In a compelling speech delivered at the Commonwealth Club of San Francisco in early February, FDA Commissioner Margaret Hamburg cited the approvals of three innovative drugs over the previous month – Genentech’s Erivedge, Vertex’s Kalydeco, and BTG’s Voraxaze – as examples of how the agency is using the advancing regulatory science to create a more flexible and innovation-supportive process that “swiftly and surely reviews product applications that come before us.”
Erivedge, approved for metastatic basal cell carcinoma, incorporates a “new finding for cancer cell growth – the so-called ‘Hedgehog pathway,’” Hamburg noted. Reviewed under the agency’s priority review program, it was “approved a month ahead of its goal date based on single study that used a non-traditional measure for success.”
Vertex’s Kalydeco, which was “conceived by a San Diego startup” and targets a genetic mutation in a subset of patients with cystic fibrosis, is the first to “treat the underlying mechanism of the disease rather than the symptoms,” Hamburg noted. Both the Kalydeco application and Vertex’s 2011-approved application for its Hepatitis C Treatment Incivek were quality-by-design-based filings, involving close collaboration during the development process with the virtual company’s contract manufacturing partners at both the API and dosage levels.
BTG’s Voraxaze, an enzyme which removes the unused portion of methotrexate from the bloodstream, was approved based on a 22-patient study.
“When strong science and quality applications come together, FDA can provide responsible and flexible regulatory oversight,” the Commissioner stressed.
She added that the agency has “a clear responsibility to make sure this happens. The successful translation of new discoveries in science, and the realization of new opportunities into real world products that people need and can depend on is our foremost goal. We must be an agency with the strong scientific and regulatory capacity that can provide the kind of clear, timely, consistent and reliable science-based, smart regulation that benefits industry and most importantly, benefits patients.”