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- 2026-05-04 16:26:36
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- Leukemia drug Xospata’s reimb limit may be resolved
- by Eo, Yun-Ho Nov 23, 2023 05:37am
- Astellas Korea is working to resolve the issue of the reimbursed number of cycles approved for its acute myeloid leukemia treatment ‘Xospata.’ Dailpharm’s coverage showed that the agenda on expanding reimbursement standards for Astellas Korea’s Xospata, a drug for patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, has recently passed the Health Insurance Review and Assessment Service’s Cancer Reimbursement Evaluation Committee review. The company applied for the reimbursement extension in November, and the progress has been made 6 months after the agenda passed the Cancer Disease Deliberation Committee in May. However, since Xospata waived the pharmacoeconomic evaluation process, the drug would also have to undergo drug pricing negotiations with the National Health Insurance Service for its reimbursement extensions. Therefore, whether its reimbursement can be extended to cover more cycles will depend on when the agenda is submitted to the Drug Reimbursement Evaluation Committee for review and whether it passes the review. The drug is indicated as monotherapy for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation (FLT3mut+). However, only patients who are eligible for allogeneic hematopoietic stem cell transplantation can receive reimbursement, for up to 4 cycles. Other than the financial issues, there are no specific reasons to limit the number of administration cycles for Xospata. In the ADMIRAL trial, Xospata was used without limiting the treatment period, and the NCCN guidelines also issued a ‘Category 1’ recommendation for the drug without restricting its treatment period. The current best option to cure AML patients is hematopoietic stem cell transplantation, but this is accompanied by a high risk of recurrence, and transplantation is not an option for the large number of elderly AML patients that exist. Therefore, there is no suitable treatment alternative other than Xospata available for patients who cannot undergo hematopoietic stem cell transplantations, and these patients are still using the chemotherapy that was developed over 40 years ago due to ineligibility for reimbursement of Xospata. Xospata targets both types of FLT3 mutations, FLT3-ITD and FLT3-TKD, and may be self-administered at home as a single oral tablet once daily without frequent hospital visits. Also, Xostapa has demonstrated improved safety and efficacy compared with existing chemotherapy.
- Company
- Reimb of COVID-19 drugs necessary with eased crisis level
- by Eo, Yun-Ho Nov 22, 2023 05:30am
- Whether COVID-19 treatments will be able to be listed for reimbursement before Korea's COVID-19 containment measures are lifted remains under attention. Two COVID-19 treatments, Gilead Science Korea’s ‘Veklury (remdesivir)’ and Pfizer Korea’s ‘Paxlovid (nirmatrelvir+ritonavir),’ are currently under review by the Health Insurance Review and Assessment Service after the companies filed applications for their reimbursement. The government had previously announced that it would work to reimburse COVID-19 treatments in H2 2024 as part of its COVID-19 risk level management roadmap. This means that the COVID-19 treatments that are being supplied free of charge will be converted and become purchased products like general medicine. When Phase 2 of the COVID-19 risk level management roadmap, which will further ease the disease control and prevention system, is implemented next month, COVID-19 will then be regarded as a Grade 4 infectious disease like influenza. Not dangerous because it’s an endemic?... Improving awareness remains a key issue However, the road to reimbursement for these treatments is not easy. Not only are the prices expensive, but the sharp decline in the public’s sense of crisis is also playing a significant role. COVID-19 has shifted from a pandemic into an endemic, but it is still producing variants that threaten high-risk patients such as those with underlying diseases and the elderly. The 'Excess mortality during COVID-19’ submitted by the Korea Disease Control and Prevention Agency in October to the office of Democratic Party of Korea Rep. Choun Sook Jung, a member of the National Assembly Health and Welfare Committee, that cites data from Statistics Korea, the number of excess deaths continued to be on the rise until recently due to the aftermath of COVID-19. According to the report, ‘excess deaths’ from COVID-19 had exceeded 65,000. ‘Excess deaths’ refer to additional deaths beyond the number of deaths normally expected to occur at a particular period. This figure indicates how many more deaths occurred compared to when COVID-19 had not occurred. Eun-Joo Choo, Professor of Infectious Diseases at Soonchunhyang University Bucheon Hospital, said, “COVID-19 is still a disease that requires active treatment and management, and many patients are still being hospitalized for the disease. Its treatments need to be listed with reimbursement immediately upon the closure of the free support system next year to ensure treatment access for the patients and to secure essential drugs.” Both drugs are necessary...due to different roles Although both were approved as COVID-19 treatments, Veklury and Paxlovid differ in terms of indication, and formulation, among others. Veklury has been verified to have slowed down disease progression in many COVID-19 patients, reducing the time to recovery and thereby reducing the burden on Korea’s medical system. It was approved to treat COVID-19 in adults and pediatric patients with mild-to-moderate COVID-19 who