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- 2026-04-04 04:42:01
- Company
- Is RWD needed for post-listing control of PE exempt drugs?
- by Eo, Yun-Ho Dec 02, 2024 05:49am
- The budget is tight, but the use of high-priced drugs continues to rise. The government is beginning to feel the strain of the systems it has put in place to increase access to new drugs, especially the pharmacoeconomic evaluation (PE) exemption system, which is increasingly being utilized and being subject to criticism. "The purpose of the post-listing control system is to secure additional evidence for medicines that have unclear safety and efficacy for patients. It will improve the quality of Korea’s healthcare by providing a medical reaffirmation process for such medicines." This was the government's stated intent. The government's good intentions aside, the industry has not received it well. This is due to the fear that the introduction of the system will eventually lead to "drug price cuts". Add to that the idea of using real-world evidence (RWE), or real-world data (RWD), to reevaluate drugs reimbursed through the PE exemption track, the concept has raised industry eyebrows. RWE lacks evidence base...encourages “Korea passing” The approval of a new drug is usually based on a randomized controlled trial (RCT). The PE evaluation and cost-effectiveness of the new drugs are also conducted based on this data. However, unlike RCTs, RWDs are not controlled data that are designed to accurately determine the efficacy of a drug. There are many variables that can introduce bias, such as what comorbidities the patient has, what prior medications they have taken, and whether the patient was adherent to the dose and frequency of the medication. The generally accepted levels of data evidence from high to low are meta-analyses, systematic reviews, RCTs, controlled clinical and observational studies, uncontrolled observational studies, case reports, and expert opinion; RWE is considered to be at the "uncontrolled observational study-case report" level. The Korean Research-based Pharmaceutical Industry Association (KRPIA) said, "RWE can be used to extract any desired results, depending on the intent of the collating entity. It is a risky idea to use data with a high risk of bias for the post-listing control of listed drugs. Since it is not generated in a controlled environment like an RCT, there are too many other factors to consider, including numerous crossovers, comorbidities, and use of concomitant medications." "RWEs can create contradictions and can overturn the results of RCTs that are higher level evidence. Rather, RWE should be used as a complementary tool to address uncertainties at the approval stage." Some argue that the introduction of such a post-listing control system could aggravate the "Korea passing" phenomenon in the pharmaceutical industry. If the government requires companies to submit data such as RWE post-listing, the companies will have to spend additional costs and time to generate such data, and if the data is then used as a basis for drug price cuts, it is inevitable that more pharmaceutical companies will be skeptical about launching new drugs in Korea. "Creating customized RWE data for each individual country is a huge burden for pharmaceutical companies," said a Market Access (MA) representative from a multinational pharmaceutical company. Even if a post-listing control system is introduced, the necessary data should be generated by a state-led registry, and the quality be assured." Must it be RWD? Can’t the drugs be managed with existing systems? Whether RWD is necessary is quite controversial. Korea's post-listing control system is already criticized by the industry for being tight. The Ministry of Health and Welfare proposed a plan to strengthen reimbursement management for high-priced drugs for serious diseases in 2023. In the plan, "high-priced drugs" were defined as ▲"one-shot drugs" that are expected to have long-term effects with a single treatment or drugs that require more than KRW 300 million/year of national health insurance finances per person, and were subject to reimbursement management measures. As a result, currently listed innovative drugs that have uncertainties (e.g., Kymriah, Zolgensma, Luxturna) are managed through the “performance-based reimbursement” type of the Risk-Sharing Agreement (RSA) scheme as well as the cost-effectiveness-based post-marketing evaluations, etc. The industry’s point is that systems already in place today are sufficient to manage the uncertainty that accompanies the effect of new drugs. "High-priced drugs have been reimbursed through various types of RSA, including performance-based and expenditure cap type RSA, so it is questionable whether additional follow-up measures are really necessary in addition to these arrangements, especially when the pharmaceutical companies already filter out patients that show uncertain efficacy and safety with their drugs and refund the expenses to the government under performance-based RSA," said KRPIA. "For ultra-high-priced drugs that cost over KRW 300 million per person a year and have a small number of target patients, they should be managed through a patient-level performance-based RSA or an individual contract-based drug performance evaluation system that generates individual data for each drug's situation," it added.
