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- 2026-04-12 10:11:26
- Company
- Will Luxturna be reimbursed by the end of this year?
- by Eo, Yun-Ho May 11, 2022 05:56am
- Attention is focusing on the procedure for registering insurance benefits for another one-shot gene therapy. According to related industries, discussions are underway to register the benefit of Novartis' Inherited Retinal Dystrophy (IRD) treatment Luxturna. As the application for benefits was submitted in September last year, it remains to be seen whether it will be possible to announce the HIRA's drug benefit evaluation committee within the first half of the year. Depending on the speed of the procedure, Luxturna can enter the benefit right within this year. Luxturna restores function by replacing the deficient and defective RPE65 gene, one of the causes of IRD, with a normal gene only once administered. In other words, fundamental treatment of diseases is possible. The drug was designated by the U.S. FDA for Breakthrough Therapy in 2014, Orphan Drug in 2016, and Priority Review in 2017, and was quickly approved in 2017. IRD is a rare and intractable disease that causes visual loss due to mutations in the genes responsible for the structure and function of cells in the retina. It includes more than 20 different ophthalmic diseases and has about 300 causative genes. IRD, which is caused by RPE65 gene mutation, causes abnormalities in the visual cycle in the retina, which converts visual information into neural signals and transmits it to the brain. The RPE65 gene mutation reduces the RPE65 protein, which is essential for visual circuits, and destroys retinal cells, gradually narrowing the field of view, and eventually reaching blindness. Kang Se-woong, chairman of the Korean Retina Society (Samsung Medical Center's ophthalmology professor), said, "Luxturna's single injection is expected to restore not only vision but also independent walking without the help of a guardian"
- Company
- Domestic COVID-19 vaccine developers at a crossroads
- by Kim, Jin-Gu May 11, 2022 05:55am
- HK Inno. N announced it will voluntarily discontinue the development of its COVID-19 vaccine due to the sudden change in the spread of COVID-19 in Korea. With the marketing approval of SK Bioscience’s COVID-19 vaccine imminent, the indecisiveness among the other vaccine developers in Korea is increasing According to industry sources on the 10th, HK Inno. N has decided to discontinue the development of its COVID-19 vaccine, ‘IN-B009’ in Korea. The company had been developing a recombinant vaccine, the same as SK Bioscience’s GBP510 or Novavax’s Nuvaxovid in terms of platforms. The company pointed to the rapid change in the Korean COVID-19 situation as a reason for discontinuing its trial. Unlike in July last year when HK Inno. N received approval for the Phase 1 trial of IN-B009, the company said the need for COVID-19 vaccines had reduced significantly. HK Inno. N said, “Most of the people in Korea have secured immunity to COVID-19, being infected or through multiple vaccinations. The COVID-19 situation has now changed rapidly. With the situation moving to the endemic phase, there is no clear purpose for us to enter a late-stage clinical trial." ◆Second company after Genexine to discontinue development… 7 companies remain The company became the second after Genexine to drop the development of its vaccine. Before HK Inno. N, Genexine had announced that it will voluntarily discontinue its Phase II and Phase III trials in March. At the time, Genexine also explained that it “determined that the business feasibility of GX-19N was low in the rapidly changing COVID-19 market in consideration of the global vaccine supply and demand.” With Genexine and HK Inno. N’s voluntary withdrawal, 7 domestic COVID-19 vaccine developers now remain in the market. The companies that remain are SK Bioscience, Eubiologics, Cellid, Geneone Life Science, Quratis, Eyegene, and ST Pharm. Among the companies, SK Bioscience’s GBP510 is soon to be commercialized. On the 29th of last month, the company applied for the marketing authorization of GBP510 under the name ‘SKY Covione.’ The drug is currently under review by the Ministry of Food and Drug Safety and is likely to receive an approval within the month. Eubiologics is continuing the development of its‘EuCorVac-19’ through a global Phase III trial. After receiving approval for its trial in three countries in Africa, Eubiologics also initiated a Phase III trial in the Philippines on the 7th of this month. As such, the company has entered Phase III trials by securing a comparator vaccine overseas. In Korea, the company also received approval for its Phase 3 trial protocol and is in search of comparator vaccines. ◆ Companies change trial plan due to low marketability…concerns deepen among developers Excluding the two, no other company has entered Phase III trials yet. The industry is paying attention to the possibility that more companies will stop developing COVID-19 vaccines. Cellid has started its Phase IIb trial for 'AdCLD-CoV19-1' in Korea. Cellid has prepared a two-track strategy to continue clinical trials for its vaccine for primary inoculation that was in development while developing another vaccine as a booster shot (additional inoculation). Geneone Life Science changed its strategy from developing a vaccine for primary inoculation to developing a booster vaccine. Last month, the company announced that it had completed the registration of subjects for the Phase IIa clinical trial of 'GLS-5310', which is being developed as a DNA vaccine. After confirming the safety and efficacy of clinical trials in a Phase IIa trial, the company plans to continue on studying its vaccine through Phase IIb and III trials as a booster vaccine. The changed COVID-19 situation is analyzed to have played a decisive role in the changes in the development strategy of the two companies. With the COVID-19 pandemic entering the endemic stage, the market competitiveness of vaccines has been greatly reduced. The other companies are having difficulty in the patient recruitment stage. Quratis and Eyegene received approval for their Phase I and Phase I/2a trial in July and August last year but are still recruiting patients. ST Pharm has received approval for the Phase I trial plan of its mRNA platform-based 'STP2104' in March this year and is starting to recruit patients.
- Policy
- “Dualized RWD collection for Kymriah is inefficient”
- by Lee, Tak-Sun May 11, 2022 05:55am
- With the registration of the ultra-expensive one-shot treatment Kymriah, the opinion was raised that a plan should be prepared to ensure the efficiency of the system by standardizing the dualized real clinical data (RWD) collection required by different institutions. That the separate collection of data by the Ministry of Food and Drug Safety and the Health Insurance Review and Assessment Service is a burden on the medical staff. Ji-Hye Byun, Assistant Research Fellow of the Evidence-based Research Division at HIRA claimed so in the 22nd issue of ‘HIRA’ that was recently published. Kymirah is the world’s first chimeric antigen receptor T-cell (CAR-T) therapy that was developed by Novartis Korea. The drug was approved as the first advanced biopharmaceutical under the ‘Advance Regenerative Bio Act’ in March of last year. The drug injects genetic information that can recognize specific antigens of cancer cells to the surface of an immune cell (T cell) collected from the patient and is indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Its listing had raised much attention, being a ‘one shot’ treatment that can bring an effect with a single administration and is an ultra-high-priced drug that costs 500 million won per shot. The government listed the drug for insurance benefit at 360 million won per shot in April and decided to cover the drug price with the National Health Insurance. However, the issue is that each organization requested RWD as a condition in the approval and listing process. The MFDS designated Kymirah as an advanced biopharmaceutical and ordered the company to conduct a 15-year long-term follow-up study on the drug. Also, the company needs to submit a post-management form to HIRA at the time of administration, 6 months after administration, and 12 months after administration. The global registry, EU’s EBMT will also be collecting treatment information on domestic patients, Byun said, “As a result, the HCPs will have to bear the burden of inputting and submitting RWD of similar content to three institutions - the MFDS, HIRA and EU EBMT- after patient treatment. We need to improve the domestic RWD collection system to become more efficient.” Byun explained that in Europe, the authorities are working to understand the differences in the RWD collection environment of each country and establish governance for RWD utilization among all patients. EU’s EBMT was also established as a single registry to serve the multi-purpose of post-marketing safety research, reimbursement management, and clinical research. Byun added that HIRA already owns the infrastructure to manage such information, and argued that it is necessary to use this infrastructure to separate the additional information necessary as RWD and devise a way to link it with the hospital's electronic medical record system.
