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FDA Hearing on Expanding Access to Antivirals for HCV Patients

June 1, 2010

On April 30, 2010, physicians and patients provided public testimony to the Food and Drug Administration (FDA) in favor of allowing seriously ill people with hepatitis C (HCV) access to “direct-acting” antiviral therapies.

Left unchecked, HCV can cause liver cancer, cirrhosis, end-stage liver disease and liver failure. The standard treatment combines pegylated interferon (P-IFN) and ribavirin (RBV). However, nearly 50% of patients do not respond to it. “Current control of hepatitis C is not working,” said Peter Lurie, MD, of the FDA's Office of the Commissioner. Further, patients who do respond often experience debilitating side effects that can last the duration of the treatment—either 24 or 48 weeks. Interferon’s side effects include severe flulike symptoms, depression, fatigue and insomnia. Ribavirin can cause anemia, skin rash, fatigue and birth defects.

The hearing was held in response to a petition submitted in the Fall of 2009 by Mark Antell and Paul Brayshaw, representing the bleeding disorders community. In the petition, the advocates requested a public hearing for all stakeholders, plus expanded access to promising HCV drugs and combination regimens for patients unresponsive to standard P-IFN/RBV. Both spoke about the seriousness of HCV in the bleeding disorders community and the urgent need for expanded access to treatments. Others who could benefit from a broader regimen of drugs include patients with cirrhosis, HIV and those who are minorities.          

Healthcare providers at the hearing expressed concern about limited access to clinical trials. Diana Sylvestre, MD, who treats HCV-infected intravenous drug users in the San Francisco area, mentioned that only a small subset of “real-world” HCV patients are included in trials of new drugs due to their drug use, co-morbidities and mental illness.

The FDA is creating a guidance document on the use of direct-acting antiviral agents, which is expected to be released this year. If approved, an expanded access program could be applied to several novel HCV treatments now in clinical trials. The therapy farthest along in development is the investigational drug Telaprevir, a protease inhibitor manufactured by Vertex and partner Johnson & Johnson. The results of a phase II trial published in The New England Journal of Medicine (NEJM) in April showed that HCV patients previously treated unsuccessfully with P-IFN/RBV had a much greater chance of clearing the virus when telaprevir was added to their treatment regimen. Success is measured by patients’ ability to achieve sustained virological response (SVR) for at least six months after completing therapy. In the NEJM study, more than 50% of patients in the P-IFN/RBV/telaprevir groups achieved SVR vs. only 14% in the P-IFN/RBV group.     

Source: MedPage Today, April 30, 2010