- LOGIN
- MemberShip
- 2026-04-09 06:29:30
- Company
- Companies entering the Middle East caught in the crossfire
- by Kim, Jin-Gu Oct 12, 2023 05:38am
- On the 7th (local time), the Palestinian armed political faction Hamas attacked Israel (CNN photo) On the 7th (local time), the Palestinian armed political faction Hamas attacked Israel (CNN photo). As the situation in Israel and nearby regions has deteriorated rapidly due to large-scale airstrikes by the Palestinian armed political faction Hamas, domestic pharmaceutical and bio companies that have entered or are seeking to enter the Middle East appear to be paying close attention. The prevailing view is that the immediate damage will not be significant, but there is also the view that if the conflict spreads throughout the Middle East region, disruption to each company's long-term plans will be inevitable. Middle East pharmaceutical export companies, “Israel’s share is small… the damage will not be significant.” According to the pharmaceutical industry on the 11th, companies that export pharmaceuticals directly to Middle Eastern countries expect that the immediate damage from this dispute will be minimal. This is because Israel, where the conflict occurred, does not have a large export volume, and in the case of neighboring Middle Eastern countries, the terms of pharmaceutical export contracts are structured so that it is difficult to cause immediate damage. An official from a pharmaceutical company that exports medicines to Saudi Arabia and the United Arab Emirates (UAE) said, "Domestic pharmaceutical companies trading with Middle Eastern countries are generally not very shaken." “Because it’s not big,” he said. In fact, according to the Korea Customs Service, medicines exported from Korea to Israel last year amounted to $7.74 million. Compared to major export countries such as the UAE ($30.29 million), Saudi Arabia ($26.04 million), Egypt ($22.85 million), and Iraq ($18.88 million), it is around 25-40%. This year is also similar. As of last August, pharmaceutical exports to Israel amounted to $3.79 million, less than half of those to Saudi Arabia ($24.31 million), the UAE ($17.05 million), Iraq ($12.4 million), and Egypt ($9.77 million). The terms of contracts with Middle Eastern countries are also cited as a reason why immediate damage is not expected. It is said that usually, pharmaceutical export contracts with countries where there is a risk of conflict are concluded with the supply of medicines after receiving an advance payment. An official from another pharmaceutical company that exports medicines to Middle Eastern countries said, “In the case of major Middle Eastern countries where there is a possibility of conflict, contracts are written by exchanging cash instead of a letter of credit transaction.” He added, “Usually, 100% is received in advance or 50% is paid first. “We receive the remainder after supplying medicine,” he explained. He added, “Generally if a contract cannot be fulfilled due to war or other reasons, the contract period is automatically extended unless there are special circumstances.” However, it was also predicted that damage would be inevitable if this conflict spreads to nearby countries and is prolonged. He said, “The key export countries are Saudi Arabia, Egypt, and the UAE. We are closely watching the possibility of conflict spreading to these countries.” SK Bioscience, SK Biopharmaceuticals, Daewoong, etc. seeking to enter the Middle East “There will be no significant impact as it is in the early stages” Large pharmaceutical companies seeking to enter the Middle East region are in a similar mood. As expansion into the Middle East region has not yet been confirmed, the prevailing view is that the damage will be minimal. An official from SK Bioscience said, “We are just beginning to explore expansion into the Middle East,” and added, “This dispute will not have a significant impact on business discussions.” SK Bioscience proposed a partnership to build a base vaccine hub in the Middle East early this year. SK Bioscience's plan is to enter the Middle East market based on its successful experience in developing a COVID-19 vaccine. Officials from SK Biopharmaceuticals and Daewoong Pharmaceutical also agreed, saying, “We are closely monitoring the local situation, but we do not expect there to be a significant impact on the existing plan.” Last August, SK Biopharmaceuticals signed a technology export contract for the epilepsy treatment drug 'Cenobamate' with Middle Eastern pharmaceutical company Hikma. In addition, it was decided to grant priority negotiating rights to Hikma when products are launched in 16 Middle Eastern countries, including Saudi Arabia, the UAE, and Egypt. Daewoong Pharmaceutical applied for product approval for Fexuclue, a P-CAB-based gastroesophageal reflux disease treatment, to the Saudi Arabian health authorities at the beginning of the year. In May last year, product approval for botulinum toxin ‘Nabota’ was received in Saudi Arabia. Daewoong Pharmaceutical plans to target the Middle East region, including Saudi Arabia, focusing on Nabota and Fexuclue. In the case of Samsung BioLogics, it signed a CDMO contract with the Israeli pharmaceutical company KAHR Medical in 2021 for an immunotherapy drug (substance name: DSP502). It provides a one-stop service from cell line development of DSP502 to clinical raw drug production, finished product production service, and IND approval. Regarding this, a Samsung BioLogics official said, “CDO contracts usually do not have a long supply period.” He said, “It has been two and a half years since the contract was signed, and the contractual supply is now almost complete.”
