Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the Antiviral Drugs Advisory Committee to the U.S. Food and Drug Administration (FDA) voted unanimously to recommend FDA approval of telaprevir for people with genotype 1 chronic hepatitis C. The Committee recommended by a vote of 18-0 the approval of telaprevir for those who were not treated previously and those who were treated previously but not cured with currently available medicines. Telaprevir was studied in all major subgroups of people who were treated previously and not cured: relapsers, partial responders and null responders. The FDA is expected to make a decision on the approval of telaprevir by May 23, 2011, under the Prescription Drug User Fee Act (PDUFA). The FDA is not bound by the Committee's recommendation, but usually follows its advice.
In Phase 3 studies, telaprevir was given for 12 weeks in combination with pegylated-interferon and ribavirin (P/R) followed by P/R alone for a total of 24 weeks or 48 weeks of treatment. Data from these studies that were reviewed by the Committee showed that people who received telaprevir-based combination therapy achieved significantly higher rates of sustained viral response (SVR, or viral cure) compared to treatment with 48 weeks of P/R alone, regardless of their experience with prior treatment. Among people who were not treated previously, 79 percent achieved a viral cure with telaprevir-based combination therapy compared to 46 percent who achieved a viral cure with P/R alone.
Approximately two-thirds of people in Phase 3 studies who were not treated previously and who received telaprevir-based combination therapy were eligible to complete their treatment in six months - half the time needed with currently available medicines. The FDA Committee discussed Vertex's request for the approval of response-guided therapy to allow for a six-month treatment duration for people who were not treated previously as well as for those who relapsed after prior treatment with P/R alone (prior relapsers). Side effects observed with telaprevir-based combination therapy were consistent across the Phase 3 studies. Rash and anemia occurred more frequently among those treated with telaprevir-based combination therapy compared with those who received pegylated-interferon and ribavirin alone.
"Hepatitis C is a curable disease with potentially devastating consequences if left untreated, so we are pleased by the Committee's unanimous recommendation to approve telaprevir for a broad group of people with hepatitis C," said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. "We look forward to working with the FDA as it prepares to make its decision next month."
Safety and Tolerability Information for Telaprevir
The safety profile of telaprevir has been well characterized. Data from more than 40 clinical studies across a broad group of nearly 4,000 people were included in the new drug application. The side effects observed with telaprevir-based combination therapy were consistent across the Phase 3 studies. The most common side effects, regardless of treatment arm, were fatigue, pruritus (itchiness), nausea, headache, rash, anemia, flu-like symptoms, insomnia and diarrhea with the majority being mild to moderate.
Rash and anemia occurred more frequently among those treated with telaprevir-based combination therapy. In Phase 3 studies, discontinuation of all medicines due to either rash or anemia during the telaprevir/placebo treatment phase was approximately 1 percent for rash and 1 percent for anemia. Rash was primarily characterized as eczema-like, manageable and resolved following discontinuation of telaprevir. More than 90 percent of rash was mild to moderate and investigators in the studies primarily used topical corticosteroids and/or antihistamines to treat rash. Anemia was primarily managed by reducing the dose of ribavirin.