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- 2026-03-14 11:09:48
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- Luxturna, a one-shot retinal disease treatment
- by Eo, Yun-Ho Apr 11, 2023 06:11am
- Luxturna, a one-shot retinal disease treatment, is once again aiming to enter insurance coverage. As a result of the coverage, Novartis Korea recently resubmitted a reimbursement application for Luxturna, a treatment for Inherited Retinal Dystrophy. This is a quick resumption of the process after the HIRA's non-reimbursed decision last month. As the company's will to be listed on the salary is firm, it remains to be seen whether this re-challenge will be successful. This drug submitted an application for reimbursement in September 2021, but there was no progress in the listing process so far, and it was first introduced this year. Although it is expensive one-shot gene therapy, it seemed difficult to register because the disease is not directly related to life. Luxturna restores the function of the defective or defective RPE65 gene, one of the causes of IRD, by replacing it with a normal gene with just one administration. This means that the fundamental treatment of the disease is possible. Therefore, the key is how much Luxturna can achieve the value of preventing blindness. This drug was designated by the US FDA as Breakthrough Therapy in 2014, Orphan Drug in 2016, and Priority Review in 2017, and obtained expedited approval in 2017. IRD is a rare and intractable disease in which vision loss occurs due to mutations in the gene responsible for the structure and function of retinal photoreceptors. It includes more than 20 different eye diseases, and there are about 300 causative genes. IRD caused by RPE65 gene mutation causes an abnormality in the visual cycle in the retina, which converts visual information entering the eye into nerve signals and transmits them to the brain. RPE65 gene mutation reduces the RPE65 protein, which is essential for visual circuitry, and destroys retinal cells, gradually narrowing the field of vision and eventually leading to blindness. Luxturna proved its effectiveness through a phase 3 clinical trial targeting patients with hereditary retinal diseases in which a biallelic mutation in the RPE65 gene was confirmed. As a clinical result, the functional vision of patients treated with Luxturna improved statistically significantly compared to the control group who did not receive treatment at 1 year of treatment. As a result of evaluating the average score of the Multi-Luminance Mobility Test (MLMT), which evaluates the ability to pass through an obstacle course of various heights in various levels of illumination by recreating a daily walking environment, as the primary evaluation variable at 1 year of treatment, Luxturna treatment group The score change of was 1.8 points, which was 1.6 points higher than the control group's score change of 0.2 points.
- Company
- One-shot CAR-T tx, approved for domestic items
- by Eo, Yun-Ho Apr 11, 2023 06:11am
- According to related industries, Janssen Korea's Kavicty was approved last month, and Novartis Korea's Kymriah obtained additional approval for indications on the 5th. The second domestically approved CAR-T new drug, Kavicty, is an anticancer drug that inserts genetic information to recognize BCMA into the patient's immune cells (T cells) and then injects these T cells into the patient's body. B-cell maturation antigen, which is selectively expressed during plasma cell differentiation and not expressed in other major organs, represents an ideal target for plasma cell cancer (multiple myeloma). This drug is indicated for patients with relapsed or refractory multiple myeloma who have received at least four prior therapies, including ▲proteasome inhibitors ▲immunomodulators, and ▲anti-CD38 antibodies. In the case of Kymriah, the indication was expanded to treat adult patients with recurrent or follicular lymphoma after two or more treatments. With this expansion of the indication, Kymriah ▲relapses or secondary relapses after transplantation and subsequent relapses or refractory B-cell ALL (Acute lymphoblastic leukemia) and ▲ in pediatric and young adult patients under the age of 25 A third indication was obtained following recurrent or diffuse large B-cell lymphoma after two or more systemic treatments. Kymriah's new indication was based on ELARA, a phase 2 clinical trial targeting adult patients with relapsed or refractory follicular lymphoma (n=97). As a result of the study, the Overall Response Rate was 86.2%, including 69.1% of Complete Remission. Meanwhile, in March 2021, it was approved as a domestic advanced regenerative medicine bio law No. 1 treatment, and in April 2022, insurance benefits are applied to the two previously approved indications.