are at high risk of progressing to severe disease. It can be administered regardless of the severity of the patient’s disease and aids in quicker recovery by offering a lower risk of disease progression with earlier treatment. Also, it is a treatment whose efficacy and safety have been proven through several clinical trials and RWE and is the only COVID-19 treatment that can be used on patients with severe COVID-19. In particular, it has little drug-drug interactions (DDI), and comes in an injectable formulation that can be administered to patients who have difficulty taking oral medications. Paxlovid, which comes in an easy-to-take oral formulation, is approved to treat mild-to-moderate COVID-19 in adults who are at risk of progressing to severe COVID-19, which includes hospitalization and death. Paxlovid needs to be taken within five days of developing symptoms and is administered twice daily for 5 days. In the EPIC-HR trial that was conducted on non-hospitalized symptomatic adult subjects with COVID-19 who are at high risk of progressing to severe disease and have no prior history of vaccination, Paxlovid significantly reduced the proportion of people with COVID-19-related hospitalization or death from any cause through 28 days of follow-up by 86% compared to placebo among patients treated within 5 days of symptom onset and who did not receive COVID-19 therapeutic monoclonal antibody treatment.
- Company
- Mitsubishi’s Lou Gehrig's disease drug Radicut's sale soars
- by Nho, Byung Chul Nov 21, 2023 05:48am
- The latecomer injection formulations are taking over the Lou Gehrig's disease treatment market that had been occupied by injectables in the past. According to the industry’s pharmaceutical distribution performance data, Mitsubishi Tanabe Pharma Korea's Radicut Inj (edaravone) has occupied 39% of the market share in the related treatment market 8 years after receiving marketing authorization, narrowing the gap with YooYoung Pharm's Yooritek (riluzole, 41%). Radicut, which was approved in 2015 by the Ministry of Food and Drug Safety, had posted sales of KRW 500 million, KRW 1.3 billion, KRW 1.1 billion, KRW 1.6 billion, KRW 2.2 billion, and KRW 0.9 billion in H1 of 2018, 2019, 2020, 2021, 2022, and 2023, respectively. It has outperformed Sanofi Aventis’s Rilutek since 2020. During the same period, Sanofi Aventis’s sales of Rilutek were KRW 1.2 billion, KRW 2.6 billion, KRW 0.1 billion, KRW 0.3 billion, KRW 0.7 billion, and KRW 0.4 billion, respectively. After posting record-high sales of KRW 2.6 billion in 2019, Rilutek recorded sales worth KRW 0.4 billion in H1 this year, drawing a downward-sloping performance curve. Until earlier this year, Yooyoung Pharm was the only domestic company to supply Lou Gehrig's disease treatment. Therefore the fact that it had recorded record-breaking performance of KRW 2.4 billion in 2020 and 2021 with Yooritek against global pharmaceutical companies Sanofi and Mitsubishi is one aspect to note. Clockwise from the top: Yooyoung Pharm’s Yooritek, Mitsubishi Tanabe Pharma Korea’s Radicut, and Sanofi’s Rilutek Radicut is indicated to slow down the progression of functional impairment in patients with amyotrophic lateral sclerosis (ALS). Rilutek and Yooritek are indicated to extend life or the time to mechanical ventilation for patients with amyotrophic lateral sclerosis (ALS). Lou Gehrig's disease is a rare condition that cannot be cured until now. Its annual drug cost is around KRW 3.3 million, which is relatively cheap compared to the ultra-expensive rare and anticancer drugs that cost hundreds of millions of won. Another interesting aspect is that Radicut, which contains edaravone, was initially prescribed as a treatment for cerebral infarction, and then added an indication for Lou Gehrig's disease. Radicut’s efficacy and effect in suppressing the progression of functional impairment have been confirmed through clinical trials conducted on Lou Gehrig patients in Japan. Lou Gehrig's disease is a representative rare and intractable neurological disease that selectively degenerates only motor neurons, causing irreversible paralysis of limbs and respiratory muscles. Most patients affected with Lou Gehrig's disease die within 2 to 5 years after onset without mechanical ventilation. It is estimated that there are approximately 350,000 patients worldwide and 2,000 to 3,000 in Korea. It occurs in approximately 1 to 2 people per 100,000 per year, and its incidence increases from the late 50s. Its incidence rate is 1.4 to 2.5 times higher in men than in women. Meanwhile, SK Chemicals also succeeded in listing Teglutik (200ml), a suspension formulation containing riluzole, that it imported from the Italian pharmaceutical company Italfarmaco at an insurance price of KRW 134,970 earlier this year. The ceiling insurance price is KRW 133,970 per vial for a 15-day use. Therefore, the price is the same as the price of existing tablet formulations (KRW 4,499 per day) in terms of daily dosage. As it comes in a suspension formulation, it is administered directly using oral syringes, and through percutaneous endoscopic gastrostomy (PEG) tubes for those who have difficulty taking it directly. Patients with Lou Gehrig's disease often experience dysphagia (difficulty swallowing) an early symptom due to weakened tongue and throat muscles. Although tablets are considered more convenient for intake take than injections in general, for Lou Gehrig's injections are more convenient than tablets for the reason above. This is why Mitsubishi Tanabe Pharma Korea’s injectable Radicut is rapidly expanding its market share. Therefore, patients with dysphagia had difficulty taking the table formulation of riluzole, which was administered twice a day.
