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GlaxoSmithKline: FDA Approval Of Two Orphan Drugs Targeting Metastatic Melanoma

May 30, 2013

GlaxoSmithKline (GSK) receives FDA approval on May 29, 2013, for the following two orphan drugs for patients with advanced or unresectable Melanoma :

•   Tafinlar (Dabrafenib)
•   Mekinist (Trametinib).

Both drugs are approved as single agents, not as a combination treatment. Tafinlar, a BRAF inhibitor, is approved for Melanoma whose tumors express the BRAF V600E gene mutation. Mekinist, a MEK inhibitor, is approved for Melanoma whose tumors express the BRAF V600E or V600K gene mutations. Both drugs are approved with a companion genetic test, ThxID BRAF test, made by France’s bioMérieux, which will help determine if a patient’s Melanoma cells have the V600E or V600K mutation in the BRAF gene. About 50% of Melanomas arising in the skin have a BRAF gene mutation. Even though both drugs are approved as single products, GSK has :

“ … high hopes for ongoing studies that test how a combo approach could delay recurrence of cancer in high-risk patients …. GSK races with Roche/Genentech to advance combination therapies against melanoma … Genentech, the US biotech unit of Roche, is doing late-stage testing of a rival dual therapy consisting of the experimental MEK inhibitor GDC-0973 from its partner Exelixis and its approved BRAF inhibitor Zelboraf.”

In 2013, approximately 77,000 Americans will be diagnosed with Melanoma and 9,480 will die from the disease (National Cancer Institute.

Trafinlar and Mekinist are the third and fourth new drugs approved by the FDA for metastatic Melanoma in the past two years. The other two drugs are approved in 2011 for metastatic or unresectable Melanoma :

•   Genentech’s orphan drug Zelboraf (Vemurafenib)
•   Bristol-Myers Squibb’s orphan drug Yervoy (Ipilimumab).

According to a GSK spokesperson, per a article, in the US, the following are the wholesale acquisition costs for the two drugs :

•   Tafinlar  is $7,600 for 30-day supply
•   Mekinist is $8,700 for 30-day supply.

According to analysts at Cowen and Company, sales are forecast to reach approximately $350 million/drug in 2016.

Inclusion Of Biomarker Test With Orphan Drugs

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 Xalkori (Crizotinib) is developed in parallel with a companion diagnostic to identify the presence of the ALK fusion gene in a subset of non-small cell lung cancer (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.

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. More biopharmaceutical companies are starting to investigate the use of biomarkers to identify new orphan indications as part of the Orphan Drug Company Life Cycle Management Strategy.


FDA’s May 29, 2013 Press Release

GlaxoSmithKline’s May 29, 2013 Press Release.

Please Note : Please Note: FDA Official Logo from FDA website.

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

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