- Company
- oHCM drug 'Camzyos' gets greenlight for reimb
- by Eo, Yun-Ho Nov 29, 2024 05:55am
- Product photo of BMS Korea’s Camzyos 'Camzyos,' a new drug to treat obstructive hypertrophic cardiomyopathy (oHCM), will finally be listed for reimbursement. The Ministry of Health and Welfare (MOHW) held the 23rd Health Insurance Policy Review Committee meeting on November 28, announcing that BMS Korea’s Camzyos (mavacamten), a new drug used to treat obstructive hypertrophic cardiomyopathy (oHCM) will be reimbursed. As a result, prescriptions for Camzyos are expected to increase starting in next year (December). Camzyos, which was approved in May 2023, received a re-assessment status during the Drug Reimbursement Evaluation Committee (DREC) review of the Health Insurance Review and Assessment Service (HIRA). After that, it passed the DREC review and entered a drug pricing negotiation in August, but no decision was reached regarding Camzyos during the negotiation period (60 days). Camzyos is the only drug that selectively inhibits cardiac myosin-actin cross-bridge formation, which is the cause of oHCM. Camzyos' mechanism involves dissociating myosin from actin, relaxing overstimulated heart muscle, and thereby improving left ventricular outflow tract (LVOT) structure and LVOT outflow obstruction. Due to the lack of available treatments for oHCM for a long time, off-label medications have been used to manage symptoms. After Camzyos launched, the European Society of Cardiology (ESC) updated its guidelines for managing cardiomyopathy for the first time in about nine years. Previously, the guidelines for HCM were based on evidence limited to small-scale monitoring data, retrospective analysis results, and consensus opinion. However, Camzyos has completely changed this situation. Two large-scale, phase 3 clinical trials conducted as randomized controlled trial (RCT) have confirmed the significant effect of Camzyos. Consequently, ESC guidelines recommend Camzyos with the highest evidence level A for the first time in treatment options. American College of Cardiology (ACC) and the American Heart Association (AHA) are preparing to update their guidelines. Furthermore, based on this phase 3 trial evidence, the U.S. FDA granted Camzyos Breakthrough Therapy Designation (BTD) and approval. Meanwhile, the efficacy of Camzyos was demonstrated through Phase 3 EXPLORER-HCM trials. In this trial, Camzyos improved primary endpoints, which were the patient’s symptoms (NYHA classification) and exercise capacity measured with peak oxygen uptake (pVO2), more than twofold compared to the placebo. 20% of the patients treated with Caymzyos met the NYHA classification and pVO2 improvements. It also reduced the LVOT outflow obstruction index by four-fold after exercise. 7 out of 10 patients who received Camzyos treatment had improved indexes and ended up not considering surgery, and they maintained the effects for 30 weeks.
- Company
- Deregulate to foster new modalities like ADC, gene therapy
- by Son, Hyung Min Nov 29, 2024 05:55am
- Seung-Kyou Lee, Vice President of KoreaBio New modalities (therapeutic approaches) such as antibody-drug conjugates (ADCs), obesity drugs, and gene-editing technologies need dramatic regulatory relief to settle in Korea, said experts. On the 14th, KoreaBio held a seminar on trends and prospects of the bio-industry at the El Tower in Seocho-gu, Seoul. At the event, Seung-Kyou Lee, Vice President of KoreaBio, presented his assessment of the bio-industry this year and its prospects. Lee cited gene therapy drugs, obesity and diabetes drugs, and ADCs as new modalities that the Korean pharma-bio industry is interested in and developing. With the recent commercialization of gene therapy drugs such as Exa-cel, and Lyfgenia overseas, domestic companies with genetic scissors technology have also started busily developing new drugs. Genetic scissors are molecular biological tools that recognize and cut specific areas in the DNA to edit genes. Currently, domestic biotech companies such as ToolGen, Organoid Sciences, and GeneKOre have entered the market and started developing products. Lee said that the LMO Act should be revised to vitalize the domestic gene therapy market. Recently, the LMO Act was proposed to distinguish between genetically engineered organisms (GEOs) and genetically modified organisms (GMOs) to exempt them from regulations. The LMO Act aims to separate GEOs from GMOs and exempt