FDA News

FDA grants orphan drug designation to Imfinzi for small cell lung cancer

Photo of Jose Baselga
José Baselga

The FDA granted orphan drug designation to durvalumab for the treatment of small cell lung cancer.

Durvalumab (Imfinzi, AstraZeneca) — a human monoclonal antibody that binds to PD-L1 — already is approved in the United States for treatment of patients with unresectable, stage III non-small cell lung cancer who underwent chemotherapy and radiation. The agent also is approved for previously treated patients with advanced bladder cancer.

The phase 3 CASPIAN trial compared the combination of durvalumab, standard etoposide and platinum-based chemotherapy with chemotherapy alone as treatment for patients with extensive-stage small cell lung cancer.

The trial met its primary endpoint, with patients assigned the combination achieving a statistically significant and clinically meaningful improvement in OS, according to an AstraZeneca-issued press release. Full results will be presented at an upcoming medical meeting.

“This orphan drug designation comes on the heels of positive results from the phase 3 CASPIAN trial, which is the first trial to offer the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer,” José Baselga, MD, PhD, executive vice president for research and development for oncology at AstraZeneca, said in the release. “We are eager to expand treatment options for patients facing such a devastating diagnosis and look forward to working with regulatory authorities to bring forward new options as soon as possible.”

The phase 3 ADRIATIC trial is evaluating durvalumab with concurrent chemoradiation as therapy for patients with limited-stage small cell lung cancer.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

Photo of Jose Baselga
José Baselga

The FDA granted orphan drug designation to durvalumab for the treatment of small cell lung cancer.

Durvalumab (Imfinzi, AstraZeneca) — a human monoclonal antibody that binds to PD-L1 — already is approved in the United States for treatment of patients with unresectable, stage III non-small cell lung cancer who underwent chemotherapy and radiation. The agent also is approved for previously treated patients with advanced bladder cancer.

The phase 3 CASPIAN trial compared the combination of durvalumab, standard etoposide and platinum-based chemotherapy with chemotherapy alone as treatment for patients with extensive-stage small cell lung cancer.

The trial met its primary endpoint, with patients assigned the combination achieving a statistically significant and clinically meaningful improvement in OS, according to an AstraZeneca-issued press release. Full results will be presented at an upcoming medical meeting.

“This orphan drug designation comes on the heels of positive results from the phase 3 CASPIAN trial, which is the first trial to offer the flexibility of combining immunotherapy with different platinum-based regimens in small cell lung cancer,” José Baselga, MD, PhD, executive vice president for research and development for oncology at AstraZeneca, said in the release. “We are eager to expand treatment options for patients facing such a devastating diagnosis and look forward to working with regulatory authorities to bring forward new options as soon as possible.”

The phase 3 ADRIATIC trial is evaluating durvalumab with concurrent chemoradiation as therapy for patients with limited-stage small cell lung cancer.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

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