Biotech Notebook: Vertex, Medicines Co.

Nor do I believe that the FDA will be swayed by the "treatment-resistant hepatitis C patients are just like HIV patients" argument. Hepatitis C is a serious and fatal disease, but it also progresses more slowly than HIV. Many patients, even those who have failed prior treatment, can live a long time without the disease progressing to the point where life-saving re-treatment becomes imperative.

Cowen and Co. biotech analyst (and hepatitis C guru) Rachel McMinn is a Vertex bull, but she too says the company will have a challenge convincing the FDA to approve telaprevir for these hard-to-treat Hep C patients without running a new study.

"It's not a zero percent chance, but it's really a tough call," says McMinn, adding that the FDA will likely struggle with the notion that the unmet medical need for these patients is great enough now to justify an approval instead of waiting two years for more definitive and complete data on telaprevir's efficacy and safety.

None of this means that Vertex shouldn't push the FDA to review telaprevir as soon as possible; and the company has stated it will do just that once the final data from PROVE 3 are in hand in late 2008, early 2009.

If Vertex is successful, telaprevir could be filed with the FDA in mid-2009 with an approval in late 2009. If not, the drug will likely be filed in the second half of 2010 based on data from an ongoing phase III study in treatment-naïve hepatitis C patients.