- Company
- IL inhibitor reimb as 1st-line Tx for ankylosing spondylitis
- by Eo, Yun-Ho Nov 21, 2023 05:48am
- Interleukin inhibitors may soon be available as first-line treatment for ankylosing spondylitis in Korea. On the 20th, the Ministry of Health and Welfare announced an amendment notice that contains a plan to expand insurance reimbursement for the 2 IL-17A inhibitors approved in Korea - Novartis Korea's 'Cosentyx (secukinumab)' and Lilly Korea's 'Taltz (ixekizumab)’ – as treatments for ankylosing spondylitis. With the notification, the reimbursement standard extension for the two drugs, which had remained pending for over a year, is expected to be implemented within the year. Novartis and Lilly both submitted an application for reimbursement extension for their respective drugs based on the ‘ASAS-EULAR axSpA treatment recommendations 2022’ in July last year. Although the actual process for their reimbursement extension was completed earlier this year, time was wasted due to a delay in final approval on the Ministry of Health and Welfare’s part. Interleukin inhibitors are available with reimbursement as a first-line treatment for ankylosing spondylitis in over 30 countries. In Korea, both drugs are currently only approved for use with reimbursement in the second-line treatment setting, after patients are prescribed TNF-α inhibitors. The grade of recommendation and level of evidence for IL-17A inhibitors were raised to Grade A and 1a in the 2022 update of the ASAS-EULAR recommendations, to the same level as TNF-α inhibitors. The guidelines referred to how both agents are currently being used as first-line biological agents to treat ankylosing spondylitis and officially confirmed their equal therapeutic positions as treatments for ankylosing spondylitis. Moreover, the use of TNF-α inhibitors is associated with tuberculosis. So patients had to undergo prior therapy to treat latent tuberculosis before using TNF-α inhibitors. However, despite the anti-TB treatment, the occurrence of tuberculosis due to the use of TNF-α inhibitors is still reported in several studies. Therefore, another treatment option was dire for patients who are at high risk of infection such as tuberculosis, or who have comorbidities, such as heart failure, and have complications in being prescribed TNF-α inhibitors. The number of ankylosing spondylitis patients has rapidly increased by nearly 50% over the past 10 years (35,592 in 2013, 52,616 in 2022). It is a disease that mainly occurs at a young age, and domestic patients go through about 40 months of wait until they receive an accurate diagnosis. In addition, patients can receive a disability level up to Level 2 at the highest, rendering it a serious disease and eligible for exemption from military service.
