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- 2026-04-12 21:39:38
- Company
- MSD’s 15-valent vaccine ‘Vaxneuvance’ comes to Korea
- by Eo, Yun-Ho Mar 10, 2022 05:54am
- MSD aims to challenge the dominance of ‘Prevenar 13’ in the domestic pneumococcal vaccine market. According to industry sources, MSD Korea has submitted an application and is being reviewed for the marketing authorization of its 15-valent pneumococcal vaccine ‘Vaxneuvance,' and may be approved within the year. If released, Vaxneuvance will compete with Pfizer Korea’s 13-valent pneumococcal vaccine Prevenar 13, the leading product in the field that has an overwhelming share of the market. Vaxneuvance, which is a 15-valent pneumococcal conjugate vaccine, induces active immunization for the prevention of invasive pneumococcal disease caused by streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F. The vaccine can be used in adults 18 years of age. MSD is also undergoing clinical trials on pediatric patients. In the clinical trial, Vaxneuvance was non-inferior to Prevenar 13 (PCV13) for the 13 shared serotypes as assessed by opsonophagocytic activity (OPA) Geometric Mean Titers (GMTs). Also, its immune responses were superior to Prevenar 13 shared serotype 3 and for the two serotypes unique to Vaxneuvance, 22F and 33F. Ratio 1.62 (95% CI 1.40, 1.87)]. In particular, in the Phase III PNEU-AGE (V114-019) trial, Vaxneuvance demonstrated greater OPA GMT ratios for serotypes 22F and 33F. However, randomized controlled trials assessing the clinical efficacy of Vaxneuvance compared to Prevenar 13 have not been conducted. Vaxneuvance holds significance in demonstrating its effect in preventing serotypes 22F and 33F, the common serotypes that cause invasive pneumococcal disease (IPD) in adults. Serotypes 22F and 33F are not contained in Prevenar 13. Meanwhile, Pfizer had also developed a follow-up product for its Prevenar13. Last year, the US FDA approved Pfizer’s marketing authorization for its 20-valent pneumococcal conjugate vaccine, ‘Prevnar 20.’
- Company
- The sales of Prolia & Evenity exceed ₩100 billion
- by Mar 10, 2022 05:53am
- From the left, Amgen Prolia and EvenityAmgen has opened an era of 100 billion won in the osteoporosis treatment market. Sales of the two products are increasing rapidly as sequential treatments leading to Evenity-Prolia are being put forward. According to IQVIA, a pharmaceutical research institute, sales of Amgen's osteoporosis treatment, Denosumab, stood at 92.1 billion won last year, up 22.7% from 75.1 billion won a year earlier. Sales of another treatment, Evenity, increased 220.3% from 3.8 billion won in 2020 to 12.3 billion won last year. The total sales of the two products amounted to 104.4 billion won. Amgen introduced Prolia in Korea through GSK in 2014 and has been devoted to the osteoporosis treatment market by recovering its copyright in 2016. By actively selling Prolia in partnership with Chong Kun Dang, sales surged from 3.7 billion won in 2017 to 14.3 billion won in 2018 and 47.3 billion won in 2019. Prolia, which entered the 70 billion won range in 2020, reached 92.1 billion won last year, approaching the entry of 100 billion won blockbuster drugs. Prolia, a bone absorption inhibitor, has become the first standard treatment therapy for osteoporosis with superior effect and convenience of administration than the previously widely used bisphosphonate (BP). Bisphosphonate formulations are tricky, such as taking sufficient amounts of water and not lying down for at least 30 minutes after taking them on an empty stomach 1-2 hours before meals. In addition, it was difficult for patients to continuously take medicine because side effects such as gastrointestinal disorders could occur when used for a long time. Prolia only needs to be administered once every six months. Even after 10 years of long-term treatment, the effect of continuous bone density improvement and consistent safety profile were confirmed. As Prolia has become a standard treatment, all BP drugs are on the decline. Lilly's Forsteo, once called the strongest in the market, saw its sales fall 24.8% from 17.1 billion won in 2020 to 12.9 billion won last year. During the same period, Daewoong Pharmaceutical's Dowong Zoledronic Acid Inj also fell 12.9% from 11.2 billion won to 9.8 billion won. MSD's Fosamax products maintained sales from 12.2 billion won to 12.1 billion won. Amgen's new osteoporosis treatment Evenity approved in June 2019 is also cruising. Evenity, which raised 3.8 billion won in 2020, has been listed since last year and surpassed 10 billion won at once. Evenity is the first