them from the regulations that apply GMOs, ultimately to foster Korea’s GEO industry. "Gene therapy is rapidly emerging as a global industry trend. If Korea’s regulations are not eased, it will be difficult for domestic companies to grow," he said, adding, "GMO exemptions or ease of regulations for gene-edited crops/seeds is necessary." Diabetes, obesity drugs, ADCs also on the rise..."Need more government R&D support for biotechs" Treatments for obesity and diabetes have also emerged as a major R&D trend in the Korean biotech industry. The use of glucagon-like peptide (GLP-1) injectables has grown dramatically due to their ability to achieve effective weight loss. As GLP-1-based obesity drugs such as Saxenda, Wegovy, and Zepbound have shown dramatic weight loss effects in clinical trials, newcomers are mostly targeting GPL-1. Currently, various domestic pharmaceutical biotech companies such as Hanmi Pharmaceutical, Peptron, Inventage Lab, Dong-A ST, and Progen have entered the market. Lee explained, "The number of companies developing drugs for diabetes and obesity will continue to grow. However, investment sentiment in biotech companies is weak. This is why it is time to expand government R&D support for biotech companies." He pointed to "ADC" as one of the modalities that will emerge as an R&D trend this year and beyond. ADCs are anticancer drugs made by linking antibodies that bind to specific target antigens on the surface of cancer cells with drugs that have cell-killing properties. ADCs have the advantage of using the selectivity of the antibody to its target, and the killing activity of the drug that selectively acts on cancer cells, thus increasing the therapeutic effect while minimizing side effects. LigaChem Biosciences, Pinotbio, and Orum Therapeutics have succeeded in exporting ADC technology to multinational pharmaceutical companies and are being recognized for their technology. "It's time for a different level of effort to be invested compared to what we've been doing so far," said Lee, adding that securing funding is essential for biotechs to focus more on drug development. "I hope a small fund of KRW 30 billion to 50 billion can be created to be invested in the biotech sector." "We need to improve the conditions for maintaining the special listing in bio technology, such as relaxing the legal loss requirement and the threshold of KRW 3 billion in sales, and we would like to see support and incentives established for the self-reliance of Materials, Components, and Equipment businesses and stabilization of the supply chain of raw materials and pharmaceuticals."
- Company
- Samsung Bioepis names Kyung-Ah Kim as new CEO
- by Nov 29, 2024 05:55am
- Kyung-Ah Kim, President and CEO of Samsung Bioepis Samsung Bioepis has changed its CEO for the first time since its launch. Kyung-ah Kim (56), Executive Vice President and Development Division Leader, has been appointed as CEO. This is the first female CEO appointed in the Samsung Group. Hansung Ko, who has served as CEO for 12 years since the launch of Samsung Bioepis, will take over as head of Samsung Future Business Division. Samsung Bioepis today announced the promotion of Kyung-Ah Kim to the position of President and CEO. Born in 1968, Kim completed her bachelor's and master's degrees in pharmacy at Seoul National University. She then earned a doctorate in toxicology from Johns Hopkins University in the United States. Kim is from the Samsung Advanced Institute of Technology (SAIT), which marks the start of Samsung Group's bio business. She joined SAIT in 2010 as a principal scientist in bio-drug development. Then she joined Samsung Bioepis in 2015 and has played a key role in all aspects of the business, including biosimilar development, process, quality, and licensing. The company said, “As the first female professional CEO of Samsung Group, Kim will not only provide a vision for growth and an opportunity for female talents to take on bold challenges, but will also serve as a role model for women, who make up more than half of Samsung Bioscience's workforce, and is expected to bring new energy into the organization.” Hansung Ko, who was appointed CEO of Samsung Bioepis upon its foundation in 2012 and led the company for 12 years, will head Samsung Electronics' Future Business Division. The Future Business Division was established in November last year, and Mr. Koh will lead the discovery of new businesses for Samsung Group.
