On the 2nd (local time), U.S.-based Biosplice Therapeutics announced that the European Medicines Agency (EMA) had initiated the centralized review procedure for Loresibivint on the 30th of last month. This means the EMA determined that the data submitted for Loresibivint was sufficient to proceed with a formal review, including evaluations of efficacy and safety.
Currently, marketing authorization reviews for Loersibivint are underway simultaneously in several countries. Biosplice submitted a marketing authorization application for Loersibivint to the UK Medicines and Healthcare products Regulatory Agency (MHRA) last August. Prior to that, in January of this year, it submitted a New Drug Application (NDA) to the FDA. SamilPharmaceutical signed a technology licensing agreement with Biosplice in 2021, securing the rights to develop, obtain approval for, and exclusively commercialize Loresibivint in South Korea.
Outline for UK in First Half of Next Year, EU in Second HalfErich Horsley, CEO of BioSplice, stated, “The UK validated the marketing authorization application for Loresibivint on August 28, and the European Union (EU) did so on September 30, and both have begun the review process,” adding, “Accordingly, Loresibivint is currently undergoing regulatory review in key markets.”
The results of the approval review are expected to become clear next year. The UK’s MHRA applies an actual review period of 150 days for innovative drugs, or up to 210 days if additional review is required. Considering that the review of Roersibivint began on August 28, a decision on approval is expected as early as the first half of next year.
The process in Europe is expected to take longer than in the UK. While the EMA’s centralized review process allows for an actual review period of up to 210 days, the review clock pauses during the period for submitting supplementary data. In addition, after the Committee for Medicinal Products for Human Use (CHMP) under the European Medicines Agency (EMA) issues a marketing authorization recommendation, the application must undergo the final approval process by the European Commission (EC). According to the EMA, medicines that undergo the centralized review process take an average of 421 days from the start of review to final approval. Applying this to Loresibivint, it is highly likely that a decision on European approval will be made sometime between the second half of next year and the end of the year.
The new osteoporosis drug Eladinose began EMA review in November 2021, received a positive opinion from the CHMP in October 2022, and obtained final approval in December of the same year. It took approximately one year from the formal start of the review to final approval.
Loresibivint “Closest to Becoming the First DMOAD”Loresibivint is
the closest to becoming theworld’s
firstdisease-modifying osteoarthritis drug (DMOAD).
Unlike existing osteoarthritis treatments that merely reduce pain or inflammation, a DMOAD refers to a treatment that slows structural damage to the joints or inhibits the progression of the disease itself. However, despite the development of numerous candidate compounds to date, no DMOAD has yet been officially approved in the U.S. or Europe, as it has been difficult to simultaneously demonstrate improvements in pain, function, and structural integrity.
A leading competitor is “Sprifermin” from Merck KGaA in Germany. Developed to induce cartilage regeneration by utilizing fibroblast growth factor 18 (FGF18), it demonstrated the potential for structural improvements—such as increased cartilage thickness—in Phase 2 clinical trials. However, it failed to demonstrate a clear difference in pain relief and functional improvement as actually experienced by patients, preventing it from reaching commercialization.
Other candidates have faced similar obstacles. “GLPG1972,” jointly developed by Belgium’s Galapagos and France’s Servier, garnered high expectations for its mechanism of action—inhibiting ADAMTS-5, which is involved in cartilage degradation—but failed to demonstrate a significant efficacy signal in the global Phase 2b ROCCELLA trial.
“UBX0101,” developed by Unity Biotechnology in the U.S., also garnered attention as a senolytic therapy targeting the root cause of osteoarthritis by removing senescent cells; however, subsequent key clinical development was halted after it failed to meet the primary endpoint for pain improvement in a Phase 2 clinical trial.
Uncertainties have also arisen among competing candidates in late-stage development.
Kolon TissueGene, Inc.(950160) The cell-gene therapy “TG-C” failed to demonstrate statistical significance in its primary endpoint compared to a placebo in its first U.S. Phase 3 clinical trial, which was announced this year, increasing uncertainty in the race to become the world’s first DMOAD.
In contrast, RoerCivibint not only improved pain and function but also widened the medial joint space width (mJSW) in the knee, thereby meeting the requirements for a curative treatment. A narrowing joint space is a key structural indicator of cartilage loss and the progression of osteoarthritis. While many competing therapies have repeatedly failed in clinical trials, RoerCivibint has pulled ahead by pursuing marketing authorization in the U.S., the U.K., and Europe.
CEO Hosley emphasized, “Approximately 50 million adults in the U.S. alone suffer from osteoarthritis, yet there is still no curative treatment,” adding, “Loresibivint is a life-changing treatment for osteoarthritis patients in the U.S., the U.K., and Europe,” thereby expressing confidence in the commercialization of the world’s first DMOAD.
An industry insider noted, “Throughout the history of DMOAD development, there have been recurring issues, such as cartilage improving but pain persisting, or pain signals remaining despite a lack of structural improvement,” and added, “There is a gap of at least several years between Lore Sibivint and other treatments.”
Loresibivint: Even by conservative estimates, Korean sales are projected to reach 400 billion won
As global approval procedures progress, SamilPharmaceutical’s domestic rights to Loresibivint are also drawing attention.
If approval is granted in the U.S. and Europe, the pace of domestic commercialization is likely to accelerate. While new drugs approved overseas are not automatically approved in Korea, if the Ministry of Food and Drug Safety (MFDS) recognizes the applicability of overseas clinical data to the domestic market, there is no need to conduct a large-scale Phase 3 clinical trial specifically targeting Koreans. If it can be demonstrated that there are no significant ethnic differences based on data from Koreans and Asians included in overseas clinical trials or on pharmacokinetic characteristics, the drug may also be exempted from separate bridging studies.
In fact, Eisai and Biogen’s Alzheimer’s treatment “Leqembi” received approval from the Ministry of Food and Drug Safety in 2024 based on data from Korean and Asian participants included in the global Phase 3 clinical trial, without conducting a separate domestic bridging trial. Novartis’ rare disease treatments “Kymriah” and “Zolgensma,” as well as Amgen’s “Kyprolis,” were also introduced in Korea by utilizing overseas clinical data through bridge study exemptions.
Loresibivint, a local injection administered directly into the joint cavity, also exhibited low systemic exposure in early clinical trials, with blood drug concentrations falling below the limit of detection. If the FDA and EMA grant approval based on global clinical data in the future, concerns regarding pharmacokinetic differences between Koreans and non-Koreans may also be relatively low.
SamilPharmaceutical stated, “Last July, we classified Loresibivint as a new drug for osteoarthritis in our R&D pipeline,” adding, “We are aiming for a launch next year.”
Meanwhile, the number of patients with knee osteoarthritis in South Korea is estimated at approximately 3.07 million. Industry estimates suggest that among them, approximately 2 to 2.7 million patients with KL grades 2–3 could be the primary target population for Loresibivint.
Based on the annual treatment cost of 3 million won currently under consideration by SamilPharmaceutical, the theoretical potential market size amounts to approximately 6 trillion to 8 trillion won. Even assuming an actual market penetration rate of just 3%, the annual sales potential is approximately 180 billion to 240 billion won; if the penetration rate expands to 5%, it could grow to 300 billion to 400 billion won. SamilPharmaceutical recorded sales of 210.2 billion won last year.
