Bayer HealthCare receives Orphan Drug Designation for its Anti-Cancer Agent in the U.S. for the Treatment of Mesothelioma

Bayer HealthCare announced today that its investigational anti-cancer agent BAY 94-9343, a mesothelin-targeting antibody-drug conjugate (ADC), has been granted orphan drug status for the treatment of patients with mesothelioma by the U.S. Food and Drug Administration (FDA).

This investigational agent is currently in Phase I clinical development.

‘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. ‘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.’

In September 2011, Bayer started the enrollment of patients in an open label Phase I dose escalation study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BAY 94-9343 for the treatment of patients with advanced solid tumors. Patients are currently treated at three study centers in the U.S. (Maryland, Tennessee, and Texas). For more information about this study (NCT01439152), please visit

About BAY 94-9343 (antibody-drug conjugates)

Antibody-drug conjugates (ADCs) comprise antibodies linked to cytotoxic agents, and combine the targeting properties of an antibody with the cell-destroying effect of a potent, small molecule cytotoxic agent.

BAY 94-9343 (Mesothelin-ADC) is an antibody-drug conjugate directed against the target molecule mesothelin which is highly expressed on mesotheliomas and on other cancers, e.g. many ovarian and pancreatic tumors. BAY 94-9343 demonstrated potent, targeted anti-cancer activity against mesothelin-expressing tumors in preclinical models. BAY 94-9343 uses a fully human antibody entity which was generated based on MorphoSys’ HuCAL platform. In addition, BAY 94-9343 is in development under an agreement with ImmunoGen, Inc. that grants Bayer HealthCare rights to use the Company’s maytansinoid TAP (Targeted Antibody Payload) technology. BAY 94-9343 is an investigational agent and is not approved by the FDA, the European Medical Association (EMA) or other Health Authorities.

About Orphan Drug Designation in the United States

In the United States, the Orphan Drugs Act (ODA) provides for the orphan drug designation which aims to encourage the development of drugs involved in the diagnosis, prevention or treatment of a medical condition affecting fewer than 200,000 people in the country. The designation grants U.S. market exclusivity to a drug for a particular indication for a seven-year period if the sponsor complies with certain FDA specifications. Additional incentives for the sponsor include tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee. The designation does not shorten the duration of the regulatory review and approval process.

About Mesothelioma

Malignant mesothelioma is a rare type of cancer. It develops from transformed cells originating in the mesothelium, the protective thin membrane encompassing the body’s internal organs and cavities. There are three recognized forms of malignant mesothelioma: Pleural mesothelioma is the most common type of the disease (roughly 70% of all cases) and occurs in the outer lining of the lungs and internal chest wall, peritoneal mesothelioma occurs in the lining of the abdominal cavity, and pericardial mesothelioma originates in the pericardium, the sac that surrounds the heart.

Malignant mesothelioma is usually caused by exposure to asbestos and the inhalation of asbestos particles. Inhaled or ingested asbestos fibers may cause an inflammation of internal tissue and disrupt organ function leading to the development of mesothelioma. In most cases, the symptoms of mesothelioma such as shortness of breath, lower back pain or side chest pain will only appear years after exposure and are frequently ignored or dismissed. Malignant mesothelioma is extremely aggressive and due to a long latency period cases are often not detected until the disease has reached advanced stages. As a result the prognosis for patients is poor, despite treatment with radiation therapy, chemotherapy and surgery. The survival rate of mesothelioma following diagnosis is usually about two years depending on type, location, and stage.

Learn more about mesothelioma.

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Last update: March 06, 2014. 08:41:31 pm.