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Health Canada expands Orkambi label to include access to infants

Pharmaceutical Technology

This label expansion makes Orkambi the only disease-modifying CF medication available to patients of this age in Canada. This label expansion follows a similar label change in the US in September 2022. Health Canada is granting this new label expansion based on recent results from a Phase III study.

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Overcoming regulatory issues with investigational medicinal product labelling 

Pharmaceutical Technology

Issues with regulatory requirements and documentation can also cause significant delays, while any inaccuracies in translations on labelling can mean that dosage and storage information is not correctly understood or followed. And accurate labelling and translation are critical for this sector.

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FDA updates stimulant labelling requirements to reduce misuse

Pharmaceutical Technology

According to the FDA update, current labels for these drugs do not have up-to-date warnings about the risks of drug misuse and abuse and fail to mention that most individuals who misuse prescription stimulants source drugs from other family members or peers. Please check your email to download the Report.

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Dawn of a new indication: how to study drugs for aging 

Pharmaceutical Technology

Based on preclinical research for their impact on aging, well-known drugs like metformin and rapamycin are already being considered for off-label usage, due to their accepted safety profile and cost-effectiveness. Bellantuono worries that labeling aging as a disease indication could be “agist” and would have social implications.

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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The multi-centre, open-label, single-arm clinical trial has been designed to evaluate the efficacy, tolerability, and safety of a single dose of NGN-401 delivered using a one-time ICV procedure. Neurogene stated that the FDA IND clearance allows it to commence a Phase II/II trial of NGN-401 in female paediatric Rett syndrome patients.

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The “Medical Bypass”: new drugs to strike obesity

Pharmaceutical Technology

weight reduction in obese individuals, as per its label. Furthermore, in order to make up for shortages Novo Nordisk’s Ozempic, a version of semaglutide that is approved to treat type 2 diabetes, was used off-label for obese patients. Download the full report to understand what to expect and how to align your strategies for success.

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AstraZeneca ends Phase IV Andexxa trial after positive results

Pharmaceutical Technology

AstraZeneca will now proceed with regulatory filings in the US and EU to convert to full label approval, with full results from the trial to be presented at a forthcoming medical meeting. Download the full report to understand what to expect and how to align your strategies for success. Evaluate the effects of COVID-19 on the sector.