Company to File Drug Application Despite FDA Refusal

Pharmacyclics will file a formal protest to require the FDA to review its new drug application (NDA) for its brain cancer treatment.

The agency refused to review the company's NDA for Xcytrin, a treatment for brain tumors in lung cancer patients, because a key study did not meet its primary endpoint, the company said.

Pharmacyclics will file its request for review under protest around April 30, the company said. The FDA then has several months to review the NDA.

Pharmacyclics submitted the NDA to the FDA's Division of Drug Oncology Products in December 2006. In February, the FDA issued a "refuse to file" letter because a study did not demonstrate statistically significant differences in the treatment arms for the primary endpoint, slowing neurological damage.

Xcytrin, along with radiation, slowed some symptoms in brain tumor patients for 5.4 months more than those receiving radiation only, Pharmacyclics said. The difference was not statistically significant, but the drug could still benefit thousands of patients who lack other options, the company added.

No other treatment has been shown to provide neurologic benefits to patients with brain tumors from lung cancer, Pharmacyclics said.

The FDA has reviewed other drugs whose primary endpoints in studies did not show statistical significance, Pharmacyclics President Richard Miller said. The reviews sometimes lead to approval, especially when there are few treatment alternatives and the drug is well-tolerated, he added.

An integrated analysis of two Phase III studies on Xcytrin, which was included in the NDA, showed a statistically significant delay of 6.4 months in neurologic damage for patients taking the drug, the company said. ( http://www.fdanews.com/did/6_67/ )