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

XTalks

Neurocrine Biosciences Crenessity (crinecerfont) has been given the green light by the US Food and Drug Administration (FDA) as a new treatment for congenital adrenal hyperplasia (CAH). CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens.

Genetics 116
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Can the FDA keep the momentum going for rare disease drug approvals?

Pharmaceutical Technology

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. Major pharmaceutical acquisitions have taken place in recent months in the rare disease space, as the number of orphan drug approvals continues to grow. This was not always the case. “Up

Drugs 317
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New ‘Mechanical’ Weight Loss Pill from Oxford Medical Products Shows Positive Safety Data

XTalks

Unlike drugs that alter metabolism or appetite by interacting with hormones, the pill functions entirely mechanically. This approach aims to help users control portions and calorie consumption without the need for surgery, such as gastric banding or bypass, or the side effects commonly associated with appetite-suppressing drugs.

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Roche Invests $5.3B in Zealand’s Petrelintide, an Obesity Drug Candidate

XTalks

Amylin, a hormone co-secreted with insulin, plays a key role in regulating appetite by enhancing leptins effects the hormone that signals fullness after eating. Petrelintide, Zealand Pharmas long-acting amylin analog, is currently in Phase II clinical development and is designed for once-weekly subcutaneous injections.

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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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Eneboparatide Advances in Hypoparathyroidism Trial: Key Phase III Update

XTalks

Eneboparatide (AZP-3601), an investigational parathyroid hormone 1 receptor (PTH1R) agonist, met its primary endpoint in the CALYPSO Phase III trial, offering new data for adults with chronic hypoparathyroidism. Without proper PTH function, patients may face kidney and bone complications.

Trials 59
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Obesity Drug Petrelintide Advances into Phase IIb

XTalks

The study will assess the drugs effects on body weight, safety and tolerability over a 42-week period. Petrelintide, administered once a week under the skin, targets obesity through its action as an amylin analog, a hormone co-secreted with insulin from the pancreas.

Drugs 102