- Company
- Bukwang Pharm’s Ozanex lands in major general hospitals
- by Nho, Byung Chul Oct 12, 2023 05:37am
- Bukwang Pharm announced that its new topical antibiotic Ozanex cream(ozenoxacin)’ recently passed the drug committee of Seoul St.Mary’s Hospital and can be prescribed in major general hospitals in Korea. Ozanex, a topical antibiotic that Bukwang was first to introduce to Korea, is currently being prescribed at various major general hospitals nationwide including Seoul St.Mary’s Hospital, Samsung Medical Center, Seoul National University Hospital, Sinchon Severance Hospital, Asan Medical Center, Hanyang University Hospital, Korea University Hospital, Chonnam National University Hospital, and Kyungpook National University Hospital. Ozanex was approved by the U.S. FDA in December 2017 and newly released in Korea in December last year it is a prescription drug that is effective against staphylococcus aureus(S. aureus) and Streptococcus pyogenes(S. pyogenes), the main causes of infectious skin diseases, and is indicated for the short-tem topical treatment of impetigo in patients aged 2 months and older1. For infectious skin diseases, topical antibiotics need to be used for an appropriate period at an appropriate dose according to an expert's accurate diagnosis and prescription to reduce the development of resistance and provide effective treatment. According to Professor Jung-Soo Kim of the Department of Dermatology at Hanyang University Guri Hospital, “Topical antibiotics that can treat impetigo include ozenoxacin and mupirocin. Ozenoxacin is a new non-fluorinated quinolone antibiotic that offers improved resistance and safety. Staphylococcus aureus and Streptococcus pyogenes are the most common cause of various skin infectious diseases, including impetigo. Oozenoxacin demonstrated bactericidal activity against these two strains. In particular, in a large-scale clinical study that involved patients with impetigo, the bacterial culture test showed bacteriological eradication as early as day 3 of treatment, with a negative conversion rate of 87%. Based on such results, HCPs expect the drug will effectively reduce the contagiousness of impetigo in the early stages. A Bukwang Phamr official said, “Ozanex will settle as a new option for infectious skin diseases including impetigo. We expect Bukwang’s various skin disease treatments to support HCPS and their treatment practices in Korea.”