osteoporosis treatment that has both the effect of "promoting bone formation" and "suppressing bone absorption." It is a mechanism that stimulates mature osteoblasts and promotes bone formation by activating tissue osteoblasts, while also acts as a regulator of osteoblasts to suppress bone absorption. AACE/ACE recommend using Evenity from the initial treatment of ultra-high risk groups with a high risk of fracture. Amgen is proposing a "sequential treatment" strategy that treats patients with high risk of fracture with Prolia after one year of treatment with Evenity. By emphasizing the importance of continuous treatment of osteoporosis, it is intended to establish an environment in which patients can be treated with Prolia for a long time. Domestic societies such as the KSBMR are also carrying out various activities such as policy symposiums and revision of medical guidelines to improve awareness of osteoporosis. It is also worth noting whether Prolia and Evenity will expand their benefit range. Currently, the administration period of osteoporosis drugs is based on the bone density level of T-Score. Benefits are recognized only for patients with T-Score -2.5 or less. If the level is higher than -2.5 in follow-up observation one year after administration of the treatment, the salary is no longer recognized. Evenity can also be reimbursed when bisphosphonate is used first and then used as a secondary drug, especially T-Score -2.5 or less and two or more osteoporosis fractures must be met. In response, it is pointed out that Evenity should be used as the primary drug and improved so that it can be used even if a fracture occurs once. If the standards of Prolia and Evenity are expanded in the future, the two products are expected to face another turning point in sales.
- Company
- Why the Patent Court rejected the ‘prodrug strategy’
- by Kim, Jin-Gu Mar 10, 2022 05:53am
- Dong-A ST is challenging the substance patent of AstraZeneca’s SGLT-2 inhibitor antidiabetic treatment ‘Forxiga (dapagliflozin).’ The industry’s eyes are focused on Dong-A ST’s challenge, with the expectation that Dong-A ST’s success would bring a new strategy that can overcome the substance patent of originals. However, Dong-A ST’s attempt has been put to a stop by the Patent Court of Korea. The final decision will be made by the Supreme Court, but attention is focused on why the Patent Court of Korea overturned the first instance court's ruling and ruled in favor of AstraZeneca in its appeal against Dong-A ST. ◆ "Clear difference between the original drug and prodrug” judged in both the first and second trials According to the ruling on the 28th, the Patent Court of Korea ruled in favor of AstraZeneca on the grounds that the principle of action between the original substance of Forxiga and Dong-A ST's prodrug was the same. Pro-drug is a ‘pro’ drug that is considered a similar but different drug from the original drug. Its chemical structure is partially different in the substituents with the original drug from its manufacture to immediately before administration. However, once administered, the drug shows the same effect as the original By principle, it sounds similar to salt-modified drugs, but the difference Is clear. The salt can be changed through simple ion bonding. Its chemical structure itself does not change. However, prodrugs change the substituents through a more difficult covalent binding method. It has a different chemical structure to the original drug. The first and the second court both made the same judgment on whether the prodrug could be considered a completely new substance. Both the Intellectual Property Trial and Appeal Board (IPTAB) and the Patent Court of Korea interpreted Dong-A ST’s prodrug as a completely different substance to the original substance. The IPTAB judged that the “difference in chemical structure is clear, therefore, Dong-A ST’s prodrug is outside the scope of rights held by Forxiga’s patented invention.” ◆ Patent Court of Korea “a skill that can be easily conceived by an ordinary skilled person" However, the two courts' rulings differed in the ‘ease of substitution issue.’ The courts made different judgments on whether an ordinary skilled person in the industry may easily produce Dong-A ST’s prodrug. While the first court judged that developing the prodrug was not easy, the second instance court judged that it "was a substance anyone with ordinary skills in the industry could produce.” The evidence AstraZeneca submitted for the appeal played an important role in the ruling. The Patent Court of Korea explained, “the specific form of ‘formate ester’ that Dong-A ST used to develop its Forxiga prodrug has been found in various literature.” ◆Deleted ‘produg’ in the patent registration process …Court decides it was “common practice" at the time Dong-A ST appealed using the fact that the term ‘prodrug’ was deleted in the process of AstraZeneca’s patent registration. In fact, when AstraZeneca first filed the substance patent for Forxiga in 2006, it listed its scope as “compounds with chemical structural formula I, or other pharmaceutically acceptable salt or stereoisomer, or prodrug ester thereof.” However, the prodrug part was deleted after the first patent claim was rejected by the Korean Intellectual Property Office. The final patent registered specifies the scope as “compounds with chemical structural formula I, or other pharmaceutically acceptable salt or stereoisomer,” without the prodrug part. Dong-A ST claimed that the deletion of "prodrug esters" from the claim indicates the waiver of those rights on the company’s part. However, the Patent Court of Korea turned down the claim. The court judged “AstraZeneca did not consciously delete the part, and “it was KIPO's practice at the relevant time not to allow the term "prodrug" in patent claims for formal reasons.” ◆Up to the supreme court…’ strategy to overcome substance patents of original drugs’ at a crossroad Dong-A ST expressed dissent at the Court’s decision and filed an appeal. The case will therefore be judged by the Supreme Court. If the Supreme Court determines that there is a problem with the second trial ruling, the patent strategy of the entire domestic pharmaceutical industry may be revised. Like Dong-A ST, other companies will opt to challenge the substance patent of original drugs using the prodrug strategy. On the other hand, if the Supreme Court confirms the judgment of the second instance court, the substance patent of original drugs may continue to remain impenetrable.
- Policy
- Omicron's fatality rate is at the level of flu
- by Lee, Jeong-Hwan Mar 10, 2022 05:53am
- The quarantine authorities analyzed that if the COVID-19 vaccine is completed up to the third round, the fatality rate of Omicron mutation will be 0.07%, which is similar to that of the seasonal flu fatality rate (0.05 to 0.1%). In particular, the fatality rate for those under the age of 60 who completed the third vaccination was 0%, while the fatality rate for those who were not vaccinated was 0.6%, more than six times that of the seasonal flu. The quarantine authorities explained that they are considering a medical site proposal for recognizing rapid antigen testing as the final confirmation, and that the KDCA will make a separate announcement once it is concluded. Central Disaster and Safety Counters Headquaters made this announcement at a regular briefing on the 8th. The quarantine authorities analyzed and announced the fatality rate and vaccination effect of Omicron. According to an analysis of 135,000 people who completed mutation analysis from April last year to February this year, the fatality rate of Omicron among those who completed the third inoculation was 0.07%. This means that the tertiary vaccination lowers the Omicron fatality rate to a level similar to the seasonal flu fatality rate of 0.05 to 0.1%. Those under the age of 60 who completed the third vaccination had a fatality rate of 0%. However, the fatality rate for non-vaccination was analyzed to be 0.6%, and if not vaccinated, the fatality rate of Omicron was more than six times that of the seasonal flu. The explanation is that the difference in accuracy is not significant as the kit's own inspection ability is measured based on the standardized method of sampling in the approval process of the Ministry of Food and Drug Safety. The quarantine authorities added that in reality, the expert's sample collection method is more accurate than the general public, so it is reflected to some extent in the final accuracy and it is difficult to produce statistics on how significantly the accuracy of experts' sample collection increases the accuracy of rapid antigen testing. Son Young-rae, head of the social politics team, said, "The accuracy of the expert rapid antigen test and personal diagnostic kit is almost the same," adding, "However, in the actual field, the expert's collection method is more accurate than that of individual citizens, so the accuracy increases." It's hard to infer how much more statistically accurate it is," he said. The KCDA is currently reviewing the plan to recognize rapid antigen testing as a final confirmation, Son said. "We are considering all measures considering the accuracy and prevalence of the test."
