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Pfizer’s haemophilia B gene therapy meets phase 3 primary endpoint

pharmaphorum

during the lead-in pre-treatment period of at least six months. Key secondary endpoints demonstrated a 78% reduction in treated ABR and a 92% reduction in annualised infusion rate, and SPK-9001 was generally well-tolerated, its safety profile consistent with phase 1 and phase 2 results.

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Tributes as former NICE, MHRA chair Sir Mike Rawlins dies

pharmaphorum

A pharmacologist by training, Rawlins was the founding chairman of health technology assessment (HTA) agency NICE , leading the organisation from its inception in 1999 until 2013, and hired Sir Andrew Dillon to become the organisation’s founding chief executive.

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The benefits of mass spectrometry for expediting biologics to patients

European Pharmaceutical Review

Conversely, poor analytical characterisation will likely lead to questions from regulators and, without sufficient product and process knowledge, an IND or BLA that is eventually deemed incomplete. Incorrect DNA sequences can also lead to high levels of SVs. While SVs are typically present at less than 0.1

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Pharmaceutical industry: 2023 in retrospect

European Pharmaceutical Review

4 The use of CPCA assessments allows for the assignment of scientifically justifiable higher acceptable intakes (AIs), without impacting patient safety. 16 Hydrazoic acid is a toxic, explosive gas and presents a significant worker-safety risk during manufacture of valsartan drug substance. However, there were some notable differences.

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Botanical drugs – what is the best way forward for regulatory and market approval?

European Pharmaceutical Review

The FDA’s role in the regulation of botanical drug products includes an assessment of their safety and efficiency 5 but insufficient evidence for efficacy is one of the most common reasons why new drug candidates fail to reach this step. 10,12 The THR scheme is also limited to products used for oral or external use and/or inhalation.

Marketing 103
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Collaboration with Janssen and Eli Lilly to address lack of paediatric medications

Pharmaceutical Technology

Out of the seven biologics approved to treat moderate to severe CD in adults, only the anti-TNFαs, Remicade (infliximab), and Humira (adalimumab), have been approved for paediatric patients, with Humira receiving the most recent approval in 2014.

Medical 59
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Moving towards oral delivery of biologics

European Pharmaceutical Review

Although effective, the inconvenience of regular injections can lead to poor patient adherence, with studies showing that persistence and adherence are generally better for oral medications compared with injections. A robotic pill for oral delivery of biotherapeutics: Safety, tolerability, and performance in healthy subjects.