- Opinion
- [Reporter’s View] KRPIA pulls out ‘structural reform' card
- by Eo, Yun-Ho Oct 12, 2023 05:37am
- The Korean Research-based Pharmaceutical Industry Association (KRPIA) has pulled out the ‘expenditure structure reform’ card as a solution to finance new drug expenditures. Although the message seems somewhat familiar, it is a new and unprecedented request. On the 4th, KPRIA released the results of 'A study on the analysis and rationalization of drug expenditures for new drugs in Korea' conducted by Professor Jong-Hyuk Lee from Chung-Ang University’s College of Pharmacy. Study results showed that the expenditures spent on new drugs from the national health insurance finances accounted for 8.5% of total drug costs and 2.1% of the total national health insurance medical costs. In particular, the financial impact made by new drugs on national health insurance finances was among the lowest when compared with other OECD countries. More specifically, the annual drug expenditure spent on each new drug amounted to KRW 6.1 billion whilst the total drug expenditure spent in Korea over the past 10 years amounted to KRW 164.2 trillion. In this analysis, the financial expenditures spent on items that were waived pharmacoeconomic evaluation data submissions and were subject to RSA, which account for most of the new drugs used to treat serious diseases such as cancer and rare diseases, were low, accounting for 0.3% and 2.7%, respectively, of the total drug cost. In addition, the financial impact of new drugs according to their serious disease classification status showed that drug expenditures spent on new drugs for severe and rare diseases (cancer, rare diseases) were found to only account for 3.3% of the total drug costs, suggesting the low treatment access available for domestic patients with severe and rare diseases. If so, why are studies and claims that should have been made a long time ago being raised and regarded as unusual now? The fact that new drugs account for a small portion of drug expenditures means that drug costs of existing drugs, not new drugs, account for a sizable portion of the expenditures. In other words, Korea spends 91.5% of its drug expenditures on drugs that are not new. The direction of improving the expenditure structure, which the KRPIA has suggested, is to increase the proportion of new drugs and to reduce the proportion of drug expenditures spent on drugs that are not new. It is an agenda that is bound to bring conflict of interest between new drug developers and non-developer pharmaceutical companies. However, there is no doubt that new drugs are important to Korea’s society as a whole. Prior to pointing out the limitations and reliability of the research published this time; it is necessary to consider Korea’s current expenditure structure. However, the number of expensive drugs is indeed increasing, and the number of drugs that remain non-reimbursed due to their potential financial impact is also rising. Korea has been known for its strong health insurance coverage supported by the national health insurance system framework. Even if the government had been a little insensitive to the changes in trends in related industries, new drugs have now surely become mainstream. Therefore, it is now time to consider adapting and evolving Korea’s expenditure structure and priorities accordingly.
- Policy
- Overseas drug price comparison plan to be announced
- by Lee, Tak-Sun Oct 12, 2023 05:37am
- The final plan will likely be finalized after gathering opinions from the pharmaceutical industry. It is expected that a plan to reevaluate overseas drug prices will be revealed around the end of the year. Health authorities plan to prepare a reevaluation plan by the end of the year and then proceed with it starting next year. According to the industry on the 11th, the HIRA is in the process of preparing a draft plan for comparative reevaluation of overseas drug prices. The draft is expected to determine the reevaluation method and target, as well as the foreign drug price reference formula. Once the draft is completed, a working group is expected to be established to collect opinions from the pharmaceutical industry. Pharmaceutical organizations such as the Pharmaceutical and Biotechnology Association have requested a working group to establish a plan to compare and reevaluate overseas drug prices. As health authorities plan to prepare a final plan by the end of the year, discussions are expected to accelerate after the National Assembly audit. The pharmaceutical industry predicts that reevaluation will proceed sequentially, mainly focusing on chronic disease drugs whose patents have expired. There are concerns that if drug prices are reduced through reevaluation, the damage will increase. Accordingly, the possibility of filing a lawsuit against the government is already being discussed. Since the draft has not yet been released, it is difficult to make hasty predictions. An official in the pharmaceutical industry said, “If the completed plan is made public around the end of the year, there is a high possibility that the final plan will be prepared quickly through the working group after the National Assembly inspection. The pharmaceutical industry is concerned about major damage, and it seems that it will be possible to make a prediction after looking at the draft." The comparative reevaluation of overseas drug prices is also included in the 1st National Health Union Plan announced in 2019. Last year's HIRA included Canada as a reference country for overseas drug prices in addition to the existing seven countries (US, UK, Germany, France, Italy, Switzerland, and Japan), laying the foundation for reevaluation. Initially, there was an attempt to include Australia, but it was canceled due to opposition from the pharmaceutical industry. In December of last year, at a public hearing on measures to improve health insurance sustainability and support essential medical services, a plan to reevaluate foreign drug prices for chronic disease drugs whose patents have expired was formalized, and earlier this year, the HIRA announced that it would prepare a plan by the end of the year.
