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FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism

The Pharma Data

Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth. People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. Source link: [link].

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The regulatory round-up: Eight key FDA decisions

Drug Discovery World

Orserdu (elacestrant), Stemline Therapeutics The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February. This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

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Ascendis’s SKYTROFA; MicroTransponder’s VNS System (Vivistim); BioMarin’s Voxzogo; Zimmer-Canary’s Smart Knee Implant; COVID-19 impact on Solid Organ Transplants

Delveinsight

FDA’s Green Flag to Ascendis’s Once-Weekly SKYTROFA for Treatment of Pediatric Growth Hormone Deficiency. Growth Hormone Deficiency , commonly known as dwarfism, can constitute short stature and complications with metabolism, which arise due to inadequate production of growth hormones by the pituitary gland inside the body.

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Top 40 Best-Selling Oncology Drugs in 2023 by 2022 Data

XTalks

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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Top 40 Best-Selling Cancer Drugs in 2023 by 2022 Data

XTalks

Revlimid (Lenalidomide) Revlimid 2022 sales: $9.978 billion Company/Developer: Bristol Myers Squibb Date of first FDA approval: December 27, 2005 Indications Revlimid is FDA-approved for: Myelodysplastic disease (MDS), multiple myeloma, mantle cell lymphoma (MCL), follicular lymphoma and marginal zone lymphoma.

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

XTalks

Often, the potential patient pool for a given trial is small and the unmet need is high, incentivizing drugmakers and regulators to find innovative ways to make game-changing therapeutics available to those who need them most. Symptoms and Etiology: Caused by an overproduction of growth hormone, acromegaly primarily affects adults.

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Truqap (capivasertib), First-In-Class AKT Inhibitor, Approved for Advanced Breast Cancer, Set to Take on Novartis’ Piqray

XTalks

AstraZeneca has won its first-in-class approval for its AKT inhibitor Truqap (capivasertib) in combination with Faslodex (fulvestrant) for treating patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer. It was also given FDA priority review. billion to $1.28

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