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- Roche and Korea BIO offers opportunity to domestic companies
- by Hwang, Jin-joon Apr 11, 2023 06:08am
- KoreaBIO and Roche are hosting an open innovation event(Pic: KoreaBIO) The Korea Biotechnology Industry Organization will be providing opportunities for technology transfer and overseas expansion of domestic biopharmaceutical companies. For this purpose, Korea Bio plans to first cooperate with the multinational pharmaceutical company Roche. According to industry sources on the 10th, Korea BIO is planning to hold a ‘Korea BIO x Roche Partnering’ open innovation event on the 9th of next month. The event will be held on and offline at Roche Korea’s head office in Gangnam, Seoul, and be attended by groups wishing to partner with Roche. The event will offer one-on-one meetings between global pharmaceutical companies and domestic biopharmaceutical companies. Korea BIO planned the event to provide opportunities for domestic pharmaceutical and biohealth companies to receive a diagnosis on their commercialization potential for technology transfer and overseas expansion, etc. Domestic pharmaceutical and biohealth companies wishing to engage in technology transfer and joint research with Roche can submit an application, and prepare nonconfidential company introduction materials, personal information consent form, and company introduction material consent form to Korea Bio by the 24th of this month. The company introduction materials should only include only publicly available data. When the domestic pharmaceutical and biohealth companies submit the required documents, Roche Asia Partnering will select companies after review. Select companies will have on/offline meeting opportunities with Roche, and receive feedback related to the contents discussed. Then, a select few that wish to partner with Roche will receive the opportunity to have face-to-face meetings at Roche headquarters, global branches, and subsidiary research institutes. As collaboration areas, Roche suggested immunology, oncology, neurological disorders, research and development technology, personalized digital healthcare, rare diseases, and infectious diseases. If approved, the collaboration will be carried out with its Genentech Research Center, Roche Innovation Center Basel, Roche’s Shanghai Innovation Center, Global Product Development Department, and Product Strategy Department, etc. The industry believes that Korea BIO's open innovation project will be an opportunity for domestic companies to receive feedback from multinational pharmaceutical companies on their direction of pipeline research, further required research, and the marketability of their candidate substances. Starting with Bayer in 2021, Korea BIO had held open innovation partnering events with Boehringer Ingelheim, Novartis, French Healthcare Club, and CJ in the same year.
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- Two types of RET-targeted anti-cancer drugs for minority pts
- by Eo, Yun-Ho Apr 10, 2023 05:52am
- It does not seem easy to enter the insurance coverage of RET-targeted anti-cancer drugs targeting a very small number of patients. According to related industries, Retevmo of Lilly Korea failed to pass the HIRA held on the 6th, and Roche Korea's Gavreto submitted an application for benefits last year, but it has not yet been presented to the Cancer Disease Review Committee, and discussions are slow. However, in the case of Retevmo, as the decision to re-discuss was made, there is room for hope. Although the application for Gavreto was delayed compared to Retevmo, there is no news about it, despite the strong tendency of the government to evaluate drugs with the same mechanism. Retevmo and Gavreto both obtained FDA approval in March. Retevmo was a bit early for global first permission. Retevmo obtained marketing approval from the US FDA in May last year and Gavreto in September last year. Retevmo was approved for two indications of non-small cell lung cancer and thyroid cancer, and for indications of Gavreto, thyroid cancer was added in December of the same year after approval as a lung cancer treatment. Since these drugs suppress not only the primary mutation of the RET gene but also the secondary mutation that causes resistance to chemotherapy, it was expected that they would be able to solve the unmet demand in various types of cancer, but they are experiencing difficulties in the insurance coverage process. Cho Byung-cheol, a professor at the Lung Cancer Center at Yonsei Cancer Center, said, "Until now, there were no chemotherapy options for patients with RET gene mutation cancer, so we had no choice but to undergo chemotherapy in the same way as general cancer patients. It can be an alternative for patients who have had difficulties with relatively low anti-cancer toxicity.” Meanwhile, in the LIBRETTO-001 clinical trial, Retevmo recorded a response rate of 64% in non-small cell lung cancer patients who had previously received platinum chemotherapy and an 85% response rate in the newly diagnosed group. Gavreto significantly reduced tumors by 58.8% in non-small cell lung cancer patients who had previously received chemotherapy in the Arrow trial. In patients diagnosed for the first time, the response rate was 72%.