- Policy
- Finding ways to utilize the Patent-approval linkage system
- by Lee, Hye-Kyung Mar 10, 2022 05:53am
- The MFDS plans to prepare a collection of data so that domestic pharmaceutical companies can pioneer the Chinese pharmaceutical market by utilizing the Patent-approval linkage system within the first half of this year. The MFDS recently announced research services for "Overseas the Patent-approval Linkage System Survey and Domestic Impact Analysis." Following the signing of the Korea-U.S. FTA in 2007, the Pharmaceutical Affairs Act has been fully implemented in Korea since March 2015 through the revision of the Pharmaceutical Affairs Act twice in 2012 and 2015. The study was designed to identify recent overseas systems and trade agreements related to the Patent-approval linkage system and to support pharmaceutical companies' development of overseas markets in the future, and will run until October 31 with 55 million won in project costs. The core of this study is a plan to utilize the Patent-approval linkage system in Korea under the recently revised Chinese patent law. Since June 1 last year, China has been fully implementing the Patent-approval linkage system. This study identifies not only the size and status of the Chinese pharmaceutical market, but also the Chinese pharmaceutical market, including Korea's exports to China, and recently investigates the organization, system, and implementation status of Chinese pharmaceuticals. Based on this, the MFDS plans to prepare a data book by July to support domestic pharmaceutical companies to explore the Chinese pharmaceutical market. Investigation and analysis of international agreements are also conducted not only in China but also in relation to the Patent-approval linkage system. Through this study, the MFDS plans to investigate the recent issues and trends of countries subscribed to the agreement by comparing the background and operation status of comprehensive and progressive CPTPP, and commonalities and differences with other trade agreements such as the Korea-U.S. FTA. Based on the results of the survey, the impact analysis on the Patent-approval Linkage System, which is expected when Korea joins the CPTPP, and countermeasures of domestic pharmaceutical companies to enter overseas markets will be presented together.
- Company
- Keytruda for esophageal cancer indications is imminent
- by Eo, Yun-Ho Mar 08, 2022 06:08am
- Keytruda, an immuno-cancer drug, is expected to be able to be prescribed for esophageal cancer. According to related industries, the MFDS has finally reviewed the first round of local progressive or metastatic esophageal cancer (GEJ), in which PD-1 inhibitory immuno-cancer drug Keytruda (Pembrolizumab) of MSD is non-resectable. Permission is expected as early as the second quarter. Following colon cancer in June last year and triple negative breast cancer in July last year, the area of indications is rapidly expanding. Keytruda's indications for esophageal cancer and gastroesophageal junction cancer were approved by the U.S. FDA in March last year and the European EMA in June last year. Keytruda's effectiveness for esophageal cancer has been proven through a phase 3 clinical KEYNOTE-590 study. Keytruda, 5-FU, and Cisplatin combination therapy demonstrated statistically significant OS and PFS improvements over 5-FU+cisplatin in all pre-designated study groups. Keytruda, 5-FU, and Cisplatin reduced the risk of death by 27% compared to 5-FU+Cisplatin, and the risk of disease progression or death by 35%. In the patient group with a PD-L1 expression rate of 10 or higher, Keytruda and 5-FU+Cisplatin reduced the risk of death by 38% and the risk of disease progression or death by 49%, compared to 5-FU and Cisplatin. In patients treated with Keytruda, 5-FU, and Cisplatin combination therapy, the objective response rate (ORR) was 51.1%, of which the CR ratio was 5.9% and the PR ratio was 45.2%.