- Policy
- AD drug Dupixent’s fails 1st negotiations for RSA renewal
- by Lee, Tak-Sun Oct 12, 2023 05:37am
- The first round of negotiations to renew the risk-sharing agreement (RSA) contract for the severe atopic dermatitis treatment ‘Dupixent (dupilumab, Sanofi)’ has fallen through. The company and the National Health Insurance Service plan to discuss Dupixent’s RSA renewal through additional negotiations. According to the industry on the 11th, the first round of negotiations for Dupixent's RSA renewal fell through. Dupixent was listed for reimbursement through the RSA in 2020. Three types of RSA - refund type for initial treatment, refund type, and expenditure cap type, were applied for reimbursement. At the time, the Ministry of Health and Welfare explained, "The pharmaceutical company signed an agreement to refund a part of the amount administered for a certain period of time, and then refund a certain percentage of the drug claims amount to the NHIS while limiting the total expenditure amount." Accordingly, the first biological drug used to treat severe atopic dermatitis was listed with reimbursement in Korea. At the time of the initial reimbursement listing, its subjects were limited to adult patients with moderate-to-severe atopic dermatitis who are not adequately controlled with topical treatments or for whom these treatments are not recommended. However, starting in April of this year, a new reimbursement category was added to reimburse the drug for the treatment of severe atopic dermatitis in children and adolescents aged 6 to 17. Dupixent’s sales rose vividly after the reimbursement listing. Based on IQVIA, it posted sales of KRW 72.2billion in 2021, and KRW 105.2 billion in 2022, As the treatment of pediatrics and adolescent are now also covered and the company is seeking to expand coverage to include severe asthma and atopic dermatitis in infants and toddlers, the amount of health insurance claims are likely to only increase further. Therefore, price cuts and refund rate adjustments are inevitable during RSA contract renewal. With the RSA term expiring by the end of this year, the NHIS and Sanofi were unable to reach an agreement in the first round of negotiations that began at the end of July. However, as additional negotiations are legally granted, it is expected that an agreement will be reached before the end of the RSA term. So far, no final breakdown has occurred during RSA contract renewal negotiations. If the RSA contract is not renewed, the drug will be converted to non-reimbursed status.
- Policy
- 75% of patients who administered Kymriah is not effective
- by Lee, Jeong-Hwan Oct 11, 2023 05:37am
- More than 75% of patients who received the ultra-high-priced drug Kymriah, which costs 360 million won in one dose, did not have an improvement effect. On the 6th, Kim Young-joo, a member of the National Assembly Health and Welfare Committee (the Democratic Party, Yeongdeungpo Gap) received data from the Health Insurance Review and Assessment Service on the 'Current status of administration of ultra-high-value drugs such as Kymriah and Zolgensma and patient response evaluation'. The world's first CAR-T treatment drug, Kymria,h has an indication for the treatment of adult patients with ▲post-transplant recurrence or secondary recurrence and subsequent recurrence or impresistible B cell-grade lymphoma in children and young adult patients under the age of 25 ▲reactive or non-resistible large B-cell lymphoma (DLBCL) after two or more systemic treatments. Since April 2022, health insurance benefits have been applied, and the patient's copayment has been reduced to a maximum of about 6 million won. Zolgensma, a treatment for spinal muscular atrophy (SMA), costs 1.98 billion won for a single dose of non-paid. Zolgensma was reimbursed in July 2022. Since December 2022, the HIRA has been operating a 'high-drug management system' that monitors the dosing information of ultra-high-drug patients such as Kimlia and Jolgensmaju and the evaluation of the response to drugs after administration. Kymriah had 146 patients who were dosed after reimbursement. Of these, there were 21 cases of pediatric leukemia and 125 patients with megabloid B-cell lymphoma. Their salary contract cost was 52.6 billion won. Jolgensmaju was dosed by 12 people and the cost of the payroll was 23.8 billion won. The cost-effectiveness of these ultra-high-securing new drugs is unclear, so it is implementing a 'patient-based risk-based risk-sharing system' so that the pharmaceutical company will refund a certain percentage of the amount to the healthcare company according to the contract if it is ineffective