On September 21, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to alisertib, a selective, small-molecule, orally administered inhibitor of aurora kinase A, for the treatment of patients with small cell lung cancer (SCLC) (Press release, Puma Biotechnology, SEP 21, 2023, View Source [SID1234635327]). SCLC is an aggressive form of lung cancer with a poor prognosis, and with limited treatment options for patients whose cancer has progressed on or after platinum-based chemotherapy.

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"Obtaining Orphan Drug Designation from the FDA signifies our continued progress and commitment to the development of alisertib for the treatment of small cell lung cancer," said Alan H. Auerbach, Chief Executive Officer, President and Founder of Puma. "There is an urgent need for new treatments for patients with small cell lung cancer, and we look forward to the initiation of our Phase II trial (Study PUMA-ALI-4201) of alisertib in small cell lung cancer."

The FDA grants Orphan Drug Designation to investigational therapies being developed to treat, diagnose or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Further, Orphan Drug Designation provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified trials, waiver of certain FDA fees, and the potential for seven years of post-approval marketing exclusivity.

Puma received FDA clearance of its Investigational New Drug application for the clinical development of alisertib monotherapy for the treatment of patients with extensive stage SCLC in August 2023 and anticipates initiating the Phase II trial in the second half of 2023.