- Company
- Generation shift of ALK targeted drugs... rise of Alecensa
- by Mar 08, 2022 06:08am
- The targeted cancer therapy market for patients with ALK mutations has been restructured around second-generation drugs. The share of the first-generation drug Xalkori, which used to occupy 2/3 of the market fell to 20%, and the representative second-generation drug ‘Alecensa' took over the market. However, Alunbrig, a latecomer into the second-generation treatment market, has been rapidly chasing the market leader Alecensa that has currently occupied over half of the market. According to the pharmaceutical research institution IQVIA on the 8th, the Anaplastic Lymphoma Kinase (ALK) tyrosine kinase inhibitor (TKI) market recorded ₩54.4 billion last year, which was an 11.9% and ₩48.6 billion increase from the previous year. The ALK TKI market, whose doors were first opened by ‘Xalkori (crizotinib),’ is used to treat patients with ALK-positive non-small-cell lung cancer (NSCLC). With the introduction of next-generation drugs that have demonstrated improved efficacy, a total of 5 drugs are currently present in the market. In addition to second-generation drugs ‘Zykadia (ceritinib),’ ‘Alecensa (alectinib),’ ‘Alunbrig (brigatinib),’ a third-generation drug ‘Lorviqua (lorlatinib)’ has also entered the market last year. Compared to 2017, the market transition from first-generation to second-generation drugs is quite clear. In 2017, Xalkori had an oligopoly over the market as the only ALK TKI option and accounted for 86% of the market. Its sales had recorded nearly ₩36.5 billion that year, followed by the first second-generation drug, ‘Zykadia,' which made ₩5.1 billion, then Alecensa's ₩1.1 billion. However, in 4 years in 2021, the landscape had completely shifted with the proof that second-generation drugs have a better effect in patients with brains metastasis. With doctors opting for second-generation drugs in the first line, Alecensa’s market share rose to 60%. Shares of another second-generation drug, Alunbrig, took over 15% of the market, making ₩8 billion in sales. Xalkori’s sales fell to 24%, making ₩13.1 billion. Roche’s Alecensa and Takeda’s Alunbrig are representative second-generation ALK TKIs. By market entry, Alecensa entered the market 2 years earlier than Alunbrig. Alecensa expanded its indication to the first-line in 2018 and was granted reimbursement in December of the same year to quickly replace Xalkori. In 2018, Alecensa sold ₩10.4 billion, which was 1/4 of the sales made by Xalkori (₩49.6 billion). After receiving reimbursement in 2019, Alecensa sold ₩22.1 billion and exceeded the Xalkori's ₩20.3 billion in sales. Alecensa's sales increased to ₩29.3 billion in 2020 and ₩32.7 billion in 2021. Alunbrig, which was approved in December 2018, aimed to rapidly enter the market and catch up with Alecensa. As soon as it was approved for the first linein August 2020, the company applied for its reimbursement and succeeded in expanding its reimbursement in only 7 months. With the reimbursement approval as a first-line treatment that was granted in April last year, the company is working to expand its share in the market. Sales in 2020 were ₩3.9 billion won, far short of that of Alecensa and Xalkori, sales increased 102.9% to ₩8 billion after the reimbursement expansion. On the other hand, the first second-generation drug, Novartis’s Zykadia, has been going down a completely different path. Zykadia’s sales which had surged to ₩5.1 billion in the past had started to fall sharply with the introduction of Alecensa. It sold ₩2.2 billion in 2018, ₩0.9 billion in 2019, and only ₩0.5 billion last year. The anlaysis is that this rapid drop in sales is due to the relatively higher instance of side effects compared to Alecensa or Alunbrig. The first third-generation ALK TKI was introduced last year. Xalkori’s developer Pfizer had introduced the first-ever third-generation drug Lorviqua. Lorviqua was first to be approved as a second-line treatment for ALK-positive NSCLC in July last year and rose as a new alternative due to its ability to manage the resistance developed after first-line treatment. Lorviqua can manage the resistance caused by G1202R mutation, as well as those by F1174L (Zykadia), I1171T/N/S (Alecensa), E1210K (Alunbrig). Lorviqua is not listed for reimbursement yet and is expected to generate sales in earnest after passing the National Health Insurance Service’s Drug Reimbursement Evaluation Committee and drug negotiations with the authorities. In particular, as Lorviqua is attempting to expand its territory into first-line treatment for ALK-positive patients, its its competition with second-generation drugs are also being expected to arise soon. The drug already owns a first-line indication for ALK-positive NSCLC in the US and Euope.
