New Treatment for Rare Skin Cancer
Erivedge Works in Advanced Basal Cell Carcinoma; May Help Other Cancers
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Erivedge Effective for Advanced Basal Cell Carcinoma continued...
Erivedge approval was based on a phase 2 trial. Larger, placebo-controlled phase 3 studies usually are needed for approval. But the FDA was impressed by the study results:
- Among 63 patients with locally advanced BCC, 13 had complete remissions.
- Overall, 43% of the patients with locally advanced BCC responded to treatment, usually within months.
- Among 33 patients with metastatic BCC, 30% responded to treatment.
Another study appearing in NEJM looked at people with a rare genetic mutation that causes them to develop hundreds or even thousands of BCC tumors. It's called basal-cell nevus syndrome or Gorlin syndrome. Erivedge cut the average number of new tumors per year from 29 to two, and reduced the size of existing tumors.
It would have been even better if all patients were fully cured. But for a cancer drug, the results are remarkable.
Erivedge Side Effects, Cost
There is of course a downside. Hedgehog is an important signaling pathway. Blocking it causes serious side effects. Overall, 12% of patients in the advanced BCC study had to stop taking the drug. And seven patients died during the clinical trial, although none of the deaths were attributed to side effects.
The Erivedge label carries a "black box" warning of the potential risks of death and severe birth defects if the drug is taken by pregnant women. Side effects of Erivedge include:
Muscle spasms, some of them severe.
- Altered taste. "Food becomes tinny tasting," Oro says. "Often we see weight loss because patients don't eat as much. For cancer patients, this is not a good thing."
Hair loss (strong hedgehog signaling is needed to maintain hair growth).
- Weight loss.
Has Erivedge approval made a big impact on cancer treatment? It's too soon to say, given that relatively few patients develop advanced BCC.
"The new medication will not affect the common approach to basal cell carcinoma treatment, which is surgery," Brown University's Martin Weinstock, MD, PhD, tells WebMD. "It is the very unusual case that would merit treatment with this." Weinstock was not involved in the Erivedge studies.
Erivedge treatment costs about $75,000 for a 10-month course of treatment.