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FDA approval to Keytruda and Lenvima; FibroGen’s Roxadustat; Pfizer’s TICOVAC; Ipsen’s Palovarotene; Jazz Pharma’s Xywav; Seagen-RemeGen’s Cancer Medicine

Delveinsight

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36

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FDA Action Alert: Y-mAbs, Vanda, Alnylam and BioCryst

The Pharma Data

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

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FDA Action Alert: Urovant, Athenex, scPharmaceuticals and Vertex

The Pharma Data

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. The FDA approval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. and Europe, including Russia. Most Read Today.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Biopharma Money on the Move: December 2 – 8

The Pharma Data

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. .

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4 Life Sciences Trends for 2023

XTalks

As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.

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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease. Nizoral is prescribed off-label, while the others are FDA approved. Paget’s Disease.

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