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FDA approves Alnylam’s ultra-rare disease drug Oxlumo

pharmaphorum

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The post FDA approves Alnylam’s ultra-rare disease drug Oxlumo appeared first on.

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

Novaliq, a biopharmaceutical company specializing in ocular therapeutics, recently announced that their product Vevye (cyclosporine ophthalmic solution) 0.1 percent received approval from the US Food and Drug Administration (FDA) to treat dry eye disease. This mechanism reduces eye swelling and promotes tear production.

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World RNA Day: What impact RNA it had on drug discovery?  

Drug Discovery World

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

RNA 52
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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

There was some steady progress in neurology – in February FDA approved Lundbeck’s eptinezumab prophylactic treatment for migraine, the last from a gang of four drugs from a new class. Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets.

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FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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Two biotechs target dry eye disease after Novartis’ Xiidra problems

pharmaphorum

billion for its FDA-approved dry eye drug Xiidra during the Japanese pharma’s merger with Shire in 2019, outlining the sales potential in this market niche. Sylentis, part of PharmaMar Group, has just announced FDA approval of a phase 3 trial for eye drops containing tivanisiran, in dry eye disease associated with Sjogren’s Syndrome.