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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

The therapy is approved to be used in combination with glucocorticoid replacement therapies (steroids) to control androgen levels in adults and pediatric patients four years of age and older with classic CAH. Currently, the standard treatment for CAH involves glucocorticoids, which replace cortisol and help regulate hormone levels.

Genetics 116
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Jesduvroq (daprodustat) is FDA Approved for Anemia due to Chronic Kidney Disease

XTalks

GlaxoSmithKline LLC (GSK) recently announced that the US Food and Drug Administration (FDA) approved Jesduvroq (daprodustat), a new once-a-day oral treatment for anemia due to chronic kidney disease (CKD). The approval is currently only for adult patients who have been undergoing dialysis for at least four months.

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Vykat XR Approved as First Treatment for Excessive Appetite in Prader-Willi Syndrome

XTalks

Soleno Therapeutics has announced the FDA approval of Vykat XR (diazoxide choline extended-release tablets) for the treatment of hyperphagia an intense, persistent hunger in patients with Prader-Willi syndrome (PWS). The approval marks the first FDA-approved therapy specifically targeting hyperphagia in this rare genetic disorder.

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Xtalks Speaks with Astellas Pharma’s VP & Head of BioPharma Development Marci English About Menopause Drug Approval – Xtalks Life Science Podcast Ep. 116

XTalks

This episode features an interview with Marci English, Vice President and Head of BioPharma Development at Astellas Pharma, about the recent FDA approval of the company’s drug Veozah (fezolinetant) for the treatment of moderate to severe hot flashes and night sweats due to menopause.

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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases. T3 is a major hormone in the blood that regulates thyroid levels.

Drugs 317
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New Zepbound TV Commercial Inspires ‘Change’

XTalks

Related: Eli Lillys Zepbound Approved as First Treatment for Obstructive Sleep Apnea The commercial describes Zepbounds mechanism of action as a dual receptor against that works by activating two naturally occurring hormone receptors in [the] body [GLP-1 and GIP-1]. Its changing how much weight I lose, up to 48 lbs, he says.

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Sanofi Wins FDA Nod for Merilog, First Rapid-Acting Insulin Biosimilar

XTalks

Biosimilars are biological products highly similar to an already FDA-approved reference product, with no clinically meaningful differences in terms of safety, purity and potency. Insulin, a hormone produced by the pancreas, allows glucose to enter cells for energy. Approximately 8.4

Insulin 59