Credit: Vchal / Shutterstock

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted conditional marketing authorisation to Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), marking the world’s first approval of a CRISPR-based gene editing therapy.

Casgevy has been approved to treat patients with sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent beta-thalassemia aged 12 years or older, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.

Also known as exa-cel, Casgevy is currently being evaluated by the US Food and Drug Administration (FDA). The Prescription Drug User Fee Act (PDUFA) target action date for the drug is set for 8 December in severe sickle cell disease and 30 March 2024 in beta-thalassemia, as per a 16 November press release.

Casgevy is a genetically modified autologous CD34+ cell population that contains clustered regularly interspaced short palindromic repeats (CRISPR) edited human haematopoietic stem and progenitor cells.

The US Food and Drug Administration (FDA) has started an investigation to review the safety of chimeric antigen receptor (CAR)-T cell immunotherapies following reports of T cell malignancies in patients who received these immunotherapies.

The therapies under investigation include six approved B cell maturation antigen (BCMA)- or CD19-directed CAR-T cell therapies. The investigation follows reports collected from clinical trials and post-marketing adverse event surveillance.

The FDA had required companies to conduct 15-year long term follow-up observational safety studies to assess the long-term safety and the risk of secondary malignancies as part of the respective therapy’s approval. 

The risk of post-therapy malignancies applies to all FDA-approved BCMA- or CD19-directed CAR-T cell therapies, as per a 28 November press release. The agency added that although the therapy benefits “continue to outweigh their potential risks for their approved uses”, the FDA is evaluating the need for regulatory action regarding the risk of T-cell malignancies.

Bayer’s stock price has plummeted to its lowest since 2005 after a clinical trial investigating the anti-coagulant drug asundexian was stopped early due to lack of efficacy.

The Phase III OCEANIC-AF trial (NCT05643573) aimed to determine asundexian’s use as a treatment for people living with atrial fibrillation in a bid to prevent strokes or systemic embolisms.

The Independent Data Monitoring Committee (IDMC) found that asundexian was inferior in terms of efficacy when compared to the control arm of the trial. The OCEAN-AF trial was a part of the overall OCEAN Phase III programme. However, the committee recommended continuing another Phase III OCEANIC-STROKE trial (NCT05686070) examining the use of the drug in stroke patients. 

Following the announcement on 19 November 2023, the German pharmaceutical firm’s stock value dropped from €41.45 ($45.38) per share down to €30.77 ($33.68), as of 28 November. It is the lowest price in 18 years.

Novo Nordisk announced plans to expand one of its production facilities in Chartres, France, as it scales up its serious chronic disease product portfolio.  

Under the Dkr16bn ($2.29bn) investment, Novo Nordisk is increasing the capacity of the manufacturing site by extending the current quality control laboratory, adding aseptic production, and finishing production processes. The company predicts that this will bring over 500 new jobs once construction is completed. The investment includes additional capacity for GLP-1 products such as Novo’s Ozempic (semaglutide).   

Novo Nordisk first launched the GLP-1 therapy semaglutide, in the US in December 2017, under the brand name Ozempic. Novo Nordisk later launched the therapy under the name Wegovy for the treatment of weight loss, together with diet and physical activity.

Bayer has announced a voluntary recall of a single lot of its cancer therapy Vitrakvi (larotrectinib) oral solution 20mg/mL, available in 100mL glass bottles.

The consumer-level recall comes after the company detected cases of microbial contamination with Penicillium brevicompactum during routine stability inspections.

Vitrakvi treats NTRK gene fusion-positive solid tumours. Patients receiving the drug may therefore already be immunocompromised.  

Penicillium brevicompactum has not been found to impact human pathology in the literature, but cases of invasive disease have been reported to be caused by such Penicillium species, especially in those with underlying immunosuppression.