- Company
- Domestic production of Sputnik V was delayed
- by Kim, Jin-Gu Mar 08, 2022 06:08am
- Due to the war between Russia and Ukraine, shipments of domestic companies that consignment produce the COVID-19 vaccine Koruspharm developed by Russia are also being postponed indefinitely. Koruspharm said in a telephone interview with Dailypharm on the 4th, "We are continuously talking to the Russian side via e-mail or phone call even after the war broke out," adding, "For now, product shipments have been stagnant due to the Ukrainian crisis." According to Koruspharm, the company has currently produced Sputnik undiluted solutions for 10 million people, of which 5.3 million have completed the production of finished products. This means that the quantity that can be shipped immediately amounts to 5.3 million people. Koruspharm originally planned to supply the finished vaccine to Southeast Asia, the Middle East, and South America in line with the Russian order. Administrative procedures for shipment have also been completed. However, orders from Russia have been delayed day by day, and shipments have not been expected recently due to the outbreak of war. Koruspharm is looking for buyers directly through its own network. The company explained that it has even confirmed its intention to purchase from some Middle Eastern countries. An official from Koruspharm said, "Iit is true that Russia lacked marketing capabilities. We are looking through our own network and have confirmed our intention to purchase from some places." He went on to say, "Russia and the country must officially sign contracts in order to lead to actual shipments. When the contract between the two countries is signed, we plan to ship it immediately, he said. "We have concluded discussions with Russia to ship the rest of the vaccines in this way in the future." Regarding the payment for consignment production of vaccines, he said, "Uncertainty has grown in the collection of payments as the U.S. and Europe imposed massive economic sanctions against Russia." "To solve this problem, we are discussing with the Russian side how to receive payments directly from vaccine buyers instead of Russia," he said. Koruspharm signed a "Sputnik V consignment production contract" with RDIF in September last year. Since then, Koruspharm has formed a consortium with six companies and one institution, including Binex, Boryung Biopharma, ISU Abxis, Chong Kun Dang Bio, Quratis, and Andong Animal Cell Demonstration Support Center. The consortium plans to build production facilities of more than 100 million doses per month.
