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Keytruda is first FDA-approved drug for molecular indication

Indicates a significant development in the shift towards personalised medicine

Merck & Co MSD Keytruda

Merck & Co's run of good news for PD-1 inhibitor Keytruda shows no sign of letting up. The cancer drug has become the first to be approved by the FDA to treat all cancers with a specific tumour biomarker.

The US regulator's head of haematology and oncology products, Richard Pazdur, said the development is "an important first for the cancer community" as until now cancer drugs have always been approved based on where the cancer started. "We have now approved a drug based on a tumour's biomarker without regard to the tumour's original location," he added.

The new label means Keytruda has been cleared to treat solid tumours that have been identified as having a biomarker referred to as microsatellite instability-high (MSI-H) or its underlying cause, a DNA repair pathway defect known as dMMR.

The biomarker is most commonly seen in colorectal, endometrial and gastrointestinal cancers, occurring in around 5% of all colorectal cancers but is also seen in breast, prostate, bladder, thyroid and other tumour types. All told it is estimated that 1 in 25 tumours display a high degree of microsatellite instability, and diagnostic testing for the biomarker is both cheap and readily available.

"We're talking about 5-10% typically in a variety of different tumour types, so it's not a gigantic opportunity," Merck's R&D head Roger Perlmutter said earlier this month, although he noted that the development was very significant in the context of the shift towards personalised medicine.

Merck has been granted accelerated approval for Keytruda in this indication based on the results of five uncontrolled single-arm clinical trials involving 149 patients, so it will have to provide additional evidence of the drug's efficacy in further studies if it is to keep the indication on the label.

In the initial studies almost 40% of patients saw a partial or complete response with Keytruda at a fixed dose of 200mg every three weeks, and in 78% of them the effect lasted for at least six months.

The new approval comes just hours after Merck claimed an accelerated review for Keytruda as a third-line gastric cancer treatment, and a week after it was approved in first- and second-line bladder cancer. The drug has previously picked up approvals in melanoma, non-small cell lung cancer (NSCLC), head and neck squamous cell cancer and classical Hodgkin lymphoma.

The drug is making big gains even as it faces increasing competition with five drugs in the PD-1/PD-L1 inhibitor class of immuno-oncology drugs, now approved with at least one cancer indication.

Keytruda made $1.4bn for Merck last year adding another $584m to that tally in the first three months of this year. However it still has a way to go before it catches up with current market leader Opdivo (nivolumab) from Bristol-Myers Squibb, which brought in $3.77bn last year and $1.13bn so far this year.

BMS reported data last year showing that Opdivo was also effective in MSI-H colorectal cancer, both alone and in combination with its CTLA4 inhibitor Yervoy (ipilimumab).

Article by
Phil Taylor

24th May 2017

From: Regulatory



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