by tracking the treatment performance of each patient. According to the HIRA, as of August this year, 130 patients with lymphoma who had been treated with Kymriah for 6 months had submitted a response evaluation, of which 99 were classified as eligible for refunds. This means that more than 75% of Kymriah patients did not have a significant improvement effect. Only 1 out of 9 patients who submitted the results were eligible for reimbursement, and more than 88% of patients who received Zolgensma had the treatment effect. Health insurance and pharmaceutical companies negotiate to set the refund ratio, but the refund ratio is kept private. In the case, it is known that the refund rate is less than 50% even if there is no medicinal effect, and the refund rate is more than 50% for Jolgensmaju. The problem is that hundreds of billions of dollars are spent on medicines with low treatment performance. Rep. Kim Young-joo suggested, "In order to prevent blind spots, the target of the benefit should be expanded to patients who need ultra-high-paying treatment, such as Kymriah and Zolgensma. However, it is necessary to increase the refund rate of pharmaceutical companies if there is no treatment effect by strengthening the risk-sharing agent for the sustainable benefits of ultra-high-high-high-high-mouth new drugs." In addition, he emphasized, "The system should be improved so that patients can receive a certain part of the refund if there is no treatment effect because the patient's own burden is high even after paying," he emphasized.
- Policy
- ‘Little progress for NIP support of 9-valent HPV vaccines'
- by Lee, Jeong-Hwan Oct 11, 2023 05:36am
- The number of patients receiving treatment for head and neck cancer and oropharyngeal cancer, both of which are caused by HPV (human papillomavirus), is on the rise. In just a decade, from 2013 to last year, the number of head and neck cancer patients increased by 41.6%, and the number of oropharyngeal cancer patients increased by 56%. In this situation, voices have been raised on how President Suk-Yeol Yoon should actively and promptly fulfill its pledge to ‘expand the subject of HPV vaccination to males and provide support’ through relevant policies. On the 10th, Rep. In-Soon Nam of the Democratic Party of Korea, announced so after analyzing the ‘Patients Treated for Head and Neck Cancer and Oropharyngeal Cancer' data that was submitted by the Health Insurance Review and Assessment Service. The number of head and neck cancer patients increased by 41.6% from 302,960 in 2013 to 429,054 in 2022, and oropharyngeal cancer increased by 56% from 3,847 in 2013 to 6,003 in 2022. By gender, of the 429,054 patients with head and neck cancer, 104,881 (24.4%) were men and 324,173 (75.6%) were women. Among the 6,003 patients with oropharyngeal cancer, 4,890 were men. 81.5%), and 1,113 (18.5%) were women. The national HPV (human papillomavirus) vaccination program began for girls aged 12 in 2016 and then was expanded to include female adolescents aged 12 to 17 and low-income women aged 18 to 26 since 2022. The vaccines provided with national support are bivalent and quadrivalent vaccines, and 9-valent vaccines were not included. Rep. Nam said, “President Yoon, who had been a candidate back then, promised he would extend insurance coverage to Gardasil 9-valent vaccine during his’59-second shorts’ pledge. Also, the People Power Party’s presidential election policy pledge contains ‘Free national HPV vaccination for men starting from the age of 12. However, the expansion of HPV vaccine vaccination to men and support for vaccination has not been implemented.” Meanwhile, according to the 'HPV National Vaccination Status in OECD Countries' data submitted by the Korea Disease Control and Prevention Agency, only 7 countries, including Japan and Mexico, support bivalent or quadrivalent vaccines for female adolescents only like Korea. 18 countries including the United States, Canada, and France provide support for the use of the 9-valent vaccine for both men and women. Rep. Nam said, “The recent HPV vaccine cost-effectiveness analysis report concluded that expanding the target population or converting to a 9-valent vaccine is not agreeable in the current domestic situation. HPV is not only responsible for cervical cancer, but also head and neck cancer, oropharyngeal cancer, and anal cancer. This is why many OECD countries are actively administering HPV vaccines. cost-effectiveness studies. As we are promoting reconducting the cost-effectiveness research, I hope the government will also abide by its word and actively engage in the provision of 9-valent vaccines.”
