AstraZeneca (NYSE: AZN) today announced that the U.S. Food and Drug Administration (FDA) has approved the trade name Caprelsa for the recently approved orphan drug vandetanib, a treatment for medullary thyroid cancer that cannot be removed by surgery or that has spread to other parts of the body.
Caprelsa is a kinase inhibitor indicated for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable (non-operable) locally advanced or metastatic disease. The use of Caprelsa in patients with indolent, asymptomatic or slowly progressing disease should be carefully considered because of the treatment-related risks.
The FDA approved vandetanib on April 6, 2011 and AstraZeneca made the product available to U.S. patients before it received a trade name. The product is now available under the trade name Caprelsa.
“We chose to launch the drug as vandetanib without waiting for a trade name approval because there were no other FDA-approved medicines available for people with this rare type of thyroid cancer,” said Eric Vogel, Executive Director of Oncology. “AstraZeneca is committed to bringing meaningful medicines to those who need them most, and Caprelsa is an important part of that commitment.”
The approval of Caprelsa was based on the results of the ZETA study, a Phase III, double-blind trial that randomized 331 patients with unresectable locally advanced or metastatic medullary thyroid cancer to Caprelsa 300 mg (n=231) or placebo (n=100). In the study, patients randomized to Caprelsa showed a statistically significant improvement in progression-free survival (PFS) when compared to those randomized to placebo (Hazard Ratio [HR]=0.35; 95% Confidence Interval [CI]=0.24, 0.53; p<0.0001).This difference reflects a 65% reduction in risk for disease progression. Median progression-free survival was 16.4 months (95% CI=8.3, 19.7) in the placebo arm and was not reached (95% CI=22.6 months, non-estimatable) in the Caprelsa arm. At the primary PFS analysis, no significant overall survival difference was noted. QT prolongation, Torsades de pointes, and sudden death are included in the boxed warning for Caprelsa. The most common adverse drug reactions (>20%) seen in the ZETA trial with Caprelsa were diarrhea (57%), rash (53%), acne (35%), nausea (33%), hypertension (33%), headache (26%), fatigue (24%), decreased appetite (21%), and abdominal pain (21%).
A Risk Evaluation and Mitigation Strategy (REMS) is required for Caprelsa due to the risks of QT prolongation, Torsades de pointes, and sudden death. Only prescribers who are certified through the Caprelsa REMS program, a restricted distribution program, will be able to prescribe and dispense Caprelsa. To learn about the specific REMS requirements and to enroll in the Caprelsa REMS Program, health care professionals should call 1-800-236-9933 or visit www.caprelsarems.com.
Caprelsa is dispensed exclusively through the pharmacy business unit of Biologics, Inc., an integrated oncology management company.