- Company
- It is difficult to secure domestic funding for bio ventures
- by Hwang, Jin-joon Apr 10, 2023 05:51am
- Head of KDDF, Muk Hyun-sang, is presenting major business plans for this year. (Photo: Dailypharm)"It is not easy to change KDDF's funding method. It is possible to raise funds only when investors flock to it, but in order to attract funds from the United States, we are planning an event to invite major US VCs to invest in Korean bio ventures in July. are doing." Muk Hyun-sang, head of KDDF, made this announcement at a press conference held on the 5th at the Korea Chamber of Commerce and Industry's mid-sized conference room in Jung-gu, Seoul to mark the 2nd anniversary of KDDF's launch. Director Muk Hyun-sang explained, "I thought KDDF funds were priming water," and explained, "If you support about 10% of the necessary R&D costs, the company has raised 15 billion won by itself, but it is currently difficult." "It is difficult to increase the amount of money that can be supported by KDDF," he said. "We are looking for a way to get funds from the outside. The US VC invitation event plan is one of the ways to connect financing." KDDF plans to invest a total project cost of 2.1758 trillion won by 2030. The business goals are 60 global technology transfers worth 20 billion won or more, 35 cases worth 100 billion won or more, 4 new drug approvals from the FDA and the EMA, and the creation of one global blockbuster new drug with annual sales of 1 trillion won or more. Director Muk said, “The business carried out by KDDF cannot be seen as a general state-sponsored new drug development project.” there is, and if a company is short of funds, KDDF should create an opportunity to raise funds." KDDF is also planning to carry out 'ADCaptain' to support the ADC project, which is considered a next-generation treatment this year. KDDF plans to prioritize pipelines targeting pancreatic cancer, ovarian cancer, and triple-negative breast cancer among solid cancers and strengthen support through the ADCaptain project. It plans to provide R&D subsidy through investment without equity dilution and support up to 20 million dollars by 2025. From this year to next year, it plans to establish a joint operating committee through cooperation between participating companies and investment companies in the form of a virtual company. A science development advisory committee will also be operated. KDDF plans to select three tasks as leading material development tasks and support 800 million won each for two years. When it enters the development stage after 2024, it plans to establish a corporation and distribute shares. The CEO will be nominated by the company in charge of clinical development. The chairman of the board of directors is appointed by KDDF. R&D funds will be raised through KDDF R&D subsidy, global VC investment, and government fund investment. The required investment is expected to be 24 million dollars. Director Muk said, "It has become KDDF's mission to raise funds. We submitted a budget plan related to new drug development worth 250 billion won to the Abu Dhabi Investment Authority through the Ministry of Strategy and Finance, and we will contact Singapore's Temasek."
- Company
- GemVax’s Alzheimer’s Tx GV1001 receives IND approval
- by Kim, Jung-Ju Apr 07, 2023 05:55am
- On the 4th, GemVax & KAEL announced that it had received investigational new drug (IND) approval for the Phase II trial of its Alzheimer’s treatment candidate, GV1001, in 7 European countries as planned. With its procedures for clinical trial approvals in the 7 European countries complete, the company plans to accelerate the clinical trials in Europe, starting with the initiation of the clinical trial in Spain. A GemVax official said, “With approval complete in 7 European countries, we expect the clinical trials we initiated in Europe to speed up. The global clinical trial of our Alzheimer's disease treatment GV1001 is progressing smoothly in the U.S. and Europe, therefore, we will do our best to achieve significant clinical results." The company plans to enroll a total of 185 patients, 77 from the US and 108 from Europe in the global clinical trial for GV1001. The company explained that the first patient was registered in the US last October and that a total of six patients have been registered so far. The clinical trial will evaluate the efficacy and safety of GV1001 (0.56 mg and 1.12 mg) subcutaneously as a treatment for mild-to-moderate Alzheimer's disease (AD) for 53 weeks (12 months). In addition, GemVax has also received approval from Korea’s Ministry of Food and Drug Safety to conduct a clinical trial on GV1001 as a treatment for progressive supranuclear palsy (PSP). This is expected to become the first clinical trial conducted in Korea for PSP. As GV1001 is being studied for PSP in addition to the Alzheimer's disease clinical trials that are being actively conducted in Korea and abroad, GV1001’s scope is expected to expand further to cover a range of neurodegenerative diseases.
