OSI Pharmaceuticals, Inc. announced that it has initiated the "rolling" submission of a New Drug Application (NDA) with the US Food and Drug Administration for the use of Tarceva (erlotinib HCl) in the treatment of patients with incurable stage IIIB/IV non-small cell lung cancer (NSCLC) who have failed standard therapy for advanced or metastatic disease. Data from this clinical trial are now expected in the second quarter of this year and additional components of the NDA filing will be provided to the FDA after these data are available. Tarceva is designed to block tumour cell growth by inhibiting the tyrosine kinase activity of the HER1/EGFR receptor thereby blocking the HER1/EGFR signaling pathway inside the cell. Tarceva is currently being evaluated in an extensive clinical development program together with the company's alliance partners at Genentech and Roche.

The "rolling" submission is an FDA provision, available to drug candidates, which have received Fast Track designation, that allows for completed sections of an NDA to be submitted on an ongoing basis. Fast Track status for Tarceva in this NSCLC indication was granted by the FDA in September 2002. OSI has begun its application by submitting the completed pre-clinical and chemistry, manufacturing and controls (CMC) sections of the NDA to the FDA's Division of Oncology Drugs.

"The initiation of the 'rolling' NDA submission under the Fast Track status is a significant step in preparing for the potential registration of Tarceva," stated Colin Goddard, PhD, CEO of the company. "Beginning the FDA filing process now allows us to demonstrate our commitment of working closely with the FDA to ensure Tarceva filing is complete and thorough."

Under the FDA Modernization Act of 1997, the Fast Track status is designed to facilitate the review process by allowing the sponsor to submit sections of an NDA as they become available, before knowing the results of the pivotal clinical trials. Although the FDA has no obligation to begin reviewing sections of the NDA until the final sections of the complete NDA are submitted, the FDA can begin the review of the submitted sections, if resources permit and this can be advantageous to the overall assessment timelines. Fast Track designation is limited to a new drug that is intended for the treatment of a serious and life-threatening condition for which there is an unmet medical need.

This registration study in relapsed NSCLC, also known as BR21, is a worldwide, randomized Phase III trial testing a 150 mg once-daily monotherapy dose of Tarceva versus placebo. The 730-patient study is being conducted in collaboration with the National Cancer Institute of Canada's Clinical Trial Group and completed enrollment in January 2003. The primary endpoint in the study is improvement in patient survival and key secondary endpoints include time to symptomatic deterioration, progression-free survival and response rate.

According to the World Health Organization, there are more than 1.2 million cases worldwide of lung and bronchial cancer each year, causing approximately 1.1 million deaths annually. According to the National Cancer Institute, lung cancer is the single largest cause of cancer deaths in the United States and is responsible for nearly 30 per cent of cancer deaths in the country. NSCLC is the most common form of the lung cancer and accounts for almost 80 per cent of all the cases.