NICE, the health technology assessment (HTA) body for England, has formally agreed to the reimbursement of the world’s first CRISPR/Cas9-based gene therapy, Casgevy (exagamglogene autotemcel), also known as exa-cel, for patients with transfusion-dependent beta thalassemia (TDT).
NICE said on 8 August that Casgevy should be made available through its Innovative Medicines Fund (IMF) as an option for treating TDT in people 12 years and over when a hematopoietic stem cell transplant is suitable but no donor is available.
The IMF deal is the first national reimbursement agreement for Casgevy in Europe, a spokesperson for Vertex told the Pink Sheet, although the company has secured other types of early access arrangements in France, Austria and Italy (more below).
Casgevy is one of only two drugs to be reimbursed under a managed access scheme within the IMF since its inception. Hemgenix (etranacogene dezaparvovec), CSL Behring’s gene therapy for hemophilia B, was reimbursed under the IMF last month.(Also see "Hemophilia Gene Therapy Hemgenix Gets Funding In England, With More Talks Underway In Europe" - Pink Sheet, 1 July, 2024.).
The IMF enables more data to be collected on the clinical and cost effectiveness of innovative medicines to support a future routine reimbursement agreement. NICE said that Casgevy will be available immediately for up to 460 people with TDT who are eligible for the treatment.
The IMF has also been used previously to enable interim faster access to medicines including Alexion’s Wolman disease drug Kanuma (sebelipase alfa), although these drugs did not enter the IMF managed access program. Instead, money from the IMF was used to accelerate access to products that were backed for routine NHS commissioning (Also see "England’s Innovative Medicines Fund Picks Up Pace As Alexion’s Kanuma Gets Green Light" - Pink Sheet, 13 December, 2023.).
At its list price, Casgevy costs £1.65m ($2.1m) per course of treatment, although Vertex has agreed to provide the gene therapy to the NHS at a confidential discount.
Although Wales and Northern Ireland typically follow the guidance produced by NICE, the IMF funding applies only to patients who live in England. However, Vertex is “in discussions with NHS Wales and NHS Northern Ireland about access to Casgevy for patients in those nations,” the company’s spokesperson said.
Vertex has also submitted an application for reimbursement in Scotland, through the country’s national HTA body, the Scottish Medicines Consortium. A date for the SMC’s decision on access to Casgevy has yet to be determined, according to its website.
Access In France, Italy & Austria
Casgevy, which was developed by Vertex and CRISPR Therapeutics, has a conditional marketing authorization in Great Britain from the regulator, the MHRA, for patients over 12 years old with either TDT or sickle cell disease (SCD). The MHRA was the first global regulatory body to approve Casgevy, in November 2023, and it was approved in the US the following month.(Also see "UK Authorizes Vertex’s Casgevy, The World’s First CRISPR/Cas9 Gene Therapy" - Pink Sheet, 17 November, 2023.).
The European Commission, meanwhile, authorized Casgevy for TDT and SCD on a conditional basis in February this year.(Also see "EMA Says Yes To GSK’s Omjjara, UCB’s Rystiggo and Novartis’ Spexotras" - Pink Sheet, 10 November, 2023.).
“In France, we have secured an early access program allowing TDT and SCD patients to access Casgevy,” the Vertex spokesperson said, adding that Italy’s national regulator, AIFA, had also approved requests for early access to Casgevy for both TDT and SCD patients.
In Austria, an agreement has been secured with an authorized treatment center that will allow patients to access the gene editing medicine.
Helen Knight, director of medicines evaluation at NICE, said that although there were some uncertainties in the evidence for the long-term benefits of Casgevy, the NICE committee felt that the therapy “could represent a potential cure for some people with TDT, freeing them from the burden and risks of needing regular blood transfusions.”
The final guidance “also addresses [NICE’s] aim of reducing health inequalities associated with conditions like TDT,” she added, stating that this demonstrated the organization’s ability to address these issues proportionately and flexibly as part of its standard process.
SCD Talks Continue
Although Casgevy has secured approval in England for both SCD and TDT, the NICE IMF decision applies only to the drug’s TDT indication.
In March, NICE provisionally rejected Casgevy for reimbursement in the SCD population as it said more evidence was needed to address issues with the economic modeling. Vertex said that it was continuing to “work collaboratively” with NICE and NHS England to ensure eligible patients with SCD could access Casgevy “as soon as possible.” (Also see "Vertex Resolves To Reverse English Funding Rejection For CRISPR Sickle Cell Gene Therapy" - Pink Sheet, 15 March, 2024.).
“In the EU, Vertex is working closely with reimbursement authorities to bring this innovative therapy to eligible SCD and TDT patients rapidly as the company has done in the US, the Kingdom of Saudi Arabia and Bahrain,” the company added.