- Company
- The dilemma of price cuts for Lipiodol and Fattiodol
- by Nho, Byung Chul Apr 07, 2023 05:55am
- There are many opinions of concern that the two items of X-ray contrast medium registered as essential medicines may fall into a situation where supply is not possible. According to the industry, price cuts are scheduled for Lipiodol and Fattiodol on the 1st of next month, and the price of raw materials for these products is high. The original product, Guerbet Fattiodol Ultra, obtained domestic approval in 1998 and was recognized for its insurance price of 52,560 won in 2016. Afterward, the pharmaceutical company filed an application for drug price adjustment with the health authorities in 2018, citing lower margins compared to cost, and was found to have led to a drug price increase of 190,000 won, a 261% increase from the previous one. Lipiodol is 189,224 won in January 2022, 133,000 won in September 2022, 189,224 won in September 2022, and 133,000 won in January 2023. The drug price is scheduled to be reduced to 1,745 won. The situation is equally bad for Dongkuk Fattiodol, the only generic drug in the market approved in 2020. At the time of registration, the additional drug price was recognized and 59.5% (113,050 won) of 190,000 won was recognized, but the drug price is scheduled to be reduced to 101,745 won on the 1st of next month. Application for drug price adjustment is virtually the only way to raise or preserve drug prices, but it cannot be used because there are alternative drugs between Lipiodol and Fattiodol. If two pharmaceutical companies simultaneously stop supplying drugs due to lower margins following drug price cuts, a supply-demand crisis is expected. An industry insider said, "Estimating the cost of essential medicines and resolving the dynamics of drug price cuts is a mid-to-long-term task that the health authorities must solve." The main component of Lipiodol is Ethyl Esters Of The Iodised Fatty Acids Of Poppyseed Oil, which is used for lymphography, salivary gland imaging, carotid artery chemoembolization for liver cancer, and hysterosalpingography. Last year, Lipiodol and Fattiodol last year's performance were about 2.8 billion won and 36 million won, respectively.
- Company
- Rise of new drugs... subject of NA and public petitions
- by Eo, Yun-Ho Apr 07, 2023 05:54am
- Specific product names of new drugs are being mentioned at the National Assembly audit and public petitions calling for the application of reimbursement for specific drugs have gathered 50,000 consents. Such activities, which would not have happened 5 years ago, are actually arising in Korea. The emergence of government affairs (GA) and patient advocacy (PA) managers are not irrelevant to the rise of such new drug issues and are more fundamentally related to the industry’s entry into an era of high-priced new drugs. ◆Awakening of the patients and rising public interest=" Please doctor, you are our only hope. Please save me.’ Times have changed. Patients and their families that used to cling to their doctors and plead for help now independently search papers and find new drugs in the clinical trials database, Clinicaltrial.gov. If a drug approved in Korea is stuck in the reimbursement process, civil complaint emails and calls pour into the webpages and offices of relevant ministries including the National Health Insurance Service and the Ministry of Health and Welfare. The same goes for the pharmaceutical companies that supply the new drugs. Some have described that the complaints have been "hindering work to the point of paralysis." The magnitude of the anger in the patients and their families looking at ‘existent but unreachable drugs’ are not so different from before. However, the current generation, which consists of a larger proportion of highly educated people, has ‘administrative power.’ The rising voice has reached the National Assembly. The NA questioned and criticized the Ministry of Health and Welfare and its affiliated agencies (HIRA, NHIS) in their audit. Professional terms related to reimbursement, such as the pharmacoeconomic evaluation exemption system, ICER value, and Cancer Disease Review Committee, are also commonly mentioned. Pressure from the National Assembly and patients, not from pharmaceutical companies, is imposing an incomparable weight on the health authorities. It will also inevitably affect the reimbursement listing process. The PA and GA managers at pharmaceutical companies focus on handling issues arising from these situations. This is why the government often accuses pharmaceutical companies of triggering this phenomenon. In a way, it is a reasonable doubt. When sharing the same interest, patients can work as the best weapon for pharmaceutical