- Policy
- Reblozyl was approved after 1 yr of designation of rare drug
- by Lee, Hye-Kyung May 11, 2022 05:55am
- Reblozyl 25mg, a treatment for patients with beta-Mediterranean anemia, was officially approved for items a year after designation as a rare drug. The MFDS approved Reblozyl applied by BSM Pharmaceutical Korea. Reblozyl is equipped with▲ the treatment of adult beta-Mediterranean anemia patients who need red blood cell transfusion as an indication, and ▲MDS-RS with minimum, low, and moderate risk of ring ferrous hematopoietic stem cell or low, moderate risk of ring ferrous hematopoietic and thrombocytopenia with increased myelogenesis/myeloproliferative tumor (MDS/NMP-T) Reblozyl was designated as a rare drug on April 1 last year. Beta-Mediterranean anemia, also called Cooley's anemia, is a blood disorder genetic disease that reduces the production of hemoglobin, an iron-containing protein of red blood cells, and delivers oxygen to cells in the body. Although iron treatment through red blood cell transfusion and chronic blood transfusion therapy for survival are used to treat patients, the risk of thrombosis increases to an abnormal level. Reblozyl obtained FDA marketing approval in 2019 based on clinical results of placebo administration of 112 out of 336 patients with beta-Mediterranean anemia. At the time of FDA approval, 21% of patients administered with Reblozyl had a 33% higher blood transfusion reduction rate than 4.5% of patients administered with placebo. Common side effects of Reblozyl include headache, bone pain, joint pain, fatigue, cough, abdominal pain, diarrhea, and dizziness, and high blood pressure may occur within the dose period. Thrombotic monitoring should also be accompanied when administered. It should be prohibited if a pregnant or breastfeeding woman takes Reblozyl because it can harm the fetus or newborn.
- Policy
- President Yoon said he was grateful to the people
- by Lee, Jeong-Hwan May 11, 2022 05:55am
- President Yoon Seok-yeol said on the 10th, "I am deeply grateful to the medical staff for their respect and dedication to the people who have endured great pain in the process of overcoming the COVID-19 pandemic over the past two years." At the inauguration ceremony of the 20th president held in front of the main building of the National Assembly, President Yoon said, "We will rebuild the country as a country where the people are the true owners based on liberal democracy and market economy." In Yoon's inaugural address, "freedom" said a total of 35 times. President Yoon's ambition is to ensure the value of freedom as much as possible to make it a country where the people are the true owners and fulfill their responsibilities and roles in the international community. President Yoon insisted that the universal value of freedom should be shared as something to be done to resolve excessive collective conflicts between countries and within countries and to overcome the democratic crisis. "We must properly and accurately recognize the value of freedom," Yoon said. "We must rediscover the value of freedom. Looking back on human history, the place where free political rights and free markets were breathing has always been prosperous," he said. "If any individual's freedom is violated, even the freedom of all members of the community is threatened," he said. President Yoon said, "Freedom is by no means a winner-take-all. To be a citizen with freedom , a certain level of economic fundamentals and opportunities for fair education and access to culture must be guaranteed," he said, adding that he cannot be called a free citizen without such a thing. If someone's freedom is violated or fails to meet the conditions necessary to become a free citizen, all free citizens should help in solidarity," he stressed. "Science, technology and innovation will protect our liberal democracy, expand freedom, and sustain a dignified life," President Yoon said. "We should cooperate and unite with many countries that have achieved technological progress and innovation by respecting freedom and creativity." "We are in the 10th largest economy group in the world. Therefore, we should take an attitude as a global leader in actively supporting and defending universal international norms based on freedom and human rights values," he said. He added, "I will make sure to create a country where the people are the true masters, a country that is responsible and respected in the international community based on the values of freedom, human rights, fairness and solidarity."
