UK panel reverses decision, now recommends covering Yescarta

An independent body that offers guidance to improve health and social care in England is now backing axicabtagene ciloleucel — one of two chimeric antigen receptor T-cell therapies already approved in the United States and European Union — due to a confidential discount negotiated with the agent’s manufacturer.

The National Institute for Health and Care Excellence (NICE) initially rejected axicabtagene ciloleucel (Yescarta; Kite Pharma, Gilead) for the treatment of adults with relapsed or refractory large B-cell lymphoma.

NICE said the therapy was too expensive to justify its use in England’s National Health Service (NHS).

The list price for axicabtagene ciloleucel in the United States is $373,000.

After NHS and Gilead agreed to a confidential discount off the list price, NICE backed the use of axicabtagene ciloleucel under the NHS England’s Cancer Drugs Fund. This fund serves as an extra means for patients to access novel medicines before NICE backing, as reported in Fierce Pharma.

“Chimeric antigen receptor [CAR] T-cell therapy is one of the most promising new treatments in a generation for lymphoma and leukemia, and NHS patients will now be among the first in the world to benefit,” Simon Stevens, chief executive of NHS England, said in a press release. “The NHS has world-leading clinicians, researchers and scientists, and today’s announcement is proof-positive that we are open to constructive and flexible partnerships with industry that rapidly bring life sciences innovation to NHS patients in a way that is also fair to British taxpayers.”

As HemOnc Today previously reported, last month NICE also recommended against the use of tisagenlecleucel (Kymriah, Novartis) — the second FDA-approved CAR T-cell therapy — for the treatment of adults with relapsed or refractory large B-cell lymphoma. However, NICE announced it would recommend the use of tisagenlecleucel for children and young adults aged younger than 25 years with refractory acute lymphoblastic leukemia after reaching a deal with Novartis.

Axicabtagene ciloleucel — a CD19-directed genetically modified autologous T-cell immunotherapy — is indicated in the United States for the treatment of adults with relapsed or refractory large B-cell lymphoma who received two or more lines of systemic therapy.

The approval applies to patients with diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

The FDA based its decision in part on data from the ZUMA-1 trial, which included 101 adults with relapsed or refractory large B-cell lymphoma.

Overall, 72% of patients treated with a single infusion responded to therapy, including 51% (95% CI, 41-62) of patients who achieved complete response. At median follow-up of 7.9 months, median duration of response had not been reached (95% CI, 8.1-not estimable) among patients who achieved complete remission.

“This is an important day for patients with lymphoma who may have run out of effective treatment options leaving them just months to live,” Hilary Hutton-Squire, Gilead Sciences general manager for UK and Ireland, said in the release. “We are delighted to have been able to reach an agreement by working closely with NHS England and NICE which allows us to bring this new generation of personalized cancer treatment to patients.”

 

 

An independent body that offers guidance to improve health and social care in England is now backing axicabtagene ciloleucel — one of two chimeric antigen receptor T-cell therapies already approved in the United States and European Union — due to a confidential discount negotiated with the agent’s manufacturer.

The National Institute for Health and Care Excellence (NICE) initially rejected axicabtagene ciloleucel (Yescarta; Kite Pharma, Gilead) for the treatment of adults with relapsed or refractory large B-cell lymphoma.

NICE said the therapy was too expensive to justify its use in England’s National Health Service (NHS).

The list price for axicabtagene ciloleucel in the United States is $373,000.

After NHS and Gilead agreed to a confidential discount off the list price, NICE backed the use of axicabtagene ciloleucel under the NHS England’s Cancer Drugs Fund. This fund serves as an extra means for patients to access novel medicines before NICE backing, as reported in Fierce Pharma.

“Chimeric antigen receptor [CAR] T-cell therapy is one of the most promising new treatments in a generation for lymphoma and leukemia, and NHS patients will now be among the first in the world to benefit,” Simon Stevens, chief executive of NHS England, said in a press release. “The NHS has world-leading clinicians, researchers and scientists, and today’s announcement is proof-positive that we are open to constructive and flexible partnerships with industry that rapidly bring life sciences innovation to NHS patients in a way that is also fair to British taxpayers.”

As HemOnc Today previously reported, last month NICE also recommended against the use of tisagenlecleucel (Kymriah, Novartis) — the second FDA-approved CAR T-cell therapy — for the treatment of adults with relapsed or refractory large B-cell lymphoma. However, NICE announced it would recommend the use of tisagenlecleucel for children and young adults aged younger than 25 years with refractory acute lymphoblastic leukemia after reaching a deal with Novartis.

Axicabtagene ciloleucel — a CD19-directed genetically modified autologous T-cell immunotherapy — is indicated in the United States for the treatment of adults with relapsed or refractory large B-cell lymphoma who received two or more lines of systemic therapy.

The approval applies to patients with diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

The FDA based its decision in part on data from the ZUMA-1 trial, which included 101 adults with relapsed or refractory large B-cell lymphoma.

Overall, 72% of patients treated with a single infusion responded to therapy, including 51% (95% CI, 41-62) of patients who achieved complete response. At median follow-up of 7.9 months, median duration of response had not been reached (95% CI, 8.1-not estimable) among patients who achieved complete remission.

“This is an important day for patients with lymphoma who may have run out of effective treatment options leaving them just months to live,” Hilary Hutton-Squire, Gilead Sciences general manager for UK and Ireland, said in the release. “We are delighted to have been able to reach an agreement by working closely with NHS England and NICE which allows us to bring this new generation of personalized cancer treatment to patients.”

 

 

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