New drug 'Niktimvo' for cGVHD receives ODD in Korea

Gained attention as a new treatment option for patients who progressed after prior treatments

The AGAVE-201 clinical trial showed Niktimvo's ORR improvement

'Niktimvo,' a new drug for the treatment of chronic graft-versus-host disease (cGVHD), has been designated as an orphan drug in South Korea.

 

The Ministry of Food and Drug Safety (MFDS) announced this on the notifications of Orphan Drug Designation (ODD).

 

Niktimvo (axatilimab)'s basis for ODD indication is for the treatment of 'adults and pediatric patients over 40 kg with chronic graft-versus-host disease (cGVHD) after failure of two or more systemic therapy.' In August last year, Niktimvo secured the U.S.

 

Food and Drug Administration (FDA) approval It is a cGVHD treatment jointly developed by Incyte Corporation and Syndax Pharmaceuticals, a company in Massachusetts specializing in developing anticancer agents, through the exclusive agreement for global joint-development‧launching.

 

GvHD is a serious complication that often occurs after allogeneic hematopoietic stem cell transplant (allo-SCT).

 

Donor T cells from the transplanted graft recognize the recipient’s normal cells as foreign and attack them, affecting multiple organs, including the skin, gastrointestinal tract, liver, and lungs.

 

Since symptoms can manifest throughout the body, GvHD causes additional challenges for patients who have survived allo-SCT, significantly impacting their quality of life.

 

Corticosteroids are used as the first-line treatment; however, approximately 50% of patients fail to respond.

 

In such cases, with no established standard therapy available, there has been an unmet need for effective treatment options.

 

Regarding this, Niktimvo has emerged as a promising new therapeutic option with a novel mechanism of action to address the severe complications in chronic GVHD patients after previous treatments.

 

In South Korea, Novartis Korea's 'Jakavi (ruxolitinib)' was listed on the reimbursement list in November 2023.

 

Sanfi Korea's 'Rezurock (belumosudi)' is in the reimbursement process.

 

The efficacy of Niktimvo was demonstrated through the AGAVE-201 study, which enrolled 241 children and adult patients with refractory and chronic GvHD who received two prior systemic therapies.

 

Clinical results showed that the primary endpoint was met in every cohort treated with Niktimvo, with sustained responses observed across all organ systems and patient subgroups.

 

Among patients receiving the approved dose of 0.3 mg/kg every two weeks, 75% achieved an objective response (ORR) within the first six months of treatment, with a median time to response of 1.5 months.

 

Additionally, 60% of patients maintained their response after 12months of treatment.