- Company
- Kakao is throwing the hat into the healthcare business
- by Kim, Jin-Gu May 10, 2022 06:14pm
- Large domestic companies are throwing their hat into the healthcare business. In the past year, leading conglomerates such as Lotte, Kakao, Doosan, Hyundai Heavy Industries & Construction, GS, CJ, and OCI have formalized their entry into the healthcare market. Analysts say that Samsung and SK have succeeded in the pharmaceutical bio business one after another, stimulating these companies. ◆Lotte Biologics' pre-debut notice at "BioUSA" According to the pharmaceutical industry on the 9th, Lotte Group announced that it will attend the "BioUSA" in the U.S. in June. Lotte Biologics, which recently completed its trademark application, is expected to make its debut through BioUSA. In March last year, it became the second-largest shareholder by acquiring part of the stake in Enzychem. In August last year, Lotte Group's ESG Management Innovation Office established a new growth team (biological team) and a new growth team (health care team). Lotte Biologics, which is about to be officially launched, is known to be in charge of the CMO business of biopharmaceuticals. Lee Won-jik, a former executive director of Samsung Biologics, is said to be leading the new growth team, which will be the mainstay of Lotte Biologics. ◆ GS, CJ, Doosan, Hyundai Heavy Industries, Kakao, OCI In addition to Lotte, GS, CJ, Doosan, Hyundai Heavy Industries, Kakao, and OCI have visualized their entry into the healthcare business in the past year. GS acquired Hugel in August last year. A multinational consortium led by GS Group, APHRODITE ACQUISITION HOLDINGS LLC, signed a stock transfer contract to purchase a 46.9% stake in Hugel. The acquisition amount amounts to 1.724 trillion won. ▲ GS acquired Hugel, OCI acquired Bukwang, and CJ acquired ChunLab, signaling full-fledged entry into the pharmaceutical bio business OCI acquired Bukwang Pharmaceutical in February this year. OCI became the largest shareholder as it secured an 11% stake in Chairman Kim Dong-yeon, founder of Bukwang Pharmaceutical. It is interpreted as a signal that it will enter the pharmaceutical bio business in earnest. At this year's general meeting of Bukwang Pharmaceutical's shareholders, Lee Woo-hyun, vice chairman of OCI, became co-CEO of Bukwang with the existing CEO Yoo Hee-won. CJ officially launched CJ Bioscience under CJ group in January this year. CJ Bioscience selected Microbiome as its core business. To this end, it acquired ChunLab, a microbiome company, in October last year. CEO Chun Jong-sik, who led ChunLab, will continue to represent CJ Bioscience. Kakao newly established 'Kakao Healthcare' in March. Healthcare CIC, which was previously operated as an in-house independent corporation, was separated into separate corporations. Prior to this, Hwang Hee, a professor at Seoul National University Bundang Hospital, who was recruited in December 2021, was appointed. Kakao Healthcare is expected to focus on digital healthcare businesses such as AI-based medical solution development and service industry, health care by life cycle, and smart medical care. Doosan added a medical device business to its business area through the revision of its articles of association at a shareholders' meeting held in March this year. Doosan is planning to target the biopharmaceutical container market. In December last year, it invested $100 million in SiO2, a U.S. drug storage container company. At the same time, it secured exclusive business rights in Asia and Oceania for all SiO2 products. It is planning to start manufacturing in South Korea in the future. Hyundai Heavy Industries Holdings, the holding company of Hyundai Heavy Industries Group, changed its name to HD Hyundai at a shareholders' meeting in March this year and announced plans to support four future industries, including healthcare. Hyundai Heavy Industries Holdings established 'AMC' last year and is also developing new drugs. In the pharmaceutical industry, it is interpreted that the success of Samsung and SK, which entered the field first, served as a stimulus to large companies' entry into the bio business. Samsung Biologics, SK Bioscience, and SK Biopharm, affiliates of the two groups, have become leading companies in the industry before and after the Corona crisis. Large domestic companies began the pharmaceutical bio business with enthusiasm in the past, but withdrew one after another in the mid-2010s. Lotte Pharmaceutical was launched in 2002 when it acquired a subsidiary of Ilyang Pharmaceutical's health functional food. However, in 2011, Lotte Pharmaceutical was merged into Lotte Confectionery and withdrew from the market. In 2014, CJ spun off CJ Healthcare as a separate subsidiary of the pharmaceutical division, driving the pharmaceutical bio business. However, it sold its 100% stake in CJ Healthcare to Kolmar Korea for 1.31 trillion won in 2018. Hanwha established the pharmaceutical business department in 2996 and launched Dream Pharma by absorbing and merging H-PHARM in 2004. Although it made a large-scale investment to develop a biosimilar of Enbrel, a TNF alpha inhibitor through Dream Pharma, it eventually sold Dream Pharma to Alvogen in 2014 as product development was delayed and related contracts were terminated. AmorePacific sold its subsidiary Pacific Pharmaceutical Co. to Handok in 2013 to focus on its beauty business.