- Company
- 'Global obesity drug market 79 times bigger than Korea'
- by Son, Hyung Min Nov 29, 2024 05:55am
- Kang-Bok Lee, Marketing & Sales Excellence Lead at IQVIA Multinational pharmaceutical companies have been achieving significant growth through a strategy that focuses on core industries and key therapeutic areas (TA)s. On the 14th, KoreaBIO held a seminar on trends and prospects of the bio-industry at Yangjae El Tower. On this day, Kang-Bok Lee, Marketing & Sales Excellence Lead at IQVIA, shared the trends of the global pharmaceutical industry. According to Lee’s data, the global pharmaceutical market was worth USD 1.44 trillion (about KRW 191.6 trillion) last year. This is 79.1 times the size compared to Korea's market, which is USD 18.2 billion (about KRW 25.5 trillion), The growth of the global pharmaceutical market was driven by diabetes, obesity, anticancer, and autoimmune disease drugs. In particular, GLP-1 class diabetes and obesity drugs showed remarkable growth. Eli Lilly's GLP-1 diabetes drug Mounjaro generated KRW 6.48 trillion in sales last year, up 971.2% from 2022. Launched in May 2022, Mounjaro grew rapidly last year after generating KRW 640 billion in sales the same year. Novo Nordisk's GLP-1 class of diabetes drugs also had a strong year. Ozempic generated sales of KRW 5.48 trillion last year, up 52% from the previous year. Sales of Rybelsus, an oral treatment with the same ingredient generated KRW 1.44 trillion, up 140% from 2022. The obesity drug Saxenda generated KRW 1.225 trillion in sales last year, up 9.8% year-on-year. Wegovy surpassed KRW 300 billion in sales last year despite facing supply challenges. "The growth of global innovation brands is being driven by a small number of countries, products, and companies. Premium, innovative new drugs such as Keytruda and obesity treatments have driven growth in the global pharmaceutical industry." Global pharmaceutical companies steadily launch new drugs..."Expect to focus on core industries and TA" He expects an average of 70 new drugs to be launched between next year and 2028, with anticancer and obesity drugs driving growth. "Immuno-oncology drugs like Keytruda have a wide range of indications. The development of one drug has the effect of developing multiple therapies. Therefore, multinational pharmaceutical companies are showing a trend of new drug development that focuses on core industries and core therapeutic areas." "Oncology and obesity are expected to be the fastest growing segments. Oncology is expected to grow at a CAGR of 15% and obesity at a CAGR of 26% through 2028." In particular, Lee noted that companies that focus on one area, such as Mounjaro, Wegovy, SKYRizi, and Keytruda, are seeing strong growth, driving label expansions and ingredient re-engineering. "Novo Nordisk, Lilly, AbbVie, and MSD are companies that have seen success by focusing on a single therapeutic area. We believe that their products will continue to lead the global pharmaceutical market in the future,” said Lee. "Just as GLP-1 obesity drugs were developed as diabetes treatments and then became highly successful as obesity treatments, we expect re-engineering and labeling to emerge as a global R&D trend in the future."
- Policy
- Concerns about National Bio Committee's foreign pharma exec
- by Lee, Jeong-Hwan Nov 29, 2024 05:54am
- President Yoon Suk Yeol has been appointed as chair of the National Bio Committee. The National Bio Committee, chaired by President Yoon Suk Yeol, is set to launch in December. However, reports indicate that a global pharmaceutical company executive has been included as a committee member, raising concerns. The committee comprises up to 40 members, including the chair, vice-chair, various ministers, and officials from the National Security Office. One of the appointed committee members is an executive from a foreign pharmaceutical company. Given that the committee's primary role is to formulate national strategies for the pharmaceutical and biotechnology industries, critics have argued that appointing an executive from a major multinational pharmaceutical company has the potential for government policies or confidential information to be leaked externally. According to the pharmaceutical industry on November 27, the list of committee members of the National Bio Committee included an executive from a multinational pharmaceutical company. President Yoon Suk Yeol was appointed as chair of the National Bio Committee, and Lee Sang-yeop, vice president of the Korea Advanced Institute of Science and Technology (KAIST), will serve as vice-chair. Civilian committee members include 20 experts, including Kim Bit-naeri, Director of the Institute for Basic Science, Koh