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FDA Issues Guidance for Development of Direct-acting Antiviral Drugs for Hepatitis C

SUMMARY: The U.S. Food and Drug Administration (FDA) this week issued draft guidelines to assist pharmaceutical companies in developing and gaining approval for direct-acting agents targeting hepatitis C virus (HCV). Numerous such drugs are now in the pipeline, with the HCV protease inhibitors telaprevir and boceprevir nearest the finish line. The guidance outlines the types of non-clinical studies and clinical trials that should be done, specifying that some trials should include people with compensated and decompensated liver cirrhosis and HIV/HCV coinfected patients. The FDA welcomes comments on the draft through November 15.

Below is the text of an FDA e-mail message describing the draft guidance and the comment procedure.

FDA Hepatitis Update -- Availability of draft Guidance:
Chronic Hepatitis C Virus Infection:
Developing Direct-Acting Antiviral Agents for Treatment

September 14, 2010 -- The Food and Drug Administration (FDA) is announcing the availability of draft guidance for industry entitled "Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Agents for Treatment." At present, there are a large number of drugs for the treatment of chronic hepatitis C (CHC) in active development. The purpose of this guidance is to assist sponsors in all phases of development of direct-acting antiviral agents (DAAs), defined as agents that interfere with specific steps in the hepatitis C virus (HCV) replication cycle. The guidance outlines the types of non-clinical studies and clinical trials recommended throughout the drug development process, such as early phases of clinical development, phase 3 protocol designs, and endpoints for the treatment of CHC to support approval of treatments for CHC, including patients with compensated and decompensated cirrhosis and those co-infected with HIV. The guidance also addresses pre-approval access in the form of treatment investigational new drug applications (INDs) and intermediate-sized safety protocols (collectively known as expanded access).

Important issues addressed in this guidance include: drug development methods to reduce the emergence of drug resistance, types of trial designs to assess optimal dose and treatment duration, combination therapy with multiple investigational drugs, recommendations on development of drugs to meet unmet medical needs, and use of treatment INDs or other smaller safety protocols to provide early access of multiple DAAs for patients at risk of imminent progression of liver disease.

The draft guidance, when finalized, will represent the agency's current thinking on developing DAAs for treatment of CHC virus infection. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. An alternative approach may be used if such approach satisfies the requirements of the applicable statutes and regulations.

The draft guidance is available on the FDA web site at

Although comments are accepted for any guidance at any time, to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, please submit written or electronic comments on the draft guidance by November 15, 2010, and include the docket number, FDA-2010-D-0462, in any comment you submit.

You may submit written comments on the draft guidance to the
Division of Dockets Management (HFA-305)
Food and Drug Administration
5630 Fishers Lane, rm. 1061,
Rockville, MD 20852

You may also submit electronic comments at

Jeffrey Murray,
Center for Drug Evaluation and Research,
Food and Drug Administration,
10903 New Hampshire Ave.,
Bldg. 22, rm. 6360,
Silver Spring, MD 20993-0002,

The complete Federal Register Notice announcing availability of this draft guidance is available at


R Klein and K Struble. FDA Hepatitis Update -- Availability of draft Guidance: Chronic Hepatitis C Virus Infection: Developing Direct-Acting Antiviral Agents for Treatment. U.S. Food and Drug Administration. September 14, 2010.






















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