MFDS grants GIFT to 'rusfertide,' no records of any global approvals

Granted a fast-track review under the GIFT designation due to the status of 'No existing treatment'

Novel mechanism that mimics hepcidin…expected to be an innovative therapeutic agent that would relieve 'phlebotomy burden'

A new drug that would be a novel treatment option for patients with polycythemia vera (PV), a type of rare hematologic malignancy, has entered the fast-track review of the regulatory agency in South Korea. Notably, as this drug has not yet received final approval even in major global markets such as the United States and Europe, the medical community is closely watching its domestic approval and launch timeline.

The Ministry of Food and Drug Safety (MFDS) announced that it has designated 'rusfertide' a polycythemia vera treatment under development by Takeda Pharmaceuticals Korea, as the 71st product under the Global Innovative Products Fast-Track (GIFT) program. The designation date was June 10.

The MFDS specified that rusfertide's GIFT designation falls under the category of "no existing treatment." This decision is an outcome of a high evaluation of the drug's potential to improve efficacy and safety in a rare disease area characterized by significant unmet medical needs or where conventional therapies fail to provide adequate clinical outcomes.

'​Polycythemia vera (PV)', the target indication for rusfertide, is a chronic myeloproliferative neoplasm (a rare blood cancer) in which red blood cells proliferate uncontrollably in the bone marrow. An excess of red blood cells increases blood viscosity abnormally, exponentially elevating the risk of thrombosis (blood clots) that can lead to fatal complications such as stroke, myocardial infarction, and pulmonary embolism. Furthermore, it is a debilitating disease that severely compromises patients' quality of life through symptoms like chronic fatigue, generalized pruritus, and enlarged spleen.

Previously, patients had to rely on therapeutic phlebotomy, requiring periodic hospital visits to draw large volumes of blood to reduce elevated red blood cell counts. However, this approach has been a significant burden, causing physical and psychological distress to patients.

Rusfertide is a novel therapeutic developed to address the limitations of these conventional treatments fundamentally. It mimics hepcidin, an endogenous hormone that regulates iron metabolism in the human body. Inhibiting ferroportin, a protein that exports iron from cells into the bloodstream, blocks the iron supply required for bone marrow to produce red blood cells, thereby preventing the overproduction of erythrocytes. According to clinical studies, the drug reduces the frequency of phlebotomy sessions, significantly improving patients' quality of life.

Notably, rusfertide is still an 'early-stage novel drug' that has not yet secured final regulatory approval from any major global regulatory agency.

According to the expedited review report, rusfertide's innovativeness was recognized early on in the global market, receiving Fast Track (FT) and Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA) and being admitted into the European Medicines Agency's (EMA) PRIME (PRIority MEdicines) program. It has also been granted Orphan Drug Designation (ODD) across South Korea, the US, and Europe. However, its official approval date remains listed as 'Not Applicable' across all global regulatory agencies, including the US, Europe, and Japan, meaning it is currently an unapproved agent undergoing regulatory review.

An industry insider said, "It is both unusual and encouraging that an innovative novel drug, which has not yet achieved final approval even from leading regulatory bodies like the US FDA, has been designated under the Korean GIFT program. Thanks to the MFDS's Fast Track review support, domestic patients are expected to access this innovative therapeutic benefit relatively quickly compared to its global launch timeline."

Meanwhile, based on the GIFT designation, the MFDS plans to promptly initiate its review upon submission of the marketing authorization application for rusfertide. However, the MFDS added, "The exact indications, efficacy, and safety profile will be finalized following a review during the main regulatory approval procedure."