- Policy
- Eisai’s Jyseleca completes negotiations with NHIS for reimb
- by Lee, Tak-Sun Oct 11, 2023 05:36am
- Eisai Korea has completed negotiations with the National Health Insurance Service for its JAK inhibitor ‘Jyseleca Tab (filgotinib).’ As a result, Jyseleca is now on the verge of being listed for reimbursement in Korea. This drug is the 5th JAK inhibitor introduced to Korea. It has a mechanism of action that selectively inhibits JAK1, the main treatment target for rheumatoid arthritis. JAK1 is a substance that transmits inflammatory cytokine signals. Results of the global FINCH Phase 3 study showed that the drug improved arthritis symptoms by more than 20% at 12 weeks when administered to patients with moderate-to-severe active RA (rheumatoid arthritis) despite continued methotrexate (MTX) treatment. When evaluating the response rate during the 12-week period during which patients showed no response to methotrexate, 66% of patients responded to Jyseleca, and symptom improvement was observed. Last July, HIRA’s Drug Reimbursement Evaluation Committee approved conditional reimbursement for the drug in treating rheumatoid arthritis and ulcerative colitis. At the time, DREC judged reimbursement was adequate when the company accepted a price below the assessed amount. Eisai Korea appears to have accepted a price below the assessed value. In particular, it is said that the company skipped negotiations on the insurance price ceiling that is set with the National Health Insurance Service by accepting a price that is below 90% of the weighted average price of its alternative. Negotiations with the NHIS on the expected claims amount were also recently completed. Accordingly, this drug is expected to be listed for reimbursement after being reported to the Ministry of Health and Welfare’s Health Insurance Policy Deliberation Committee. Other JAK inhibitors that were previously introduced to Korea include Pfizer’s ‘Xeljanz,’ Lilly’s ‘Olumiant,’ ‘Abbove’s ‘Rinvoq,’ and Pfizer’s ‘Cibinqo.’
