Merck’s mirdametinib drug receives GIFT designation

MFDS to provide expedited review support… FDA approval granted in February last year

Competition expected with the existing NF treatment, Koselugo

Gomekli (mirdametinib) was approved by the US FDA in February last year

Merck’s neurofibromatosis treatment candidate mirdametinib has been designated for the Ministry of Food and Drug Safety’s (MFDS) expedited review support program, GIFT (Global Innovative products on Fast Track).

As a result, the drug’s entry into the Korean market is expected to accelerate.

According to the MFDS, mirdametinib was designated as a GIFT product on the 20th of last month.

GIFT is an expedited review program launched in September 2022 to support rapid product development and provide patients with faster access to new treatment options.

The program targets innovative medicines, including those for life-threatening diseases, rare diseases with no existing treatment alternatives, and new drugs developed by certified innovative pharmaceutical companies.

The MFDS designates GIFT products based on a comprehensive evaluation of factors such as innovative therapeutic effects, contribution to addressing public health needs, and the developer’s efforts.

Once designated, the review period is reduced by at least 25% (from 120 working days to 90 working days).

The program also applies rolling reviews, under which submitted data are reviewed on an ongoing basis, and provides close communication between regulators and developers through product briefings and supplementary meetings. In addition, regulatory consulting and other support measures are offered to facilitate rapid commercialization.

Mirdametinib is Korea’s 64th GIFT product. The proposed indication submitted to the MFDS is for the treatment of ‘pediatric and adult patients aged 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.’

Mirdametinib is a selective inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). It exerts antitumor activity by blocking MEK activity, thereby inhibiting the phosphorylation of Extracellular-regulated kinase 1 and 2 (ERK1 and ERK2) within the Mitogen-activated protein (MAP) pathway.

It received Fast Track designation from the US FDA on February 11 last year and was approved by the EMA on July 17 last year. In Korea, it was designated as an orphan drug on December 11 last year.

Mirdametinib was originally developed by the U.S. biotech company SpringWorks Therapeutics. In April 2023, SpringWorks was acquired by Germany-based Merck for USD 3.9 billion, giving Merck global commercial rights to the drug.

Once launched, mirdametinib is expected to compete with the existing neurofibromatosis therapy Koselugo (selumetinib, AstraZeneca).

Koselugo, which is also indicated for neurofibromatosis type 1, has been reimbursed by Korea’s national health insurance for patients aged 3 years and older since January 2024.

Neurofibromatosis is a type of neurocutaneous syndrome characterized by abnormalities affecting both the skin and the central nervous system. Among its various subtypes, neurofibromatosis type 1 is the most common. Depending on the location of the neurofibromas, patients may exhibit brain tumor symptoms, and spinal involvement can lead to scoliosis. As of 2024, there are 6,490 known patients in South Korea.

With recent increases in cases of reduced reimbursement for Koselugo, calls for improving reimbursement criteria are growing among neurofibromatosis patients and medical professionals.