Genentech's Erivedge approved as first treatment for advanced basal cell carcinoma

The Food and Drug Administration approved Genentech Inc.'s Erivedge (vismodegib) capsules as the first treatment for advanced basal cell carcinoma (BCC). Specifically, the drug is indicated for use in adults with locally advanced BCC (laBCC) who are not candidates for surgery or radiation and for patients with metastatic BCC (mBCC). The approval was based on results of the ERIVANCE BCC trial, a single-arm, two-cohort, open-label, Phase II study that included 104 patients with advanced...

The Food and Drug Administration approved Genentech Inc.'s Erivedge (vismodegib) capsules as the first treatment for advanced basal cell carcinoma (BCC).

Specifically, the drug is indicated for use in adults with locally advanced BCC (laBCC) who are not candidates for surgery or radiation and for patients with metastatic BCC (mBCC).

The approval was based on results of the ERIVANCE BCC trial, a single-arm, two-cohort, open-label, Phase II study that included 104 patients with advanced BCC, including 71 patients with laBCC and 33 patients with mBCC. The primary endpoint of the study was objective response rate (ORR), as assessed by an independent review. The patients were treated with Erivedge at a dosage of 150 mg/day until their disease progressed or toxicity became intolerable.

Erivedge substantially shrank tumors or healed visible lesions (producing an objective response) in 43 percent of the patients with laBCC and in 30 percent of those with mBCC, as assessed by an independent review facility. The ORR as evaluated by study investigators, a secondary endpoint, was 60 percent for laBCC and 46 percent for mBCC. The median duration of progression-free survival, according to independent review, for both cohorts was 9.5 months.

The drug is a first-in-class inhibitor of the hedgehog pathway. According to Genentech, abnormal hedgehog signaling is implicated in more than 90 percent of BCC cases.

Genentech's New Drug Application for Erivedge was reviewed under the FDA's priority review program, and the drug was approved ahead of the assigned Prescription Drug User Fee Act action date of March 8.

Erivedge was approved with a boxed warning to alert patients and health care professionals of the potential risk of death or severe birth effects to a fetus. The warning states that pregnancy status must be verified before initiating treatment with Erivedge and that patients of both sexes should be warned about these risks and the need for birth control.

The drug was discovered by Genentech and is being developed under a collaboration agreement between Curis Inc. and Genentech's parent company, Roche. According to the agreement, Genentech, Roche and Chugai Pharmaceutical Co. Ltd. are responsible for the clinical development and commercialization of the drug in different parts of the world.

The approval triggered a milestone payment of $10 million from Genentech to Curis, and Curis is entitled to royalties on product sales. The drug, which will be distributed through specialty pharmacies, is expected to be available within the next one to two weeks.

Genentech and Roche are also evaluating Erivedge in a Phase II trial of patients with operable forms of BCC.