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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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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. Xeljanz isn’t the only drug in the class to be affected by toxicity concerns of course.

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FDA firms up JAK inhibitor warnings after Xeljanz review

pharmaphorum

The FDA has concluded its safety review of Pfizer’s JAK inhibitor Xeljanz and Xeljanz XR, requiring revised warnings for the drugs as well as others in the class after finding evidence of elevated risks of serious heart-related events. The post FDA firms up JAK inhibitor warnings after Xeljanz review appeared first on.

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Botanical drugs – what is the best way forward for regulatory and market approval?

European Pharmaceutical Review

In the US, botanical dietary supplements can be sold without US Food and Drug Administration (FDA) approval, 1 prompting many vendors to promote the use of botanical products as dietary supplements , 2 rather than pursue a path of regulatory approval. 3 Is two too few? 3 Is two too few?

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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.

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New Legislation Would Cut Off Access To The Courts And Immunize FDA Actions From Timely Judicial Review

The FDA Law Blog

Senate Committee on Health, Education, Labor and Pensions (“Senate HELP”) is scheduled to take up legislation that could significantly limit access to the courts and immunize critical FDA decisions from timely judicial review. Subsection 505(q) initially was added by Section 914 of the 2007 FDA Amendments Act (“FDAAA”), Pub.