Indications for Kymriah Approval in Europe

In early May 2022, Novartis announced that the European Commission (EC) has approved the CAR-T cell therapy Kymriah (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL). Follicular lymphoma is an incurable malignancy, and patients frequently relapse despite receiving multiple treatments.

In 2018, Kymriah (Tisagenlecleucel) was first approved in Europe for the treatment of pediatric and young adult patients up to 25 years of age with refractory B-cell acute lymphoblastic leukemia, post-transplant relapse, or second or later relapse.

On May 28th, 2022, Novartis announced that the FDA granted accelerated approval to the CAR-T therapy Kymriah (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

Kymriah Clinical Trial Data

This approval was based on the ELARA trial (NCT03568461), a multicenter, single-arm, open-label study evaluating the efficacy of the CAR-T cell therapy Kymriah in patients with relapsed/refractory follicular lymphoma.

Kymriah is a CD19-directed chimeric antigen receptor (CAR) T-cell therapy used to treat adult patients with refractory or relapsed follicular lymphoma within 6 years of initial treatment. It is administered as a single intravenous infusion following lymphodepleting chemotherapy, with a target dose of 0.6 to 6.0 x 10^8 CAR-T cells.

The primary efficacy endpoints were overall response rate (ORR) and duration of response (DOR), as determined by an independent review committee. In the primary efficacy analysis of 90 patients, the ORR was 86% (95% CI: 77, 92), with a complete response (CR) rate of 68% (95% CI: 57, 77). The median DOR was not reached, with 75% (95% CI: 63, 84) of responders having an ongoing response at 9 months. For all patients who underwent leukapheresis (n=98), the ORR was 86% (95% CI: 77, 92), and the CR rate was 67% (95% CI: 57, 76).

The most common adverse reactions (>20%) in the trial were cytokine release syndrome, infections, fatigue, musculoskeletal pain, headache, and diarrhea.

Kymriah’s Approved Indications

1. Refractory B-cell acute lymphoblastic leukemia, post-transplant relapse, or second or later relapse in pediatric and young adult patients.
2. Treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.
3. Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma after two or more lines of systemic therapy.

It is anticipated that Kymriah will gain additional approved indications in the future, further contributing to human health in the medical field.

Reference

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-tisagenlecleucel-relapsed-or-refractory-follicular-lymphoma

https://www.novartis.com/news/media-releases/novartis-kymriah-receives-ec-approval-first-car-t-cell-therapy-adults-relapsed-or-refractory-follicular-lymphoma

Content Source:医伴旅