- Company
- Koselugo reattempts insurance benefit in neurofibromatosis
- by Eo, Yun-Ho May 10, 2022 06:07am
- The neurofibromatosis treatment Koselugo is once again making an attempt to receive insurance benefits in an area that has no available treatment options, According to industry sources, AstraZeneca has applied for the insurance reimbursement of its new neurofibromatosis drug Koselugao (selumetinib) recently. Koselugo is indicated for ‘the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.’ After the drug received a non-reimbursement decision by the Drug Reimbursement Evaluation Committee of the Health Insurance Review and Assessment Service in March, the company has been working to promptly prepare the supplementary data to restart listing discussions for the drug. As NF1 is a rare disease area with no available treatment option, whether Koselugao will be able to receive reimbursement approval this time remains to be seen. Until now, patients had to rely on symptomatic treatment for neurofibromatosis due to the lack of an appropriate treatment option. Neurofibromatosis is a rare disease, and 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 experience Optic nerve gliomas (brain tumors) 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. Meanwhile, Koselugo was jointly developed by AstraZeneca and MSD. The drug blocks the activation of MEK to inhibit the growth of cell lines. The Phase II SPRINT study that became the basis for Koselugo’s approval showed that Koselugo reduced tumor size by over 20% in 68% of the patients that received Koselugo and achieved its primary endpoint of ORR. Also, 82% of the patients that showed a partial response had sustained responses lasting at least 12 months. Contrary to non-treated patients, half of which experience disease progression 1.5 years after diagnosis, only 15% of patients using Koselugo showed disease progression at year 3.
- Company
- Powder-type child antipyretic analgesics have been released
- by May 10, 2022 06:07am
- Johnson & Johnson Korea (CEO Baek Joon-hyuk) announced on the 9th that it has released the first powder-type children's antipyretic painkiller Children's Tylenol powder 160 mg in Korea. Children's Tylenol powder has various effects such as headache, neuralgia, muscle pain, sprain pain, menstrual pain, toothache, and joint pain, as well as fever and pain caused by a cold, so it can be widely used for fever and labor. The new product with the unique technology of Tylenol, an original Acetaminophen product, is a fever reducer of "powder formulation" that relieves the discomfort of liquid and syrup types and improves the convenience of taking pills for consumers who are unable to swallow them. As soon as the powder touches the tongue, it melts immediately in 23 seconds, so it can be taken without water, and it is easy to carry in the form of a stick pouch. It can be eaten quickly and comfortably with a small amount, so the packaging material can be used hygienically without touching the mouth. Children's Tylenol powder has both taste and effect with its special coating. The special coating of Ethyl cellulose in children's Tylenol powder prevents the bitter taste of Acetaminophen from the tongue. Wild berries and natural berries make it easy for children who had difficulty taking medicine without feeling repulsed. Parents who had to watch their children refuse the medicine for reasons such as taste, formulation, and dosage will be able to provide new options to save them the trouble of taking the medicine. The age of taking is children aged 7 to 12 years old (can be taken by elementary school students with a weight of more than 23kg), and the recommended dose is taken directly every 4-6 hours without water. It should not be taken more than 5 times a day (75 mg/kg). It is sold at pharmacies based on 12 bags per product, and if you need to divide it separately, it is recommended to fold the pouch and divide it into small quantities such as measuring cups or spoons provided at home. "Children's Tylenol Powder is the first powder-type new product in Korea that can meet needs for young children who have difficulty swallowing pills or children who are too big to eat syrup, in the form of 15mg powder per packet," said Kim Hanna, a group manager at Johnson & Johnson's self-care division. She said, "At this point when the risk of children's COVID-19 infection continues, child Tylenol powder is expected to function as a home remedy for more convenient management of fever pains for the whole family."
