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Boehringer Ingelheim: FDA Approves Orphan Drug For NSCLC

July 16, 2013

Boehringer Ingelheim (BI) announces on July 12, 2013, that the FDA approves orphan drug Gilotrif (Afatinib) as a new first-line treatment for patients with metastatic Non-Small Cell Lung Cancer (NSCLC) with common Epidermal Growth Factor Receptor (EGFR) mutations. The EGFR mutation is to be detected by an FDA-approved test. Gilotrif receives FDA Orphan Drug Designation (ODD) in December 2012 and the FDA evaluates Gilotrif under its Priority Review program. The drug is the first FDA-approved oncology product from BI.

Gilotrif receives approval based on a study of 345 participants with metastatic NSCLC whose tumors had the EGFR mutation. The study’s data shows that Gilotrif has a delay in tumor growth (Progression-Free Survival or PFS) that was 4.2 months later than those participants receiving chemotherapy. There is no statistically significant difference in overall survival.

According to Berthold Greifenberg, M.D., Vice President of Clinical Development and Medical Affairs, BI Oncology:

“The approval of Gilotrif offers a new treatment option and provides a personalized treatment approach for patients with EGFR mutation-positive metastatic NSCLC …”.

Patients must have biomarker testing to determine if a common EGFR mutation is present. BI collaborates with Qiagen, a Netherlands holding company specializing in sample and assay technologies, to develop a companion diagnostic for Gilotrif. Qiagen’s therascreen EGFR RGQ PCR Kit is approved on July 12, 2013, by the FDA in parallel to Gilotrif and will be used to identify patients who may be eligible for treatment.

Including the biomarker test with an orphan drug is very useful for maintaining the drug’s share of the market in the face of competition. For example, Pfizer’s orphan drug Xalkori (Crizotinib) is developed in parallel with a companion diagnostic to identify the presence of the ALK fusion gene in a subset of NSCLC patients. Pfizer partnered with Abbott Molecular to develop the test. The Vysis ALK Break Apart FISH Test receives FDA approval at the same time that Xalkori receives FDA approval in August 2011.

Recently, two FDA approved orphan drugs are indicated for patients with a specific genetic mutation, where the drug is known to be effective :

  1. Vemurafenib (Zelboraf) for Melanoma with the BRAFV600E mutation as detected by an FDA-approved test
  2. Kalydeco (Ivacaftor) for Cystic Fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.

Alberto Gutierrez, Ph.D., Director of the Office of In Vitro Diagnostics and Radiological Health, in the FDA’s Center for Devices and Radiological Health, says in a Press Release:

“The approval of companion diagnostic tests and drugs are important developments in oncology, as they help us bring safe and effective treatments to patients who need them”.

Companies developing orphan drugs are also establishing biomarkers and developing the companion diagnostic test so that it can help in selecting patients for clinical trials. The diagnostic test may be approved simultaneously as part of a conditional drug approval. Biopharmaceutical companies should investigate the use of biomarkers to identify new orphan indications as part of the Orphan Drug Company Life Cycle Management Strategy.

Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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