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Opinion: The rare pediatric disease voucher program creates new treatments. I have new data to prove it

STAT

The Food and Drug Administration’s rare pediatric disease priority review voucher program , which has been providing incentives for lifesaving innovations since 2012, is doomed to disappear unless Congress reauthorizes it before the end of September. I have data showing it works. None worked. Read the rest…

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Vertex gets EU okay to treat younger cystic fibrosis patients

pharmaphorum

The new EU approval comes after the FDA approved the expanded label last year, and provides the first treatment option that tackles the underlying cause of CF in patients aged six to 11 with these mutations. Together these drugs cover the CFTR mutations seen in around half of all CF patients.

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How cinnamon lowers blood sugar

The Checkup by Singlecare

A small 2012 study demonstrated similar antidiabetic properties when participants took 1,000 milligrams of cinnamon per day over 12 weeks. Additionally, some research demonstrates the heart health benefits of cinnamon—a good thing for people with diabetes who are at greater risk of cardiovascular disease.

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Bad news for Pfizer as blockbuster Xeljanz flunks safety study

pharmaphorum

JAK inhibitor Xeljanz is one of Pfizer’s top-selling drugs, despite a ‘black box’ warning for blood clots and cancers added to its label in 2019. Now, a study designed to prove its safety has achieved the opposite.

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EC approves first-line ovarian cancer treatment

European Pharmaceutical Review

The safety profile for rucaparib observed in the clinical trial was consistent with current US and European labels for rucaparib. Since most patients have an initial response to the treatment, 80 percent will experience recurrence and require subsequent therapies, according to a 2012 paper published in Annals of Oncology.

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The Guardian view on cystic fibrosis treatments: a rollercoaster ride for the sick | Editorial

The Guardian - Pharmaceutical Industry

Its groundbreaking Kalydeco drug was licensed in Europe in 2012 and useful at first to only a small percentage of sufferers. Kaftrio is the latest to be labelled unaffordable for a cash-strapped NHS. And they have been plunged to the depths when those new drugs have been denied them because of their cost. Continue reading.

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Daiichi Sankyo grabs first approval for blood cancer drug Ezharmia

pharmaphorum

The Ministry of Health, Labour and Welfare (MHLW) in Japan has cleared Ezharmia (valemetostat tosylate) based on the results of an open-label phase 2 trial which revealed a 48% overall response rate with the drug in previously-treated ATL patients.