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US FDA approves Vertex’s Orkambi for cystic fibrosis in children

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has approved the expanded usage of Vertex Pharmaceuticals ’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged 12 months to under 24 months. It enrolled 46 children aged one to under two years with the F/F genotype.

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Partnership to advance personalised treatments for long Covid

Drug Discovery World

The partners will first develop and validate genotypic diagnostics that provide clinicians with an accurate view of a patient’s risk of disease; then, using the molecular profile of their underlying disease mechanisms, report back effective prescribing decisions and drug discovery opportunities to improve patient outcomes.

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Supercharging AI-drug discovery with strategic multiomic biobanks 

Drug Discovery World

Narain , PhD, President and CEO of BPGbio asks how a better calibre of biobanks in life sciences can lead to more drugs making it to market. We eagerly await future data read-outs from trials because after all, it is the drug that patients are waiting for, not our fancy algorithms.

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Risk adjusted net present value: What is the current valuation of Pharma Mar’s Ecubectedin?

Pharmaceutical Technology

According to Globaldata, it is involved in 5 clinical trials, which are ongoing. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). The drug candidate is a marine-derived compound.

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Congenital fibrinogen deficiency market forecast to reach $800m across 3MM by 2031

Pharmaceutical Technology

High interest has sparked across the CFD drug space as more experts are becoming aware of the clinical efficacy and large market opportunity of using HFC products to treat various AFD indications, including surgeries involving massive blood loss and unexpected trauma. While they treat the symptoms of CFD, they are not curative.

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Novel monoclonal antibody can substantially lower triglycerides in patients with acute pancreatitis

Scienmag

The investigational drug evinacumab reduced triglycerides in patients with severe hypertriglyceridemia (sHTG) and a history of hospitalizations for acute pancreatitis in a phase 2 global study led by Mount Sinai.

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Cancer, genomics and personalised medicine: Modelling the future of oncology

pharmaphorum

•What are the implications from these changes for clinical trials? How the shift towards cancer as a phenotype/genotype is being applied in clinical trials and how they are run •What are co-clinical trials and why could they hold the key to answering unmet need in cancer treatment and beyond into broader drug development?