Han-seung, CEO of Samsung Bioepis, and Kim Young-tae, President of Seoul National University Hospital. The first meeting is scheduled for next month. An issue has arisen from including an executive from a multinational company who has worked for nearly 20 years in a global pharmaceutical company. Criticism has been raised about including an executive from a foreign-based company representing global big pharma to the National Bio Committee for the Korean pharmaceutical industry is inappropriate. The National Bio Committee is responsible for reviewing South Korea's overall biotechnology policies, including research and development (R&D) and new drug approvals, to foster the high-tech pharmaceutical and biotechnology industry as a next-generation key sector. The committee comprises up to 40 members, including the President as Chair, a vice chair, ministers from various government departments, the senior secretary for Science and Technology, the Third Deputy Director of the National Security Office, and private experts from the field and academia. Criticism have been raised about including an executive from a multinational pharmaceutical company. Critics argue that such an appointment contradicts the committee's purpose of promoting domestic pharmaceutical innovation. They state that allowing a global "Big Pharma" executive to participate in discussions about policies shaping South Korea's pharmaceutical R&D, regulatory·pricing strategies undermines the committee's intent. Concerns also extend to potential security risks, as sensitive government policies or information on the pharmaceutical and biotechnology sectors critical to national security could be inadvertently·deliberately leaked externally. "The National Bio Committee is responsible for unveiling, formulating, discussing, and deciding on South Korea's R&D strategy in the pharmaceutical and biotechnology sectors. Including a multinational pharmaceutical company executive in a committee chaired by the President Yoon, where vaccine and pharmaceutical security issues may be discussed, is illogical. It is comparable to handing over our home territory," a representative from the domestic pharmaceutical industry said.
- Company
- Sales rights for Viviant·Tuvero·Akarb to be transferred
- by Kim JiEun Nov 28, 2024 05:55am
- Due to changes in sales rights for blockbuster items that are frequently dispensed, the pharmaceutical supply and demand are expected to be affected in the end-of-year period and early 2025. According to the pharmaceutical distributor industry on November 26, several pharmaceutical companies are expected to change their distributors at the end of this year or early next year. Medication with confirmed changes to sales rights is Pfizer's Viviant Tab. Pfizer Korea has recently sent an official letter to wholesale distributors, hospitals, and pharmacies notifying 'The changes to the supply chain for Viviant.' Pfizer stated in the official letter that Pfizer will be responsible for distributing Viviant Tab 200 mg, previously distributed by Handok, starting December 1. The company requested that any inquires about returns for remaining stock from previously distributed items to be maded to Handok, and any inquiries after December 1 be directed to Pfizer. Pfizer also announced that the distribution of Caverject Injection 10 ug and 20 mg, previously distributed through Novamedix will be changed to direct distribution effective December 1. Pfizer stated any inquiries about remaining distributing stock for this item could be made to Novamedix, and inquiries related to distribution after December 1 should be directed to Pfizer. In addition to these items, more products with expected changes to sales rights in early next year. Sales rights for most of Boryoung's items will be changed, and the company seems to be informing its pharmaceutical distributors. The wholesale distributor said that Daewon Pharmaceutical has been responsible for distributing Boryoung's Tuvero Tab and Akarb Tab, but Boryung will directly distribute them starting next year. Yungjin Pharmaceutical has been distributing Boryung Buspar Tab since 2018. Boryung is highly like to distribute the drug directly next year. As changes to distributors have been announced, it is expected to affect the end-of-year pharmaceutical supply and demand. "Special circumstances such as changes to the distributor are likely to affect the supply and demand. The effective date may be postponed beyond what the pharmaceutical companies have previously announced," a pharmaceutical wholesale employee said. "We must prepare for possibilities such as difficulty in returning remaining stocks and unstable supply and demand for such items in early next year."
