Roche is aiming for accelerated approval of its Hedgehog antagonist vismodegib, a small molecule licensed from Curis, of Lexington, Massachusetts, based on an uncontrolled phase 2 trial. The Basel-based company submitted a new drug application to the US Food and Drug Administration for vismodegib in September undeterred by the agency's refuse-to-file letter issued last year for T-DM1 (trastuzumab-DM1, a conjugate of Herceptin and maytansine) for mid-stage breast cancer. “It really comes down to the question of, 'Is this addressing an unmet medical need?' and that's the crux of it,” says analyst Joe Pantginis, of Roth Capital Partners in New York. Whereas for breast cancer several therapeutic options exist, vismodegib is targeted to patients with limited treatment options and no standard of care. “Unmet medical need is a prerequisite for accelerated approval based on single-arm trials,” says analyst Jason Kantor, of RBC Capital Markets in San Francisco. Vismodegib shrank tumors or healed lesions in 43% of locally advanced and 30% of metastatic basal cell carcinoma in the pivotal study. The objective response rate was 60% and 46%, respectively, and progression-free survival for both was 9.5 months. If approved, analysts expect a launch next year with sales somewhere north of $150 million.