In fiscal year 2011, FDA approved 7 “new molecular entities” (NMEs) in oncology.
4. Yervoy (ipilimumab) for late-stage melanoma (skin cancer).
Importance: Melanoma is the leading cause of death from skin disease. An estimated 68,130 new cases of melanoma were diagnosed in the
during 2010, and about 8,700 people died from the disease last year, according to the National Cancer Institute. Late-stage melanoma is devastating, with very few treatment options for patients. Prior to this year, none of the existing treatments prolonged a patient’s life. Yervoy, the first in a new class of immune-boosting drugs, was the first drug approved by FDA that was clearly demonstrated to prolong the life of patients with late-stage melanoma. United States
Actions to speed drug testing and review: Yervoy was given a Fast Track designation for its potential to address an unmet medical need—prolonging survival in patients with metastatic melanoma. Yervoy’s safety and effectiveness were established in a single international trial of 676 patients. All patients in the trial had stopped responding to other FDA-approved or commonly used treatments for melanoma. Patients who received Yervoy alone, or in combination with an experimental vaccine, lived an average of about 10 months, while those who received only the experimental vaccine lived an average of 6.5 months. Yervoy was reviewed under the FDA’s priority review program which provides for an expedited six-month review of drugs.
Safety issues: Yervoy poses a risk of serious side effects, including severe to fatal autoimmune reactions in 12.9 percent of patients treated with Yervoy, as well as a range of milder side effects. FDA decided that the benefits of Yervoy outweigh its risks, because no other treatment had been shown to prolong a patient’s life. FDA determined, however, that a Risk Evaluation and Mitigation Strategy (REMS) was required to inform health care professionals and patients about these serious risks.
Source: FDA - FY 2011 Innovative Drug Approvals