Exelixis Inc. (EXEL:US) won U.S. regulatory approval for cabozantinib to treat thyroid cancer patients, giving the company its first marketed product.
The Food and Drug Administration cleared the drug, to be known as Cometriq, for medullary thyroid cancer, the agency said today in a statement. The drug extended progression free survival for patients in a Phase 3 study to 11.2 months compared with four months with a placebo, South San Francisco, California-based Exelixis said in June.
Exelixis also is studying cabozantinib as a treatment for prostate cancer and today’s approval is the first in a series of diseases the drug may work against, Michael Morrissey, the company’s chief executive officer, said at a conference.
“I think the true value of cabo can be viewed in terms of it as a franchise, having franchise potential across multiple indications outside of the initial potential indication around thyroid cancer,” Morrissey said according to a transcript of the Nov. 15 conference. “Certainly the vast majority of our Phase 2 work over the last few years has been focused in other tumor indications.”
The treatment will carry a boxed warning alerting patients about the possibility of experiencing severe and fatal bleeding and holes in the colon, the FDA said today.
In addition to prostate cancer, Exelixis is testing cabozantinib in breast, ovarian and renal cancers, as well as solid tumors, non-small cell lung cancer and multiple myeloma. The treatment is the FDA’s 31st new drug approval this year, surpassing a seven-year high of 30 approvals in 2011.
This is the second drug in two years the agency has approved to treat medullary thyroid cancer, said Richard Pazdur, director of the FDA’s Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research. The first was AstraZeneca Plc (AZN)’s Caprelsa in April 2011.
Cabozantinib is designed to block tumors from growing and spreading by inhibiting two pathways, called MET and VEGF. The thyroid is a hormone-creating gland that regulates blood pressure, body temperature, heart rate and weight.
Exelixis declined less than 1 percent to $5.24 at the close in New York before the FDA’s announcement. The company has gained 27 percent in the past 12 months.
The market for cabozantinib in this indication is limited, said Ted Tenthoff, a New York-based analyst for Piper Jaffray, in a Nov. 26 note to clients. About 4 percent of thyroid cancers are medullary thyroid cancer, making it one of the rarer types, the FDA said in the statement.
About 56,500 new cases of thyroid cancer are diagnosed each year in the U.S., according to the National Cancer Institute. About 1,780 people die annually from the disease.
A year ago Exelixis plunged the most in 11 years after the company and the FDA failed to agree on special rules for studying cabozantinib in prostate cancer patients. Instead, the company began a late-stage trial under a normal regulatory framework. Pivotal data from that study is expected in the first half of 2014, Tenthoff said.
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