FDA Grants Nintedanib Orphan Drug Status to Treat Mesothelioma

FDA Grants Nintedanib Orphan Drug Status to Treat Mesothelioma

The US Food and Drug Administration (FDA) has granted orphan-drug status to nintedanib as a treatment for the asbestos-related cancer mesothelioma.

“Nintedanib, our triple angiokinase inhibitor, has shown promise as a potential treatment for malignant pleural mesothelioma in clinical trials to date, and this designation is a validating milestone in its development,” Martina Flammer, MD, vice president of Clinical Development & Medical Affairs Specialty Care at Boehringer Ingelheim, said in a news release. The designation shows “our ongoing commitment to researching potential treatment options for rare cancers such as mesothelioma,” she said.

Orphan drug status gives companies financial incentives to develop therapies for diseases that affect a limited number of people. Without the incentives, the companies might decide they would lose money on the development effort, and abandon it.

Nintedanib inhibits three types of protein receptors that are crucial to blood-vessel production, but also play roles in tumor growth and cancer spreading to other parts of the body. The receptors are vascular endothelial growth factor receptors 1-3 (VERGFR 1-3), platelet-derived growth factor receptors (PDGFR), and fibroblast growth factor receptors 1-3 (FGFR 1-3).

The FDA designation was based in part on results of Phase 2 of the LUME-Meso trial (NCT01907100). It was designed to assess the safety and effectiveness of nintedanib in combination with Alimta (pemetrexed) or Platinol (cisplatin) versus chemotherapy alone in patients with unresectable malignant pleural mesothelioma. Phase 2 results were presented at the 17th IASLC World Conference on Lung Cancer in Vienna, Austria.

Some Phase 2 participants received nintedanib plus Alimta or Platinol, followed by nintedanib doses alone. Other participants received a placebo plus Alimta or Platinol, followed by placebo maintenance.

A key finding of the Phase 2 results was that it took 3.7 months longer for the cancer of a patient on nintedanib to worsen. Another important finding was that patients on nintedanib live 3.8 months longer than those receiving chemotherapy alone.

The most common adverse event in the treatment group was lower numbers of the immune cell neutrophil. The drop was seen in 34 percent of patients who took nintedanib, compared with only 10 percent in the control group.

Boehringer Ingelheim is recruiting patients for Phase 3 of the LUME-Meso trial, which it hopes will  confirm the promising Phase 2 results.

Nintedanib is already approved in the US and Europe under the brand name Vargatef to treat patients with non-small-cell lung cancer. It is also marketed in the US as Ofev to treat patients with idiopathic pulmonary fibrosis.

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