Bayer HealthCare Receives Orphan Drug Designation for Agent Used in the Treatment of Mesothelioma
Bayer Healthcare announced this week that its experimental drug, BAY 94-9343, has been granted orphan drug status for the treatment of patients with mesothelioma by the U.S. Food and Drug Administration (FDA). The investigational anti-cancer agent, a mesothelin-targeting antibody-drug conjugate (ADC), is currently in a Phase I clinical trial to evaluate the safety, tolerability and maximum tolerated dose in mesothelioma patients with advanced solid tumors.
“This is an important step in the development process of this investigational agent,” said Prof. Andreas Busch, Member of the Bayer HealthCare Executive Committee and Head of Global Drug Discovery in a press release. “Malignant mesothelioma is a rare type of cancer with a poor prognosis. There is a significant unmet medical need in this patient population for new treatment options. Bayer is committed to develop therapies that can help people with cancer live longer and improve their quality of life.”
According to Bayer, BAY 94-9343 (Mesothelin-ADC) is an antibody-drug conjugate directed against the target molecule mesothelin which is highly expressed in mesothelioma and in other cancers. In a separate study, researchers found the mesothelin biomarker is “relatively elevated” in close to 40% of mesothelioma patients prior to diagnosis.
Last year Pinnacle Biologics obtained an orphan drug designation for its product PHOTOFRIN® (porfimer sodium) as adjuvant therapy to surgery in the treatment of malignant pleural mesothelioma.
The FDA notes that the Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
Mesothelioma is diagnosed in close to 3,000 Americans each year, and nearly the same number die from the asbestos-caused disease. There are nearly 7,000 orphan diseases in the United States.