Washington Editor

OSI Pharmaceuticals Inc. and partner Genentech Inc. completed the submission of a new drug application for Tarceva, a drug for the treatment of patients with advanced non-small-cell lung cancer who have failed chemotherapy.

The NDA has been granted Pilot 1 status under the FDA's Pilot 1 Program for Continuous Marketing Applications, a new program designed for investigational products that have been granted fast-track status and that have demonstrated significant promise in clinical trials as a therapeutic advance over available therapy for a disease or condition, the companies said.

Company officials couldn't be reached for comment.

Tarceva (erlotinib HCl) is a small molecule designed to target the human epidermal growth factor receptor-1 (HER1) pathway, one of the factors critical to cell growth in many cancers. HER1, also known as EGFR, is a key component of the HER signaling pathway, which plays a role in the formation and growth of numerous cancers.

The candidate is designed to inhibit the tyrosine kinase activity of the HER1 signaling pathway inside the cell, which might block tumor cell growth, the companies said.

As reported at the 40th annual American Society of Clinical Oncology meeting in New Orleans in June, the new drug application for Tarceva is based on a pivotal Phase III trial that demonstrated a 42.5 percent improvement in the median survival rate of patients with advanced non-small-cell lung cancer and a 45 percent improvement in one-year survival rates compared to placebo. (See BioWorld Today, June 8, 2004.)

The 731-patient, double-blind, placebo-controlled trial demonstrated statistically significant improvement in all secondary endpoints including time to symptom deterioration, progression-free survival and response rate.

The partners said the study results make Tarceva the first and only targeted therapy to demonstrate an improvement in survival for non-small-cell lung cancer patients.

Adverse events included rash and diarrhea, but were generally mild to moderate in severity.

If Tarceva wins regulatory approval, Genentech, of South San Francisco, and OSI, of Melville, N.Y., would share U.S. profits equally. Genentech would take a lead role in marketing the product, while OSI would provide manufacturing. Roche Holdings Inc. controls foreign rights, while Genentech and OSI would receive royalties for sales outside the U.S. (See BioWorld Today, Jan. 9, 2001.)

Initially, OSI was developing Tarceva with Pfizer Inc., though the Federal Trade Commission forced the New York-based pharmaceutical company to drop the partnership in order to complete its acquisition of Warner-Lambert Co., of Morris Plains, N.J.

OSI's stock (NASDAQ:OSIP) Monday fell $1.72 to close at $58.38, while Genentech's (NYSE:DNA) fell $1.72 to close at $46.96.