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NICE Does Not Recommend Sickle Cell CRISPR Drug Casgevy in Draft Guidance, Calls for More Data

NEW YORK – The UK National Institute for Health and Care Excellence on Thursday said it wants to see additional data before it makes a final recommendation on Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel).

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) in November became the first regulator to approve a CRISPR-based treatment after it granted Vertex conditional marketing authorization for the gene-editing therapy in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

However, in a draft guidance published Thursday on Casgevy's indication in sickle cell, NICE opted not to recommend the gene-editing therapy. NICE must recommend treatments before they are offered through the National Health Service.

NICE in a statement said that before recommending the treatment, it would like to see more data collected on Casgevy's effectiveness and the duration of treatment benefit, as well as data on quality of life from currently available treatments. NICE said Vertex should provide an updated managed access proposal to the agency incorporating plans for such data collection.

Under the managed access program, patients are able to access promising treatments that are not yet recommended due to uncertain clinical or cost considerations. "This would allow people to have treatment with exa-cel while more evidence is collected," Helen Knight, director of medicines evaluation at NICE, said in a statement.

Vertex, which leads global development, manufacturing, and commercialization of Casgevy, said in an email that it is "disappointed" with the draft NICE guidance. The company added it will be responding to NICE and taking part in a second committee meeting "to ensure the full severity and patient experience of the disease, and therefore the value that Casgevy brings to patients, is understood."   "We are committed to working collaboratively with NICE and the NHS to ensure long-term, sustainable access to Casgevy for the estimated 2,000 eligible SCD and TDT patients in the UK," it said.

Casgevy has been approved in the US and received conditional marketing authorization in the EU. The one-time and potentially curative autologous treatment incorporates ex vivo editing of the BCL11A gene in patients' CD34-positive hematopoietic stem and progenitor cells with CRISPR-Cas9, so that the edited genes increase production of fetal hemoglobin to facilitate oxygen delivery and offset a deficiency of adult hemoglobin that causes sickle cell and transfusion-dependent beta thalassemia.

NICE acknowledged that there are few treatments available to address symptoms of sickle cell, and treatments that are available can have significant side effects. If recommended, Casgevy would be available to patients for whom a stem cell transplant may be a suitable treatment option, but for whom no matched donor is identified.

"The appraisal committee considered that exa-cel could represent a potential cure for some people with SCD, freeing people from the burden of [vaso-occlusive crises] as well as addressing NICE's aim of reducing health inequalities associated with the condition," Knight said. "As such, and acknowledging the limitations with the evidence, NICE is willing to accept higher than the usual maximum for assessing cost-effectiveness."

In the US, Vertex has priced Casgevy at $2.2 million — slightly exceeding the $1.35 million to $2.05 million range the Institute for Clinical and Economic Review, a nonprofit that conducts independent value assessments of healthcare interventions, had suggested would be cost-effective.