- Policy
- The fatality rate of Omicron is low
- by Lee, Jeong-Hwan Mar 08, 2022 06:08am
- The quarantine authorities announced a full-fledged easing of distancing, saying that it is becoming clearer that the COVID-19 Omicron mutation has a lower severity and fatality rate than the delta mutation. From the 5th to the 20th, the 22 o'clock operating hours limit, which will be applied to groups 1, 2, and 3, will be increased by 1 hour to 23:00, but the next adjustment of distance will be eased. In particular, the quarantine authorities urged government agencies to actively participate in blood donation and spread the voluntary participation of the people, citing the continued stage of interest in domestic blood reserves of more than 3 days and less than 5 days. On the 4th, Lee Ki-il, the first controller of the Central Disaster and Safety Countermeasure Headquarters, made the remarks at a regular COVID-19 briefing. The quarantine authorities explained that it is not enough to keep a recently relaxed distance to resolve the difficulties of self-employed and small business owners. Considering that the quarantine system is being reorganized around the management of high-risk groups, the intention is to further ease distancing. Accordingly, the quarantine authorities will adjust the distance measure, which was originally scheduled to be applied by the 13th. The quarantine authorities analyzed that unlike Delta, it is becoming increasingly clear that Omicron has a low severity and fatality rate, and that the effectiveness and efficiency of distancing are decreasing due to its high propagation power. Based on this, the 22-hour operation time limit, which is applied to some facilities of groups 1, 2, 3 and other groups, will be eased by 1 hour from the 5th to the 20th until 23:00. Private gatherings maintain standards that allow up to six people nationwide regardless of whether they are vaccinated or not. The quarantine authorities plan to lower the level of distance in earnest after this easing. Pre-recommendations are also implemented for all inpatients with quasi-severe and moderate symptoms. As of the 4th, 731 patients with COVID-19 quasi-severe and moderate hospitalization who have passed 10 days from the date of sample collection are advised in advance to move to general beds. If the medical staff determines that additional quarantine treatment is necessary and submits explanatory data on all recommendations, if the need for additional quarantine is recognized through consultation, they can continue to receive treatment in the same hospital room without all or lost. If treatment is needed for an underlying disease, pt will move to a general bed and continue to receive treatment. The quarantine authorities also requested blood donation to stabilize blood supply and demand. Recently, the stage of interest in blood retention of more than 3 days and less than 5 days continues. In order to stabilize blood supply and demand, government agencies will take the initiative for a month in March and conduct group blood donations through a "Continuing Blood Donation Event." More than 1,600 employees from 31 organizations, including the Ministry of Employment and Labor, the MOHW, the Ministry of National Defense, the Maritime Police Agency, and the Ministry of Public Administration and Security, will participate in the event. In addition, the quarantine authorities asked the public to participate in blood donation at a blood donation house or a blood donation cafe close to them.
- Policy
- DREC rejects reimb of neurofibromatosis drug ‘Koselugo'
- by Lee, Tak-Sun Mar 08, 2022 06:08am
- The new neurofibromatosis drug ‘Koselugo (selumetinib),’ failed to receive reimbursement benefits in Korea. The Health Insurance Review and Assessment Service decided the drug as a ‘non-benefit' after deliberation on the adequacy of providing medical benefit for the drug at the 3rd Drug Reimbursement Evaluation Committee that was held this year. The drug, which is sold by AstraZeneca Korea, is used to treat neurofibromatosis type 1 (NF1) with symptomatic, inoperable plexiform neurofibromas in pediatric patients over the age of 3. In the past, patients had to rely on symptomatic treatment due to the lack of appropriate treatment options. This was why expectations were high for Koselugo as the new treatment option in an area with a dire unmet need. Around half of the neurofibromatosis type 1 patients have plexiform neurofibroma (PN), and it may appear anywhere in the body. Depending on the position and size, it may limit the range of motion or bring pain or external deformity in patients, The internally developed tumors may pressure the internal organs. Most rumors are positive and grow relatively slow, but some progress into malignant tumors and can lead to an increased risk of developing breast cancer in women. The prevalence is about 1 in 3,000. The drug was approved by the MFDS in May last year based on the Phase II SPRINT study. In the study, 68% of the patients who were administered Koselugo saw a decrease in tumor volume by over 20% and achieved its primary efficacy endpoint, the objective response rate (ORR). Also, 82% of the patients who showed partial response showed saw a duration in their response of over 12 months. Compared to how only half of the untreated patients experienced disease progression after 1.5 years, only 15% of the patient who used Koselugo experienced disease progression in 3 years. As the drug costs ₩200 million a year, the demand for its reimbursement had been quite strong, but the company was unable to even pass DREC deliberations, the 1st gate to reimbursement in Korea.
