Cometriq (cabozantinib) has been approved by the FDA for the treatment of progressive medullary thyroid cancer that has metastasized – spread to other parts of the body.
Medullary thyroid cancer, a rare form of thyroid cancer, starts off in the cells that make calcitonin, a hormone which regulates calcium levels in the blood and bones. Medullary thyroid cancer occurs either in families with specific genetic mutations that can cause at least one type of endocrine system cancer, including thyroid cancer or it may occur spontaneously among people without the genetic fault, the FDA (Food and Drug Administration) explained.
Medullary thyroid cancer can spread to the lymph nodes, lungs and bones. Current treatment includes surgically removing the thyroid as well as the lymph nodes in the neck.
According to the National Cancer Institute, approximately 56,460 people in the USA will be diagnosed with thyroid cancer this year, and 1,780 are expected to die of the disease during the same period.
About 1 in every 25 thyroid cancers are medullary thyroid cancer.
Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said:
“Cometriq is the second drug approved to treat medullary thyroid cancer in the past two years and reflects FDA’s commitment to the development and approval of drugs for treating rare diseases. Prior to today’s approval and the approval of Caprelsa in April 2011, patients with this rare and difficult to treat disease had limited therapeutic treatment options.”
Cometriq’s submission to the Agency for approval was reviewed within six months, under the FDA priority review program. This type of accelerated approval program is designed specifically for medications that offer major advances in treatment, or provide some kind of treatment when no adequate one exists. As Cometriq is designed to treat a rare disease, it also received orphan-product designation.
Abnormal kinase proteins are involved in medullary cancer cell growth and development. Cometriq is a kinase inhibitor. When taking Cometriq, you should fast at least two hours beforehand and 1 hour afterwards. The recommended dose is 140 mg orally, once daily (one 80 mg capsule plus three 20 mg capsules). You should not take Cometriq with food.
The FDA reviewed Cometriq’s safety and effectiveness by examining data on a clinical trial involving 300 participants, all of them with medullary thyroid cancer. Progression-free survival increased significantly among those on Cometriq. In some cases there were reductions in tumor size.
Below are some data showing Cometriq’s superiority over placebo:
- Those on Cometriq survived 11.2 months (average) with no tumor growth
- Patients in the placebo group survived 4 months (average) with no tumor growth
- 27% of those in the placebo group experienced tumor reductions which lasted an average of almost 15 months
- None of the participants in the placebo group experienced tumor reduction
Cometriq contains a Boxed Warning, telling doctors and patients about the risks of severe and fatal bleedings and perforations and fistula in the colon.
Side effects reported by patients taking Cometriq include: constipation, abdominal pain, worsening hypertension, new hypertension, bad taste, loss of hair color, graying of hair, oral pain, fatigue, nausea, loss of appetite, weight loss, hand-foot syndrome (swelling of fingers and toes), pain, redness, soreness and inflammation of the mouth, and diarrhea.
Cometriq is marketed by Exelixis Inc.
Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis, said:
“The approval of COMETRIQ is an important milestone for patients with progressive, metastatic medullary thyroid cancer, their families, and their physicians, as well as for Exelixis. We are grateful to the many patients who participated in the clinical development of COMETRIQ in MTC, and we are committed to making this important new therapy available as quickly as possible.”
Steven I. Sherman, M.D., Naguib Samaan Distinguished Professor in Endocrinology at M.D. Anderson Cancer Center, explained that until the introduction of targeted therapies, not much progress had been made in the treatment of advanced medullary thyroid cancer. He added that “It is gratifying to give these patients a new treatment option that has been shown in clinical trials to improve progression-free survival remarkably by nearly three-fold. The availability of a new therapeutic approach that has the potential to improve patient care and outcomes changes the MTC treatment landscape and provides patients and physicians with a new way to manage the disease.”
Dr. Sherman was a senior researcher in the Phase III study.
Exelixis Inc. announced yesterday that EMA (European Medicines Agency) has accepted for review the MAA (Marketing Authorization Application) for Cometriq for the treatment of progressive, unresectable, locally advanced, or metastatic medullary thyroid cancer. EMA is the regulatory authority for the European Union.
Written by Christian Nordqvist