January 30, 2012, 3:44 pm
F.D.A. Approves Drug for an Advanced Skin CancerBy ANDREW POLLACK
The first drug for an advanced form of the most common type of skin cancer won approval from the Food and Drug Administration on Monday.
The drug, Erivedge, made by Genentech, was approved for adults with basal cell carcinoma that has spread elsewhere in the body or those who are not candidates for the surgery or radiation ordinarily used to treat the disease.
Basal cell carcinoma is usually a slow-growing and painless form of cancer that develops on skin frequently exposed to the sun, like on the face, according to the F.D.A. If confined to a small location on the skin, it is generally considered curable by surgery and radiation treatment.
In rare cases, however, the tumors cannot be treated that way. In some cases, known as locally advanced disease, they invade nearby tissues and can become disfiguring. In other cases, they become metastatic, spreading elsewhere in the body.
The F.D.A. said in a news release that the approval was based on an analysis of 96 patients in a clinical trial with locally advanced or metastatic basal cell carcinoma. Some 30 percent of the patients with metastatic disease experienced a partial shrinkage of their tumors. For the patients with locally advanced disease, 43 percent experienced either a complete or partial shrinkage.
There was no control group getting a placebo in the trial, and the F.D.A. emphasized that it had approved the drug after a speedy review and before its March 8 deadline.
The F.D.A. has been under pressure from some patient advocates who contend that it demands too much data to approve drugs for cancer, potentially allowing people to die. Other advocates, however, say the F.D.A. needs to maintain a high bar to make sure drugs are truly safe and effective and do not offer false hopes.
Those differences came to a head over the agency’s decision in November to revoke the approval for another Genentech drug, Avastin, as a treatment for breast cancer.
The wholesale price of Erivedge will be $7,500 a month, or about $75,000 for the 10-month course of treatment that was typical in the clinical trial, according to a Genentech spokeswoman.
The company did not mention the price in its news release announcing the approval. That seems to be a fairly common practice among drug companies, perhaps to deflect attention from the costs of medicines.
Erivedge can cause birth defects or stillbirths, the F.D.A. said. The most common side effects included muscle spasms, hair loss, nausea, diarrhea, and problems with tasting.
Known generically as vismodegib, Erivedge is a capsule taken once a day. Genentech developed Erivedge with help from Curis, a small company in Lexington, Mass., that will receive a $10 million payment for the approval and a small royalty on sales of the drug.
Curis shares, which have been rising in the last few months, were down 7 percent to $4.80 as of about 3:30 p.m. Monday.
Erivedge works to inhibit the so-called Hedgehog signaling pathway by binding to a protein called Smoothened. The Hedgehog pathway is important in regulating growth and development early in life but gets less active in adulthood, according to Genentech. But in basal cell carcinoma signaling in this pathway is frequently abnormal.
On Friday, another company, Infinity Pharmaceuticals, stopped a midstage trial of a Hedgehog pathway inhibitor it was developing to treat pancreatic cancer after patients getting the drug had a shorter survival time than those getting a placebo. But the company said it would continue to test the drug, saridegib, for other types of cancer.