A Phase 3 study will be developed in order to assess the potential effectiveness of this experimental drug on adenoviruses. The initial pilot study, of which Josh is slated to be enrolled as the first patient today, will be included. The company has said they are grateful to the FDA for their assistance in expediting these studies. It appears that this 7-year-old boy will be given the experimental drug which may help combat the medical situation in which he currently finds himself, due to the cooperation between the FDA and the pharmaceutical company, Chimerix.
By Dee Mueller