In a landmark approval a month and half ahead of schedule, the first targeted treatment for basal cell carcinoma, the most common form of skin cancer, won approval today from the US Food and Drug Administration.

“Today is a huge day for people with basal cell carcinoma,” says Kristi Schmitt Burr, executive director of the Ohio-based Basal Cell Carcinoma Nevus Syndrome Life Support Network. “This absolutely changes the quality of life for those with the genetic form of the disease.”

It’s also a huge day for companies developing drugs that inhibit the Hedgehog pathway to treat cancer. As Nature Medicine reported last year, the newly approved drug—known as Erivedge (vismodegib) and marketed by the Swiss drug giant Roche and its San Francisco-based subsidiary Genentech—is the leader in a wave of new agents that target aberrant Hedgehog proteins, which can drive tumor formation by letting cell growth and differentiation go unchecked. In a 104-person phase 2 trial, Erivedge shrank tumors or healed visible lesions in 43% of the patients with locally advanced basal cell carcinoma and 30% of patients with metastatic forms of the disease.

But not all trials of Hedgehog inhibitors have been as successful. Last week, Cambridge, Massachusetts-based Infinity Pharmaceuticals announced that it was halting a phase 2 trial testing the company’s Hedgehog blocker, called saridegib, in people with pancreatic cancer. Despite Infinity’s hopes for the drug, preliminary data from the 122-person trial showed the drug did not improve overall survival in patients with metastatic pancreatic cancer compared to a placebo.

Photo courtesy of Claus Rebler via Wiki Creative Commons