Vertex Resolves To Reverse English Funding Rejection For CRISPR Sickle Cell Gene Therapy

The health technology assessment institute, NICE, is not yet ready to recommend Casgevy for sickle cell disease and says it wants more data. Meanwhile, an access agreement relating to the treatment’s use for transfusion-dependent β-thalassemia is making progress in England, as are reimbursement talks for SCD in other European countries.

3d graphic of sickled blood cell
Casgevy is a one-off gene therapy for sickle cell disease • Source: Shutterstock

Vertex is hoping to reverse NICE’s interim decision to recommend against funding its CRISPR gene editing therapy, Casgevy, for severe sickle cell disease (SCD) in England because of uncertainties over the clinical and economic evidence submitted.

The company, which developed the one-off treatment with CRISPR Therapeutics, said it was disappointed by the health technology assessment institute’s decision but told the Pink Sheet it planned to address...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from United Kingdom

UK And US Regulators In Sync On RWD External Control Arms

 
• By 

The UK regulator’s draft guideline on the use of external control arms based on real-world data reflects concepts similar to those outlined by the US Food and Drug Administration.

Why Hympavzi Secured English Funding For Hemophilia B But Not Hemophilia A

 

Pfizer told the Pink Sheet it would work with the health technology assessment institute, NICE, with the hope of making Hympavzi available to patients with hemophilia A via the National Health Service.

ClinicalTrials.gov Or ISRCTN: Getting Transparency Right For UK Trials

 
• By 

ISRCTN’s systems are being redesigned to facilitate compliance with upcoming requirements in the UK’s clinical trials legislation.

Spain Beats England To Tevimbra Reimbursement Recommendations

 

Spanish authorities have published more pricing and reimbursement reports as part of a new drive to increase transparency.

More from Europe

EU CHMP Opinions And MAA Updates

 

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.

EMA: ‘Sad To See’ Member States Against Patient Engagement Under EU Pharma Reform

 

The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.

EU To Track Decentralized Clinical Trials In CTIS To Support Innovation

 
• By 

Monitoring the use of decentralized elements in clinical trials is a priority for the European medicines regulatory network.