- Policy
- KIPO opposes to objections to new drug patent extended cap
- by Lee, Jeong-Hwan Nov 28, 2024 05:55am
- The Korean Intellectual Property Office (KIPO) The Korea Research-based Pharma Industry Association (KRPIA) and others have submitted an opinion disagreeing with the proposed legislation that would introduce a maximum cap for terms of patent protection for new drugs. The proposed legislation is intended to facilitate an earlier market launch for Korea-made new drugs. The Korean Intellectual Property Office (KIPO) stated its opposition to this issue at the 21st National Assembly. At that time, KIPO stressed that the original draft instead of a revised Patent Law that would limit the number of patents to a single (1 patent). A similar proposed legislation has been submitted to the 22nd National Assembly. Therefore, it is highly likely that KIPO would state an opinion agreeining with the original draft. On November 26, the Trade, Industry and Energy Small and Medium Venture Business Committee is scheduled to hold a small committee for legislative review. Rep. Koh Dong-jin, a member of the People Power Party, will review the draft of the revision to the Patent Law. Rep. Koh Dong-jin's proposal contains key messages, including ▲legislating a 14-year maximum (cap) on the effective duration of patent rights and ▲ limiting the number of patents eligible for extension to a single patent per drug. The proposed legislation is based on concerns that the current patent term extension system under Korean patent law differs from those in advanced countries such as the United States and Europe, potentially reverse discriminiate the domestic pharmaceutical industry. KRPIA "It worsens individual access to medications…disagreeing with the legislation" In Rep. Koh Dong-jin's draft of the revision, the Korean Research-based Pharmaceutical Industry Association (KRPIA), the Japan Pharmaceutical Manufacturers (JPMA), the Pharmaceutical Research and Manufacturers of America (PhRMA), the European Federation of Pharmaceutical Industries and Associations (EFPIA), the Biotechnology Innovation Organization (BIO), the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), and the Patent Information Initiative for Medicines (Pat-INFORMED) have shared opposing views. The opposing group argues that adopting only selective features of overseas practices regarding patent term extensions, rather than improving the entire system, could worsen public access to medicines and disrupt international collaboration. Specifically, regarding introducing a patent term cap, they highlight that South Korea recognizes only the 'domestic clinical trial period' for patent extensions, excluding the 'overseas clinical trial period' from the calculation of extension durations. They also argued that the scope of extended patent rights in South Korea is narrower compared to major countries. Specifically, there are conflicting court rulings on whether extended patent rights are limited to the indications approved at initial approval. It also remains to be seen whether the extended rights apply to all approved uses of the extended product. They also stated that there is almost no chance of appealing a rejection decision concerning patent term extension applications in South Korea. Before Rep. Koh Dong-jin's draft of the revision, KRPIA and others have previously opposed the bill proposed by Democratic Party Rep. Jung Il-young during the 21st National Assembly, which was abandoned because the legislative term expired. KIPO disagrees by citing U.S. examples During the 21st National Assembly, KIPO presented counterarguments to the objections raised against Rep. Jung Il-young's bill, advocating for the retention of the original proposal. KIPO will likely maintain the same position regarding Rep. Ko Dong-jin's proposal in the 22nd National Assembly. KIPO states that it cannot accept the opposing arguments, including the claim that South Korea does not include overseas clinical trial periods in calculating patent extension durations and the assertion that the scope of extended patent rights in South Korea is unclear compared to major countries. However, regarding the opposing argument that there is no opportunity to appeal rejection decisions on extension applications, KIPO partially accepted this concern, suggesting the need to review the introduction of a related system. KIPO disputed the claim that South Korea excludes overseas clinical trial periods and uniformly excludes the Ministry of Food and Drug Safety's (MFDS) approval supplementation period when calculating patent extension durations, stating, "Other countries do the same." KIPO explained, "The U.S. system, similar to South Korea's patent extension calculation method, includes only half of the clinical trial period and does not recognize overseas clinical trial periods." It emphasized that "maintaining the current practice for calculating extension durations is appropriate." "Considering the Supreme Court ruling, which excludes the applicant's compensation period during the MFDS's review of approval documents, we believe that the current method for calculating extension durations should remain unchanged," KIPO stated. "The length of the extension period is primarily influenced by variations in domestic clinical trial durations, which are affected by factors such as a country’s drug development capabilities and the size of its pharmaceutical market, rather than differences in extension systems." Regarding the opposition's argument that the scope of patent rights should be expanded to include the 'active ingredient' and included into patent law, KIPO countered by saying that the legislation should proceed as originally proposed. "The Supreme Court, considering the protection of patent holders and the purpose of the extension system, has ruled that the scope of extended patent rights should be determined based on the 'active ingredient and drug use based on the Patent Law,' similar to major countries," KIPO stated. KIPO partially agreed with the opposing view that there is no opportunity to contest rejection decisions on extension applications. "It is necessary to establish various concrete measures to contest rejection decisions to secure procedural rights for patent holders," KIPO stated. "However, these remedies should be introduced alongside measures to address rejection decisions related to delays in registration, which will require a more thorough review."
