Genentech submits vismodegib NDA to FDA for treatment of advanced basal cell carcinoma

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the company has submitted a New Drug Application for vismodegib to the U.S. Food and Drug Administration (FDA) for the treatment of people with advanced basal cell carcinoma (BCC) for whom surgery is considered inappropriate. Vismodegib is an investigational, oral, targeted medicine designed to selectively inhibit signaling in the Hedgehog pathway, which is implicated in more than 90 percent of BCC cases. BCC is the most common type of skin cancer, which is generally considered curable by surgery. However, when it advances, BCC can cause disfiguring and debilitating effects and can ultimately be life-threatening.

"We are excited by the pivotal study results that showed vismodegib substantially reduced tumor size or healed lesions for people with this rare skin cancer, which has no approved treatments," said Hal Barron, M.D., chief medical officer and head, Global Product Development. "We look forward to continuing our discussions with the FDA about these data."

This submission to the FDA is based on results from the pivotal ERIVANCE BCC study that evaluated vismodegib in people with advanced BCC. The trial showed vismodegib substantially shrank tumors or healed visible lesions (overall response rate, or ORR) in 43 percent of patients with locally advanced BCC (laBCC) and 30 percent of patients with metastatic BCC (mBCC), as assessed by independent review, the primary endpoint of the study.

The ORR as assessed by study investigators, a secondary endpoint, was 60 percent for laBCC and 46 percent for mBCC. The median duration of progression-free survival (PFS) by independent review for both metastatic and locally advanced BCC patients was 9.5 months.

The most common drug-related adverse events were muscle spasms, hair loss, altered taste sensation, weight loss, fatigue, nausea, decreased appetite and diarrhea. Serious adverse events (SAEs) were observed in 26 patients (25 percent). Four patients (4 percent) had SAEs that were considered to be related to vismodegib, including one case each of: blocked bile flow from the liver (cholestasis), dehydration with loss of consciousness (syncope), pneumonia accompanied by an inability of the heart to pump enough blood (cardiac failure) and a sudden arterial blockage in the lung (pulmonary embolism). Fatal events were reported in seven patients (7 percent); none were considered by investigators to be related to vismodegib. In all cases, patients had other pre-existing diseases or symptoms that were related to their presumed cause of death.

Source: Genentech