companies. Also, the accusations are not 100% false. Some companies use patient groups to raise public opinion and criticize the government. In addition, there are companies that drop the introduction of some drugs due to lack of marketability in Korea despite public criticism, and companies that deliberately delay drug price negotiations to be included in the government’s coverage enhancement plan. They all do exist. However, patients are now a 'double-edged sword' for the companies as well. Patients are moving faster than the listing plan set by the companies after approval, and the reimbursement standards the company sets through discussion with the authorities arouse impatience and anger amongst ineligible patients. The PA and GA meet with the National Assembly and patients to explain, clarify issues, and discuss support plans. Due to the highly sensitive nature of the work, education is essential to avoid crossing the line. One thing clear is that the position was created due to the prevailing trend, even more so than for the company’s pursuit of profit. A PA official at a multinational pharmaceutical company said, "Even support programs for non-reimbursed treatments are also required to abide by the Fair Trade Act. Although people may suspect that we have alternative purposes, I believe everyone will feel the same way when they meet actual patients and their families. There are times I clash with the company due to such effect.” The official added, “Among the issues patient organizations always raise, there are many opinions that the government (especially the MOHW) should prepare an official communication channel (with dedicated personnel and departments) for patients and implement a system that regularly and continuously holds an ear out to the patients' voices. It is now the time for both the government and the pharmaceutical industry to accept the patients’ request." ◆Drug become better and more expensive, system’s scope narrows=Patients that were incurable in the past are now allowed to live their life with the benefit of a drug and even dream of a cure. Advanced cutting-edge new drugs we encounter now offer surprising efficacy and safety. In addition to one-shot CAR-T therapies, approvals for new drugs with various mechanisms of action such as targeted anticancer drugs, cancer immunotherapies, and ADCs, as well as new drugs applied to specific targets or all-comers are lining up their applications. Korea’s reimbursement system has also evolved. Institutional devices for the introduction of high-priced new drugs, such as the Risk Sharing Agreement (RSA) scheme and pharmacoeconomic evaluation exemption system have been prepared, and many drugs have been listed with reimbursement thanks to such systems. Nonetheless, the current system seems too narrow to accommodate these rising new drugs. Although the companies applied for reimbursement listing, more and more drugs remain in the discussion stage for 1 to even 3 years at most. Also, the rejection rate in the CDDC stage, which is an essential gateway to reimbursement for anticancer drugs, has peaked. A study found that 76% of respondents in the industry pointed to the expansion of the RSA system and the separation of the refund-type RSA as ways to improve the current insurance reimbursement system, and 16% answered that the reimbursement listing system needed fundamental improvement. In other words, an increasing number of pharmaceutical companies are looking to change the reimbursement system to list their new drugs, rather than finding a way within the system. GAs play a pivotal role in this context. If the MA appeals to the MOHW, NHIS, and HIRA on the need for a drug, GAs play a broader role in drawing a public picture. This is also the reason why an increasing number of GAs in the industry are former NA aides. However, internal and external conflicts still remain. Internally, MAs, who are typically pharmaceutical industry experts, have a good understanding of drugs and the drug pricing system, but GAs are often not from the pharmaceutical industry. In other words, the perception that 'GAs don't know the industry well' exists in compnaies. One manager explained that internal disputes arise between the MA that speaks on behalf of HIRA, and the GA that speaks on behalf of the NA. A GA manager from a multinational company said, "I feel most upset when people mistakenly believe that I’m not working because I have to work outside the office to meet various people. Pharmaceutical companies also follow the trend and communicate better internally. Only pharmaceutical companies that can create synergy with various internal departments can succeed in leveraging GA. Integrate the internal message first to progress further."
