This week in drug discovery (21-25 November)

News round-up for 21-25 November by DDW Digital Content Editor Diana Spencer 

Gene therapies have made the headlines over the last few days, with treatments for rare AADC deficiency, lung cancer and haemophilia B given the go ahead from regulators. This week has also seen the first immunomodulatory treatment for type 1 diabetes approved in the US and a possible breakthrough in the treatment of neurodegenerative disorders. 

The top stories: 

Gene therapy could ‘transform prognosis’ for AADC deficiency patients 

PTC Therapeutics’ Upstaza (eladocagene exuparvovec) has received authorisation from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK. 

FDA approves first disease-modifying therapy for T1D 

The US Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for anti-CD3-directed antibody TZIELD (teplizumab-mzwv), the first immunomodulatory treatment for type 1 diabetes (T1D).   

NICE recommendation a step-change for rare and aggressive type of lung cancer 

The National Institute for Health and Care Excellence (NICE) has published a positive recommendation for Takeda’s EXKIVITY (mobocertinib) for treating EGFR Exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy within a Final Appraisal Document (FAD). 

Hope for drug that slows neurodegenerative disease 

Scientists have found a new drug treatment that can slow the progression of neurodegenerative disease in mice. The breakthrough research may offer fresh hope in tackling currently untreatable conditions such as Alzheimer’s disease (AD). 

FDA approves first gene therapy for haemophilia B 

The US Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B.

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