- Policy
- Hyaluronic acid eye drops, start to revise the standard
- by Lee, Tak-Sun Oct 11, 2023 05:36am
- The HIRA has started to revise the standards to limit the use of Hyaluronic acid sodium eye drops. It is known that the revision of standards will be completed as early as the end of the year, and will be implemented from next year. According to an industry example on the 10th, the HIRA has entered a review of the revision of the standards for sodium hyaluronic acid eye drops at the request of the Ministry of Health and Welfare. Therefore, we plan to hold an expert advisory meeting soon to continue related discussions. Earlier, the Pharmaceutical Benefit Evaluation Committee held on the 6th of last month recognized the benefit of endogenic diseases for Hyaluronic acid eye drops that have been reassessed. Exogyneutic disease is caused by post-surgery, pharmaceutical, trauma, wearing of content lenses, etc. Endogenic diseases include Sjogren's syndrome, skin blemish syndrome (Steven's Johnson syndrome), and dry eye syndrome. The main indication of hyaluronic acid eye drops is an endogenic disease, so related companies sighed with relief. However, instead of recognizing the benefit of endogenic diseases, the drug review committee put a clue that it is necessary to set the prescribed amount per patient's visit and the total annual prescription amount per patient in the standard for proper use. As a result, it is more likely to affect sales due to the revision of standards. During the discussion of the re-evaluation, it was known that a plan to limit 1 bottle of 60 to 4 per year was also considered. It is said that the HIRA reported the results of the drug level to the Ministry of Health and Welfare, and the Ministry of Health and Welfare requested a revision of the salary standard. Therefore, it is expected to speed up the revision of salary standards through expert advisory meetings, etc. Since the results of this year's benefit decent revaluation will be reflected from January next year, it is known that the discussion on the revision of the salary standard of the Hyaluronic acid drop system will be completed within this year and implemented at the same time as the results of the reassessment in January next year. An official from a related company explained, "If you limit the amount of use, even if the benefit is recognized for endogenic diseases, damage to sales is inevitable." The three-year average claim amount of hyaluronic acid eye drops is 231.5 billion won, and about 400 items are listed as reimbursement.
- Company
- Pricing negotiations for Koselugo’s reimb start after 2 yrs
- by Eo, Yun-Ho Oct 11, 2023 05:36am
- Koselugo, which is a treatment for pediatric patients with neurofibromatosis, has entered its last gateway to receiving reimbursement in Korea. According to industry sources, AstraZeneca has recently started drug pricing negotiations with the National Health Insurance Service to set the insurance price for its new neurofibromatosis drug, Koselugo. Whether Koselugo, which passed the Health Insurance Review and Assessment Service’s Drug Reimbursement Evaluation Committee review on the 7th of last month after 3 attempts, will be able to settle on a final drug price during negotiations and be listed for reimbursement remains to be seen. This drug has remained non-reimbursed for 2 years now. It has only been able to enter the NHIS negotiation stage after much trouble. Koselugo, which was designated as the first drug subject to fast-track review in October 2020, obtained marketing authorization from the Ministry of Food and Drug Safety on May 28, 2021, and submitted an application for its reimbursement in May 2021, but failed to pass the DREC gateway in March last year. The company quickly supplemented the data and restarted discussions in April this year, bringing the situation to what it has become now. Until now, patients had to rely only on symptomatic treatment for neurofibromatosis due to the lack of an appropriate treatment option. Neurofibromatosis is a rare disease. 85% of the patients with neurofibromatosis have neurofibromatosis type 1 (NF1), which is caused by a mutation in the neurofibromin tumor suppressor gene located on chromosome 17. The incidence of NF1 is approximately 1 in 3,000. Its first symptom is café-au-lait spots 1 to 3 centimeters in diameter early in life. Since then, the patients have experienced Optic nerve gliomas (brain tumor) at age 6, and scoliosis around age 6-10. In adulthood, lisch nodules, or iris hamartomas, occur predominantly in patients with NF1. If possible, treatment includes surgical removal of affected sites or chemotherapy and radiation therapy. However, most recur even after surgery, and as the patient must undergo a major operation, its treatment puts an immense burden on both the medical staff and the patient. Recurrence is even more frequent among pediatric patients, which means the patients must live with painkillers and often suffer from speech and movement disorders even after receiving several operations. Koselugo was jointly developed by AstraZeneca and MSD. The drug blocks the activation of MEK to inhibit the growth of cell lines. In the Phase II SPRINT study that became the basis for Koselugo’s approval, Koselugo reduced tumor size by over 20% in 68% of the patients who received Koselugo and achieved its primary endpoint of ORR. Also, 82% of the patients who showed a partial response had sustained responses lasting at least 12 months. In contrast to the non-treated patients, half of whom experienced disease progression 1.5 years after diagnosis, only 15% of patients using Koselugo showed disease progression at year 3.
