New gene tests may give cancer patients quicker path to treatment – BY JULIE STEENHUYSEN CHICAGO June 6 , 2014 1:45am EDT

A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.   REUTERS/Jason Reed

A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.


A view shows the U.S. Food and Drug Administration (FDA) logo at the lobby of its headquarters in Silver Spring, Maryland August 14, 2012.

Credit: Reuters/Jason Reed

(Reuters) – A new way of evaluating tumors may soon help cancer patients identify the underlying genetic link to their disease – and the best possible treatment – all in a single test.

Researchers are set to begin clinical trials using a more comprehensive testing method that looks for all of the known genes that may be active in a tumor.

The new method could guide patients to the right drug earlier, potentially replacing current tests known as companion diagnostics that only look for a specific biological trait or “biomarker.” The presence of a biomarker can predict whether a new class of drugs called targeted therapies will work on particular tumors.

Results of these broader tests could even be used to quickly identify which patients might benefit from experimental drugs being tested in clinical trials. U.S. health officials see it as the future direction of cancer diagnostics.

“We really are moving away from this one drug, one biomarker, one companion diagnostic,” said Dr Richard Pazdur, the U.S. Food and Drug Administration’s oncology chief.

In advanced melanoma, for example, about half of patients’ tumors have a mutation in the BRAF gene. Roche makes a drug called Zelboraf that blocks that pathway, at least for a time. To get Roche’s drug, patients need to be evaluated with an FDA-approved companion diagnostic test. One of the tests is also made by Roche.

In many cases, the FDA requires single-biomarker companion diagnostics as part of the drug approval process, but the broader testing model opens the door to additional players in the diagnostics space, including U.S.-based Foundation Medicine Inc and Thermo Fisher’s Life Technologies.

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U.S. FDA approves Novo Nordisk’s drug for rare blood clotting disorder Mon Dec 23, 2013 4:11pm EST

(Reuters) – The U.S. Food and Drug Administration said on Monday it has approved Novo Nordisk’s drug Tretten to prevent bleeding in patients with a rare blood clotting disorder.

 Screen Shot 2013-12-24 at Dec 24, 2013 5.38

Patients with congenital Factor XIIIA-subunit deficiency do not make enough of the Factor XIII protein that is important for normal blood clotting, the FDA said. Factor XIII is composed of subunits A and B and Factor XIII deficiency is usually caused by a deficiency of the A subunit.

Tretten was studied in 77 patients with the disorder and was effective in preventing bleeding in 90 percent of the patients when given monthly, the FDA said. Side effects included headache, pain in the extremities and at the injection site.

Congenital Factor XIII affects an estimated one in 3 million people globally and is associated with life-threatening bleeding, impaired wound healing and miscarriage.

The drug is expected to generate sales of $84 million by 2018, according to the average estimate of three analysts polled by Thomson Reuters.

Novo Nordisk acquired the rights to Tretten from ZymoGenetics Inc in 2004. Novo agreed to pay a royalty on sales, although details of the royalty payment were not disclosed. ZymoGenetics was acquired by Bristol Myers Squibb Co in 2010.

The product was approved last year in Europe and Canada. It is sold in Europe under the brand name NovoThirteen and contains the active substance catridecacog, which is structurally the same as the human Factor XIII A subunit. It is produced by genetically engineered yeast cells.

(Reporting by Toni Clarke in Boston; Editing by Alden Bentley and Bill Trott)

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