- Company
- LG Chem releases DPP-4+SGLT-2 combo Zemidapa Tab
- by Kim, Jin-Gu Apr 06, 2023 05:53am
- On the 4th, LG Chem announced it will be releasing its new antidiabetic combination drug ‘Zemidapa Tab’ on the 8th. Zemidapa is a fixed-dose combination of the DPP-4 inhibitor class market leader Zemiglo (gemigliptin) and SGLT-2 inhibitor class market leader dapagliflozin (Product name: Forxiga). Zemidapa is the only option that offers a combination of the said two ingredients. According to the amendment made to the reimbursement of antidiabetic combination therapies that are applied from this month, Zemiglo will be allowed to be additionally prescribed to patients taking metformin and SGLT-2 inhibitors whose glucose level is not properly controlled. Zemidapa will be allowed to be prescribed with reimbursement starting on the 1st of next month. LG Chem conducted a Phase III trial on 748 patients whose blood sugar is not adequately controlled with existing two-drug combination therapy. The company had invested over KRW 20 billion in the clinical trial. Results showed that the three-drug Zemiglo+metformin+dapagliflozin combination has a greater blood glucose level improvement effect than the two-drug combination (metformin+dapagliflozin or Zemiglo+metformin). In-Cheol Hwang, Head of LG Chem’s Chronic Disease Business Unit, said, "The combination of the DPP-4 inhibitor that stimulates insulin production and the SGLT-2 inhibitor that excretes glucose from the body, have a complementary therapeutic effect. By securing large-scale clinical evidence in domestic patients, and building on the unique competitiveness of Zemidapa of being the only combination of the two ingredients, we plan to continue to lead the KRW 1 trillion domestic diabetes market.” In line with the release of Zemidapa, LG Chem plans to hold symposiums to inform about the clinical efficacy of Zemidapa for healthcare professionals in the nation. After launching Zemiglo, a new diabetes drug developed by LG Chem in 2012, the company has additionally launched the combinations Zemimet and Zemiro based on Zemiglo. According to UBIST, a market research institute, prescriptions for the Zemiglo product line last year recorded KRW 133 billion.
- Company
- NeuroBo, a subsidiary of Dong-A ST, applies for NASH candida
- by Apr 06, 2023 05:53am
- A researcher at Dong-A ST is testing a candidate substance. (Photo by Dong-A ST)Dong-A ST announced on the 4th that its US subsidiary NeuroBo Pharmaceuticals has applied to the US Food and Drug Administration (FDA) for a phase 2 clinical trial plan for NASH treatment candidate DA-1241. This clinical trial will be conducted for 16 weeks on 87 patients with NASH. It is a multicenter, randomized, double-blind, placebo-controlled, parallel-comparison clinical trial to confirm the efficacy and safety of DA-1241. NeuroBo plans to initiate phase 2 clinical trials for DA-1241 in the US within the third quarter of this year. The target study end date is the second half of 2024. DA-1241 is a first-in-class mechanism of action on GPR119. The possibility of developing it as a NASH treatment was confirmed in preclinical trials. After administration of DA-1241, improvement effects such as liver cirrhosis, inflammation, fibrosis, lipid metabolism, and glucose control were observed. NASH is a disease in which triglycerides accumulate in liver cells regardless of alcohol intake. It is characterized by inflammation and fibrosis in the liver. If the symptoms are severe, they can cause liver diseases such as liver cirrhosis, liver cancer, and liver failure. Its prevalence is 2-4% worldwide and 3-5% in the United States, but it is a disease with high unmet medical demand because there is no treatment yet. NeuroBo is a NASDAQ-listed company located in Boston, USA, and is responsible for the global development and commercialization of DA-1241 and DA-1726. DA-1726 is being developed as a treatment for obesity and NASH. It is a candidate substance that induces weight loss by simultaneously acting on the GLP-1 receptor and the glucagon receptor to suppress appetite, promote insulin secretion, and increase peripheral basal metabolism. In addition to the weight loss effect, the possibility of developing a NASH treatment was confirmed through preclinical studies. NeuroBo plans to apply for a phase 1 clinical trial of DA-1726.
