Treatments in the Pipeline

The Food and Drug Administration (FDA) approved Erivedge (vismodegib), on Jan. 30 for patients with locally advanced basal cell carcinoma (BCC) that can’t be treated with surgery or radiation; and for metastatic BCC, making Erivedge the first drug approved by the FDA for metastatic BCC. In another first, Erivedge is the only drug approved that works by inhibiting the Hedgehog pathway, which is involved in controlling cancer cell division and is active in most BCCs. The drug is a pill taken once a day.

The approval is 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, taste changes and weight loss. Some serious side effects may occur, so a boxed warning will be included on the drug package alerting physicians to the potential risks of death or birth defects for an unborn baby.

For details, visit erivedge.com or call 888-249-4918.

On Jan. 27, the FDA 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, helping 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 4.7 months on standard treatment, Nexavar (sorafenib).

Inlyta is a pill taken twice daily. 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 details, visit inlyta.com or call 877-744-5675.

For those who receive intravenous Velcade (bortezomib) injections for multiple myeloma or mantle cell lymphoma, there is now a new way to administer the drug. On Jan. 23, the FDA approved Velcade for subcutaneous (under the skin) injections.

The new method of administration results in fewer side effects, especially damage to the peripheral nerves, which leads to peripheral neuropathy (sensations of pain, numbness or tingling). In a company-sponsored trial, patients experienced less peripheral neuropathy when they had injections under the skin, 38 percent compared with 53 percent for intravenous injections.

The FDA OK’d expanded usage of Gleevec (imatinib) on Jan. 31 for treating gastrointestinal stromal tumors (GIST).

Label updates includes information from a large study that showed patients who took Gleevec for three years had better overall survival (92 percent after five years) compared with those who took Gleevec for one year (82 percent at the five-year mark).

Initially, Gleevec received accelerated approval for advanced or unresectable GIST, and was then approved for adjuvant therapy after surgical removal of GIST.

Also, Gleevec received approval for patients with surgically removed CD117-positive GIST.

Due to two more cases of progressive multifocal leukoencephalopathy (PML), a new boxed warning has been added to the label of the lymphoma drug Adcetris (brentuximab vedotin). PML is a rare but serious infection that damages nerves in the brain and can be lifethreatening.

A warning against using Adcetris with bleomycin due to increased risk of lung toxicity was also added to the label.

Pixantrone was pulled from the FDA approval pipeline in January. The FDA rejected the drug in 2010, but the drug’s manufacturer appealed and resubmitted it 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.

On Jan. 17, the FDA approved Voraxaze (glucarpidase) for patients with toxic levels of methotrexate in their blood after kidney damage.

Methotrexate is used to treat many cancers, in combination or alone, and is normally passed from the body through the kidneys. However, high doses can cause kidney damage, raising the level of the drug in the system. Voraxaze breaks down methotrexate so it can then be eliminated from the body.

Side effects include low blood pressure, headache, nausea and vomiting.

On the pain front, another form of fentanyl received FDA approval for breakthrough cancer pain on Jan. 4. Breakthrough cancer pain is a pain flare that “breaks through” even when the patient is already taking pain relief medication. Subsys is an under-the-tongue spray approved for patients who are tolerant to opioid medications used for persistent pain.

Side effects are similar to other forms of fentanyl and include vomiting, nausea and constipation.