European Medicines Agency issue positive opinion on CAR-T Therapy

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has today issued a positive opinion on the use of two new CAR-T therapies in Europe.

Video: Zack Pemberton-Whiteley, campaigns and advocacy director for the charity discusses CAR-T and European approval

What is the EMA?

The European Medicines Agency (EMA) are responsible for regulating the safe use of medicines across Europe. As part of this, they assess new medicines, before issuing marketing authorisations (licenses) allowing them to be used in Europe.

The CHMP is a scientific committee of the EMA, their recommendation is a key step in the approval of any medicines. Their recommendations are based on a scientific evaluation of data to determine whether the medicine meets the necessary quality, safety and efficacy requirements and that it has a positive risk-benefit balance.

After a CHMP has been approved, the decision is reviewed by the European Commission (EC). Full licencing from the EMA is expected approximately 60 days later.

What is CAR-T?

Chimeric antigen receptor t-cell therapy, better known as CAR-T therapy, is an emerging immunotherapy that is showing promise in the treatment of blood cancers.

CAR-T is a specific type of immunotherapy that involves collecting and using the patient’s own immune cells to target the cancer. Patient’s T-cells are collected from their blood. T-cells are types of white blood cells that initiate an immune response to specific antigens. Antigens are markers found on the surface of cells that are damaged or foreign (e.g. tumour cells or viral cells).

Genome editing tools are used to insert the gene of a tumour specific CAR into the t-cell DNA. Chimeric Antigen Receptors (CARs) contain an antigen receptor – that identifies and binds to a specific antigen – and t-cell signal activating machinery. This ensures that once the receptor binds to the antigen, signals are sent to initiate the t-cell immune response.

These engineered t-cells are then infused back into the blood of the patient by intravenous infusion. The engineered CAR T-cells should be able to identify the antigens on cancer cells and trigger an immune response against them.

What has it been approved for?

Kymriah® (Tisagenlecleucel-T) has been indicated for the treatment of:

  • Patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse
  • Adult patients with diffuse large B-cell lymphoma (DLBCL) that is relapsed or refractory (r/r) after two or more lines of systemic therapy

Additionally, Yescarta® (axicabtgene ciloleucel) is used for treating:

  • Adult patients with relapsed or refractory diffuse large B‑cell lymphoma (DLBCL) and primary mediastinal large B‑cell lymphoma (PMBCL), after two or more lines of systemic therapy.

If these recommendations are upheld in the final EMA decisions, these will be the first CAR-T therapies to be licensed for use in Europe. Although both have already been licensed by the FDA (Food and Drug Administration) for use in the United States.

When will it be available in the UK?

CAR-T is expected to be approved by the Europeans Medicines Agency (EMA) for use in approximately 60 days.

However, before it will be available on the NHS, it must be assessed by the National Institute for Health and Care Excellence (NICE) to determine if it is a cost-effective use of NHS resources.

NICE appraisals of Kymriah® (Tisagenlecleucel-T) and Yescarta® (axicabtgene ciloleucel) are already ongoing, with decisions not expected until the end of 2018.

Response from Leukaemia Care:

Whilst the potential of CAR-T therapy is promising, it is important to avoid overhyping it at this stage. It must be remembered that CAR-T is not without risks and can have significant side effects. However, we strongly welcome this CHMP decision and are working closely with NICE to ensure prompt access for patients.

Zack Pemberton-Whiteley, Campaigns and Advocacy Director at Leukaemia Care said:

“This CHMP decision brings an innovative option closer to European patients who have exhausted most alternative treatments. It is important to note that CAR-T therapy may only be suitable for a limited group of patients. And, working with healthcare professionals to ensure safe and timely access for those who need it will be of paramount importance.”

You can read more about CAR-T in our ‘Know Your Rights’ toolkit:

http://leukaemiacare.org.uk/wp-content/uploads/CAR-T-Therapy-Web-Version.pdf

 

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