Two new drugs approved, one pulled from the pipeline

Skin Cancer

Ahead of its March 8 deadline, the Food and Drug Administration (FDA) approved Erivedge (vismodegib, GDC-0449) on Jan. 30 for patients with locally advanced basal cell carcinoma (BCC) that can't be treated with surgery or radiation or who have metastatic BCC, making Erivdege the first drug approved by the FDA for metastatic BCC.

In another first, Erivedge is the first drug approved that works by inhibiting the Hedgehog pathway, which is involved in controlling cancer cell division and active in most BCCs. The drug is a pill taken once a day.BCC, along with squamous cell carcinoma, is a non-melanoma skin cancer and one of the most common types of skin cancer, with an estimated 3.5 million cases diagnosed each year. Advanced BCC is rare, however, and disfiguring, which is why the drug fills an unmet need in this patient population.

Last April, we covered some of the research about Erivedge (then known as GDC-0449) and even the first patient to try the drug.

The approval in based on a multicenter trial with 96 patients. Of those with metastatic BCC, 30 percent had partial tumor shrinkage, and of those with locally advanced BCC, 43 percent had partial or complete tumor shrinkage. Side effects include nausea, fatigue, hair loss, diarrhea, changes in taste and weight loss. Some serious side effects may occur, so a boxed warning will be included on the drug alerting physicians to potential risk of death or birth defects for an unborn baby. Doctors are required to verify pregnancy status before starting treatment.

A month's supply should cost $7,500, and Genentech (the drug's manufacturer) estimates a treatment course will last 10 months, totaling $75,000.

The Patient Action Network Foundation has announced they will now offer co-payment assistance up to $7,500 per year for out-of-pocket expenses. To see the eligibility guidelines and for more information about the program, visit the Patient Action Network Foundation. This drug should be available in one to two weeks, per the manufacturer.For more information about Erivedge, visit www.erivedge.com.

Kidney Cancer

Just a few days before, on Jan. 27, the FDA also approved Inlyta (axitinib) for patients with advanced renal cell carcinoma (kidney cancer) who haven't responded to previous treatment.

Inlyta targets the vascular endothelial growth factor (VEGF) pathway, which is important to the development of new blood vessels in tumors, which helps tumors grow. The approval follows a 723-patient trial in which patients on Inlyta had a median progression-free survival of 6.7 months compared with the 4.7 months on the standard treatment, Nexavar (sorafenib).Inlyta is a pill taken twice daily and is expected to cost around $8,900 per month.

Side effects include diarrhea, fatigue, high blood pressure, decreased appetite and nausea. Because it can cause high blood pressure, individuals with this condition should have it controlled before taking Inlyta. Also, sometimes serious bleeding problems can occur, so patients with untreated brain tumors or gastrointestinal bleeding should not take Inlyta.For more details, visit www.inlyta.com.

Non-Hodgkin Lymphoma

Finally, pixantrone was pulled from the FDA approval pipeline earlier this week. In 2010, the FDA rejected the drug, but Cell Therapeutics (its manufacturer) appealed and resubmitted the drug for approval as a treatment for non-Hodgkin lymphoma patients who no longer responded to other therapies. An FDA advisory meeting was set for Feb. 9, with a possible approval in April, but the manufacturer pulled the application to allow the company more time to prepare for the review. It plans to resubmit later this year.