MFDS reviews emergency import of 'anakinra'

A treatment for adverse reaction of CAR-T

Anakinra had been withdrawn from the Korean market…The Korean Society of Hematology says, "There are no alternative medications"

Product photo of Kineret (anakinra)

The Ministry of Food and Drug Safety (MFDS) is reportedly conducting a review of whether to urgently import anakinra (brand name Kineret) for the treatment of severe adverse reactions associated with CAR-T cell therapy.

 

On June 2nd, in response to an official inquiry from specialized journalists, MFDS stated, "MFDS is currently reviewing the necessity of emergency import based on the safety, efficacy, and overseas usage status of this product and its indication." The current MFDS review is a follow-up action to the emergency import request for anakinra submitted by the Korean Society of Hematology.

 

The Pharmaceutical Safety Bureau, which received the request, transferred the item to the MFDS Biopharmaceuticals Policy Division for an official review process.

 

CAR-T cell therapy is an advanced, precision treatment that genetically modifies a patient's immune cells to target and attack cancer cells.

 

It has garnered significant attention as a treatment offering different effects compared to conventional anti-cancer drugs, particularly for relapsed and refractory acute lymphoblastic leukemia, diffuse large B-cell lymphoma, and multiple myeloma.

 

In South Korea, the treatment costs KRW 400-500 million.

 

With national health insurance reimbursement applied, the patient's out-of-pocket burden has significantly decreased.

 

Yet, while this high-cost therapy is supported with reimbursement, some adjuvant medications used to manage severe immune adverse reactions that can occur after treatment remain unreimbursed.

 

'Tocilizumab' or 'high-dose steroids' are primarily used as first-line agents.

 

However, for patients who are refractory to these, anakinra, an Interleukin-1 (IL-1) inhibitor, serves as a crucial therapeutic alternative.

 

However, anakinra's general use in Korea has been blocked since its withdrawal from the domestic market based on the manufacturer's decision.

 

Anakinra is an interleukin-1 receptor antagonist manufactured by Sobi of Sweden.

 

It's approved in countries such as the United States (FDA), Europe (EMA), and Japan (PMDA) for treating conditions including rheumatoid arthritis, CAPS (Cryopyrin-Associated Periodic Syndromes), FMF (Familial Mediterranean Fever), and Still's Disease.

 

The 2025 European CAR T Handbook by the GoCART Coalition recommends high-dose anakinra administration for Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), and its guidelines include co-administration with corticosteroids for patients with IEC-HS (Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome).

 

Specifically, the guidelines suggest anakinra as an essential treatment option for high-risk patients who do not respond to steroid and tocilizumab therapies.

 

Furthermore, a study by Gazeau N et al.

 

(Transplant Cell Ther.

 

2023) showed that among 43 patients who developed Grade 2 or higher complications after CAR-T therapy and were administered anakinra, the treatment-related mortality rate within 28 days was 0%, and the overall response rate was 77%.

 

Meanwhile, in South Korea, anakinra is currently supplied only in a limited manner through the Korea Orphan & Essential Drug Center (KOEDC) as an urgently imported drug.

 

Yet, it is not permitted for treating adverse reactions of CAR-T.

 

Consequently, some hospitals are attempting to offer limited prescriptions through their own Institutional Review Board (IRB) approvals.

 

However, the treatment is neither reimbursed nor guaranteed legal protection, making nationwide application impossible.

 

The Korean Society of Hematology stated, "Currently, medical professionals are forced to off-label use anakinra, even risking illegal practice, to save patients' lives," and added, "This does not align with the fundamental principles of national public health systems and the right to life, and urgent institutional reform is desperately needed." The Korean Society of Hematology, which has submitted requests to the Ministry of Health and Welfare (MOHW) and the HIRA for several years following the reimbursement of CAR-T cell therapy, submitted a request via the 'Anti-Corruption & Civil Rights Commission (e-People)' portal in April regarding the allowance of off-label use of anakinra and institutional improvement.

 

In response, MFDS replied that "upon request from a central administrative agency or professional organization, we can review the expansion of the reimbursement scope based on the necessity for patient treatment, availability of alternatives, and overseas approval status." In response, the Korean Society of Hematology formally submitted an emergency import request for anakinra to the MFDS Pharmaceutical Safety Bureau on May 19.

 

The request included information such as product details, approval status in major overseas countries, usage cases in the U.S.

 

and Europe, the necessity of its use in life-threatening situations, domestic demand prediction (less than 100 cases annually), and the lack of drug alternatives.

 

The Korean Society of Hematology stated, "Rapid immune modulation is essential in life-threatening severe complications, and currently, there are no drugs with a similar mechanism to anakinra available in South Korea." They emphasized, "This emergency import request is an essential measure to protect patient lives and ensure access to treatment, and prompt institutional reform is required."