- Company
- Sales of generic for Galvus are not affected
- by Kim, Jin-Gu May 10, 2022 06:07am
- A reversal occurred once again in the patent dispute for the DPP-4 inhibitor-based diabetes treatment Galvus. The original signing Novartis won the remand hearing that took place after the Supreme Court's dismissal. The pharmaceutical industry believes that the decision will not have a significant impact on sales of generic for Galvus, which was released earlier this year. This is because the material patent in question expired during the dispute. According to the pharmaceutical industry on the 4th, the Korean Intellectual Property Tribunal overturned the previous ruling in a lawsuit to invalidate the extension of the duration of the material patent of Vildagliptin and decided to win Novartis. The Galvus patent dispute has been complicated by the reversal. Ahngook and Hanmi filed a trial in 2017, claiming that some of the extended duration of material patent of Galvus was invalid. In the first trial, Generic companies won. In 2019, the Patent Tribunal ruled that 187 days of the extended duration were invalid. Novartis won the second trial. In 2020, the Patent Court ruled that 55 of 187 days were invalid. The case went to the Supreme Court on Novartis' appeal. In October last year, the Supreme Court ruled against the company and sided with the company. It was a ruling that Novartis, which won the second trial, was not eligible to appeal. The case was dealt with in the first trial. In the end, the Patent Tribunal took Novartis' side. Even 55 days recognized by the patent court did not constitute invalidity. However, the pharmaceutical industry predicts that there will be no problem with the sale and prescription of generic for Galvus even with the Korean Intellectual Property Tribunal's decision. This is because the material patent expired during the dispute. The Galvus material patent expired on March 4. Hanmi Pharmaceutical, Ahngook Pharmaceutical, Angooknewpharm, Kyongbo, Korea United Pharmaceutical, Samjin, and Shin Poong then released the generic. Hanmi Pharmaceutical, which launched Vildagle as generic for Galvus in January this year, is also determined to continue marketing and sales of its products as usual. An official from Hanmi said, "The material patent of Vildagliptin has already expired on March 4, and there is no impact on the distribution, sale, and prescription of the previously released Vildagle. We plan to appeal to continue to focus the company's capabilities for the success of Vildagle."
- Product
- Unsure efficacy of Alzheimer's drug aducanumab
- by Choi-sun May 10, 2022 06:07am
- The Korean Dementia Association has reviewed the clinical potential of aducanumab (Aduhelm), the first new drug for Alzheimer's disease to be introduced to the field in 18 years. With the efficacy of the drug under constant debate, and being barely approved for its effect in reducing surrogate biomarkers such as amyloid reduction in Korea and turned down in Europe, KDA’s position is that verification over time is required to confirm the drug’s actual effect. On the 16th, KDA held its KDA Annual Fall Conference at the Baekbeomgimgu Memorial Hall. At the event, held a panel discussion session with 4 members including KDA Chair Dong-Won Yang and KDA President Ae Young Lee. In June 2021, the US FDA granted conditional approval for aducanumab as a treatment for Alzheimer’s. Aducanumab, which is a monoclonal antibody developed to target the mechanism of Alzheimer’s Disease, had become the first new drug to be approved for Alzheimer’s since 2003. As abnormal accumulation and aggregation of beta-amyloid plaques are reported in the brains of patients with Alzheimer’s, the widely-accepted theory in the field was that these protein buildups (plaques) generate neurotoxins and gradually deteriorate the cognitive function of the brain. Aducanumab was developed to remove this plaque, and although it is not a cure, its effect in was in slowing down cognitive decline in patients was recognized. But still, there is an ongoing debate on whether the drug is ‘clinically effective’ in academia, including among FDA’s internal advisors. As the drug failed to demonstrate efficacy in the clinical trial that used the change from baseline on the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) as the scale, experts judged that it is difficult to definitively acknowledge the drug’s effect only with the surrogate endpoint that demonstrated reduced amyloid accumulation. Fully aware of such controversy during its review, the FDA had set a condition upon approval that Biogen will have to conduct post-approval studies to verify the treatment’s anticipated