- Policy
- Trial for targeted drug 'VERT-002' for NSCLC begins in KOR
- by Lee, Hye-Kyung Nov 28, 2024 05:55am
- A clinical trial of 'VERT-002,' an innovative targeted treatment for degrading a c-MET gene, will begin in South Korea. C-MET gene is known as one of the cancer-inducing factors of non-small cell lung cancer (NSCLC). On November 27, the Ministry of Food and Drug Safety (MFDS) has granted approval of 'a first-in-human (FIH) multi-center, dose-escalation, dose-expansion Phase I/II clinical trial with VERT-002 for patients with locally advanced or metastatic solid tumors, including NSCLC.' This clinical trial will be conducted at the Asan Medical Center in Seoul and the Severance Hospital. The VERT-002 candidate product was developed by Vertical Bio, a Swiss-based biotechnology company specializing in developing antibodies for cancer treatment. The French pharmaceutical company Pierre Fabre Laboratories acquired Virtual Bio last year, adding a pipeline of cancer agent discovery. A degrader of MET, VERT-002 is a monoclonal antibody with a unique and differentiating mechanism of action. The company anticipates that VERT-002 will likely become a best-in-class treatment for cancer associated with MET alterations. Global clinical trials for the candidate are being conducted worldwide, including in South Korea. The Phase ½ clinical trial for a first-in-human (FIH) multi-center, dose-escalation, and dose-expansion in patients with NSCLC harboring MET alterations began in October. NSCLC is the most common form of lung cancer, accounting for about 85% of newly diagnosed lung cancer cases. Also known as the hepatocyte growth factor receptor (HGFR), MET is a factor regulating tumor genes in a patient sub-group with NSCLC. 1-4 MET exon 14 (METex14) skipping mutations and MET amplification have been identified as the major tumor gene, and MET amplification appears to be mechanisms of tolerance to selected targeted treatment. With its unique and differentiating mechanism of action, VERT-002 works by targeting clinically proven tumor-inducing factors and induces degradation of the c-MET tumor gene. Meanwhile, Pierre Fabre recorded sales of 467 million euros in 2020 just with anticancer agents.
- Policy
- Handok’s thrombocytopenia drug Doptelet approved in KOR
- by Lee, Hye-Kyung Nov 28, 2024 05:54am
- Handok's new rare disease drug ‘Doptelet (avatrombopag)' was approved in Korea on the 26th. The Ministry of Food and Drug Safety approved Doptelet as a treatment for thrombocytopenia for patients with liver disease. Handok is reaping the benefits of forging a strategic partnership with the global biopharmaceutical company Sobi-Handok last year. At that time, the company decided to introduce two new drugs, Empaveli (pegcetacoplan) and Doptelet, a rare disease treatment, into the country, both of which have now been approved in Korea. Doptelet is an orally administered second-generation thrombopoietin receptor agonist (TPO-RA) approved in the U.S. for the treatment of thrombocytopenia in adult patients with liver disease. Patients with moderate-to-severe thrombocytopenia are at increased risk for serious or fatal bleeding events. Patients with thrombocytopenia are commonly known to receive platelet transfusions to increase their platelet count prior to surgery. The approval of Doptelet was based on safety and efficacy data from two Phase III clinical trials on 435 patients. Doptelet is the first oral treatment option for patients with chronic liver disease which offers the advantage of eliminating the need for platelet transfusions prior to surgery for many patients. Meanwhile, Handok formed a joint venture with Sobi in April this year, following a strategic partnership last year. Sobi-Handok will be responsible for developing and selling Sobi’s rare disease drugs in Korea, with a 49:51 shareholding ratio between Handok and Sovi, respectively.
