7/102 sources · 2 publishersUpdated Nov 1, 05:27 UTC
UK regulator approves expanded use of Vertex's Casgevy for children 5 and older
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy Casgevy for patients aged five and up with severe sickle cell disease or transfusion‑dependent β‑thalassemia. The decision follows safety and efficacy data reviewed by the regulator. NHS England and NICE have agreed to fund the therapy for this younger age group.
Why it matters
The approval opens a larger patient pool in the UK, potentially boosting Casgevy sales and supporting Vertex's revenue growth, as analysts project $1.6 billion in sales by 2032.
Key facts
- 1The MHRA granted a label expansion for Casgevy to treat patients aged five and older with severe sickle cell disease and transfusion‑dependent β‑thalassemia. pharmaceutical-technology.com
- 2Vertex and CRISPR Therapeutics co‑develop Casgevy, a non‑viral gene therapy that targets the BCL11A gene to reactivate fetal haemoglobin production. pharmaceutical-technology.com
- 3NHS England and NICE have agreed to cover the therapy for the newly approved age group. thepharmaletter.com
- 4Vertex reported revenue growth of 78% quarter‑over‑quarter and 151% year‑over‑year in Q2 2026. pharmaceutical-technology.com
- 5Analysts at GlobalData forecast Casgevy sales of $1.6 billion in 2032. pharmaceutical-technology.com
Summary written by AlphAI AI Desk from 2 of 2 sources. Not investment advice. Figures are as stated by the linked sources.