clinical benefit in addition to the amyloid PET results. However, the EMA refused to grant marketing authorization for the drug. Through a statement, KDA had announced, “Despite much controversy, approval of aducanumab is in itself encouraging news for patients, clinical doctors and researchers in the field,” and stressed that the association will make efforts to rigorously verify the drug’s effect with science in the future to members while sharing relevant information. The panel discussion also focused on whether the amyloid plaque removal mechanism translates to real clinical efficacy. Jae Hong Lee, Professor of Neurology at Seoul Asan Medical Center, said, “As the drug wasn’t able to reach the primary efficacy endpoint of the trial, it is difficult to readily embrace the FDA's approval result. However, I believe the FDA approved the new drug despite headwinds as the promise of a cure with new drugs in development is a long way off. Lee added, “Considering the strict standards that have been applied to new drugs for Alzheimer's disease so far, the drug was difficult to be approved. To some, the CDR-SB improvement in patients who were administered the 10mg high-dose over 14 times in the post-analysis may seem far-fetched.” “Results show only 0.1% of the patients passed the final clinical review. This is an unconventional and proactive attitude on the FDA’s part when considering FDA’s traditional approval standards. In the ENGAGE trial, the drug failed to demonstrate its clinical efficacy but demonstrated a reduction in biomarkers, therefore, whether the improvement in biomarkers will bring clinical effect needs to be seen,” said Lee, warning caution over overinterpretation of the trial results. In other words, although clinical improvement can be expected due to the reduced biomarker effects demonstrated by amyloid and tau protein, Lee believes the time is needed to verify the efficacy of the drug. KDA Chair Dong-Won Yang said, “It is quite interesting that the drug continued to reduce amyloid plaques and tau in up to 1 and 2 years after high-dose administration. We need to recognize its effect in removing amyloid plaques.” Sang-Yoon Kim, Professor of Neurology at Seoul National University Bundang Hospital said, “The drug does have an amyloid reduction effect, but its clinical effect may differ by months. Also, the tau protein reduction was observed, but due to the small number of patients, we would need to wait longer to observe its actual effect," he said. KDA President Ae Young Lee limited the drug’s utility in terms of its side effects. ARIA (amyloid-related imaging abnormalities) more commonly occurred in the aducanumab administered group (43%). In addition, ARIA-E occurred in about 50% of the 7th dose and 90% of the 12th dose of aducanumab. President Lee said, “With over 40% of patients administered the high-dose experiencing ARIA, observation according to administration period and the number of doses will be needed. As the occurrence rate differs by up to 7 times in ApoE E4 carriers, we need to play caution in using the drug for treatment.” Professor Kim said, “ Patients were recruited under very strict standards for the clinical trial, to the extent that patients with other diseases were excluded from the trial. This is very different from the characteristics of patients in the actual clinical environment. As there is a possibility of side effects in case patients have heart conditions or other diseases, a guideline for its administration needs to be set.” Kim added, “Since the effect has been confirmed in a specific patient group in the trial if the drug is introduced in Korea, we would also need to strictly select a patient group in consideration of the side effects and effects in line with the clinical trial. We need to leave the decision on whether to administer the drug at the discretion of each clinician,” However, considering the current treatment environment in which there is no suitable new drug, most HCPs have shown a positive response to ‘making an attempt’ with aducanumab. The larger issue is its price, which costs tens of millions of won. Professor Lee said, “Price of aducanumab fell from the ₩65 million early on to ₩25-30 million currently. If the price becomes more reasonable, I plan to attempt administering the drug to patients who are less likely to develop ARIA and see an effect. Chair Yang said, “I believe the drug may be of help as I've seen many patients whose condition deteriorates with amyloid accumulation. It would be great if we can use the drug in the early stages of the disease, but we need to gradually increase the dose with continuous MRI monitoring due to the possibility of ARIA."