- Company
- Yuhan Leclaza clinical phase 3 cost exceeds 100 billion
- by Chon, Seung-Hyun Nov 20, 2023 05:50am
- The development cost invested by Yuhan's new anti-cancer drug Leclaza in the Phase 3 clinical trial has exceeded 100 billion won. Three years after the start of the clinical trial, the cost of development is also increasing. Yuhan was approved as a primary treatment based on the efficacy and safety of Leclaza confirmed in the clinical trial, and the salary expansion was also in the countdown. According to the Financial Supervisory Service on the 16th, as of the end of the third quarter, the total development cost accounting for as intangible assets by Yuhan Corporation was 119 billion won. It increased by 3.9 billion won in three months from 1151 billion won at the end of the first half of the year. In 2019, the Financial Supervisory Service set a standard that accounting assets can only be processed if there is a technical feasibility for R&D tasks such as new drugs. The Financial Supervisory Service proposed the asset-enabled phase of R&D costs, with phase 3 clinical trials for new drugs and phase 1 clinical approval for biosimilars. Generics can be assetized after the biometric test plan is approved. The intangible assets reflected by Yuhan in its development expenses are the largest proportion of Lexha. At the end of the third quarter, Leclaza development cost of intangible assets was 101.1 billion won. It means that more than 100 billion won has been invested in aleclaza clinical phase 3 expenses. Leclaza is a treatment for non-small cell lung cancer that was approved as the 31st new drug developed in Korea in January 2021. Patients with locallyprogressive or metaplastic non-small cell lung cancer who are resistant to T790M after administration of the first and 2nd generation epithelial growth factor (EGFR) tyrosinase suppressant (TKI) are subject to administration. It interferes with the transmission of signals involved in the growth of lung cancer cells and acts as a mechanism to inhibit the proliferation and growth of lung cancer cells. Leclaza recognized 32.6 billion won in development costs as intangible assets for the first time in the fourth quarter of 2020. As the clinical phase 3 trial started in earnest, the development cost was reflected as an intangible asset. Leclaza development cost of intangible assets increased to 61.4 billion won at the end of 2021 and rose to 88 billion won, an additional 26.6 billion won last year. This year, the development cost of Leclaza’s intangible assets increased by a total of 13.3 billion won. 5 billion won and 4.7 billion won in clinical phase 3 expenses were invested in the first and second quarters, respectively. In the third quarter, 3.4 billion won was added, exceeding 100 billion won for the first time. Leclaza is challenging the primary treatment health insurance benefits. In June, Lexar received additional permission as a primary treatment, two years and five months after receiving domestic approval. Reclaza identified statistically significant progression-free survival (PFS) improvements in a multi-country clinical phase 3 trial. A Phase III trial (LASER301) conducted on 393 active EGFR mutant-positive topical advanced or metaplastic lung cancers that had never been treated demonstrated superior safety and effectiveness over conventional therapies. The clinical results were unveiled at the Asian General Conference of the European Society of Oncology in Singapore last year. Yuhan has recently applied to health authorities to apply Leclaza primary treatment. Yuhan plans to provide patients with free drugs for clinical trials for primary therapeutic purposes through the Expanded Access Program (EAP). The price of Leclaza’s monthly medicine is about 6 million won. According to IQVIA, a pharmaceutical research institute, Leclaza’s sales in the first half of last year were 10.3 billion won, up 49.5% from the same period last year. Sales in the first quarter were 5.1 billion won, up 57.4% from the previous year, and in the second quarter, it recorded 5.2 billion won, up 42.5% from the previous year. Cumulative sales since the release of Leclaza have been 30.2 billion won. Cumulative sales exceeded 30 billion won within two years of domestic launch.
- Company
- ‘1+3 will also impact Korea’s patent linkage system'
- by Kim, Jin-Gu Nov 20, 2023 05:50am
- The so-called ‘1+3 restriction on bioequivalence testing’ system is expected to impact the Drug Approval-Patent Linkage System as well. If applied, the 1+3 system is expected to eradicate the practice of multiple generic companies jointly filing patent challenges and improve the practicality of the first generic exclusivity right (first generic exclusivity). Also, the ‘Bill for the Amendment to the Drug Patent Term Extension System’ that is pending at the National Assembly is expected to have a positive effect on generic companies that challenge the original drug’s patent. However, there is also a possibility that this bill will not be passed due to the nearing end of the 21st National Assembly session. "1+3 restriction will improve the effect of first generic exclusivity system… but be disadvantageous for generic companies" Myung Hee Lee, Senior Researcher at the Korea Institute of Intellectual Property presented the prospects mentioned above at the ‘2023 Medical Intellectual Property Policy Forum’ that was held at the Korea Intellectual Property Service Center on the 16th. During her presentation, Lee pointed to 2 changes that will significantly affect the patent linkage system in the future. One is the Bioequivalence Testing 1+3 Restriction System, and the other is the Drug Patent Term Extension System.’ The 1+3 system limits the participating generic makers to 3 consignees per consignor when conducting joint biological equivalence tests or clinical tests. This means that the number of times the clinical data owned by the pharmaceutical company that directly conducted the trial can be shared is limited to 3. Regarding this, Lee predicted that there would be a change in the existing practice of multiple pharmaceutical companies challenging one patent at the same time. At the same time, it was predicted that the effectiveness of the generic exclusivity system would improve. Lee added, “We are currently analyzing the impact of the implementation of the systems. Because the system was not applied to existing applications at the time, the impact of the new system will begin to be felt in earnest from 2022.” “In the past, many companies challenged patents at the same time. In this process, the criticism was that the original purpose of the first generic exclusivity system had faded somewhat. With more than 20 pharmaceutical companies receiving exclusivity at the same time, the significance of the ‘exclusivity’ offered by the system had faded somewhat.” Lee added, “Simultaneous filing of patent challenges will also decrease. We are also observing situations where consignees who have not been granted data from the consignor companies that have directly conducted the bioequivalence tests are unable to join in the early release of generics. This will lead to many small pharmaceutical companies giving up development of generic drugs.” In the discussion session that followed the presentation, In-Bum Kim, an expert consultant at Kimg & Chang added, “From the generic companies’ part, it is now time to seriously consider selecting and focusing on a specific subject. If the companies had all challenged a single patent together, it is now the time for them to each select and pursue a field they can do well at.” "Patent term extension system is beneficial for generic companies…but will it be able to pass NA remains a question" On the other hand, the amendment to the drug patent term extension system was expected to have a positive effect on generic companies. The amendment, which is currently pending at the National Assembly, was presented to limit cases of excessive patent term extensions by original companies. The drug patent term extension system extends the patent term of original companies by the time the company spent on clinical trials or approval, by up to 5 years. However, criticism arose that the system rather works in favor of the original companies in Korea than in the US and Europe. The amendment limits the 'effective patent term’ to 14 years from the time of application for term extension to its expiration, and allows the company to only apply for term extension of one patent among the many that are registered for a single drug. Lee said, “The amendment will be detrimental to original companies and beneficial to generic companies. It is expected to serve in favor of generic companies on the other side of the 1+3 system.” However, whether it will pass the National Assembly review remains in question. To be enacted, the bill must be passed within this year’s session ahead of the general election in April next year. The panels who participated in the discussion predicted that the possibility of passing the amendment in the 21st National Assembly was low. Professor Cha-ho Jung from Sungkyunkwan University Law School who chaired the panel discussions, said, “If it was a bill with no objections, it might have passed; but due to strong opposition from original companies, it will not be easy for the bill to pass NA. I believe the chances are low."
- Company
- 160 billion won Daewoong·Takeda Luphere·Leuplin
- by Nho, Byung Chul Nov 17, 2023 06:00am
- Daewoong Pharmaceutical's Luphere and Takeda Pharmaceutical Leuplin are leading the market in the field of prostate cancer treatment. According to the drug distribution performance data, Luphere and Leuplin ranked 1st and 2nd based on single product sales in the first half of this year, with a performance of 15.4 billion won and 13.9 billion won, respectively. In the last five years, these two products have been fiercely competing for a leading position. In 2019-2020, Leuplin surpassed Luphere, which recorded 21.5 billion and 22.4 billion won at 32.2 billion and 315 billion won, and in 2021 and 2022, Luphere surpassed Leuplin, which achieved 242 billion and 27.5 billion won with 30.7 billion and 30.4 billion won. Ipsen Korea's Diphereline, which ranked third, recorded 22.7 billion, 24.8 billion won, and 13.7 billion won in 2021 and 2022. The noteworthy point is that in the first half of 2023, the combined performance of Digital herelin PR/SR is 162 billion won, which is more than the performance of Luphere and Leuplin. Diphereline is growing from 200 million in 2019 to 4.5 billion won in 2022, and Diperellin pier is also expanding its appearance from 8.9 billion in 2019 to 248 billion won in 2022. In the first half of this year, Hanol Eligard, AZ Zoladex Depo, Zoladex LA depo, and Lorelin Depo recorded sales of about '9.5 billion', '8.8 billion won, and '7 billion' respectively. In the past three years, the combined sales of Zoladex Depot and Zoladex LA depot have been at the top with 26.4 billion won, 30.7 billion won, and 331 billion won. Ferring Pharmaceutical Permagon, which remains at the bottom of the ranking, showed a slight increase in sales compared to last year, with a performance of 130 million won in the first half of this year. The reason for the low sales is identified as the acquisition of a single indication compared to competing products. Indications for Luphere and leuplin, include prostate cancer, endometriosis, premenopausal breast cancer, central puberty, and reduction of myoma. Zoladex has the most various efficacy effects, such as prostate cancer, endometriosis, premenopausal breast cancer, and assisted reproduction for pituitary inhibition and adjuvant therapy for early breast cancer in the process of ovulation. 7.5 mg and 22.5 mg of leuprorelinacetate is used to relieve advanced prostate cancer, and 45 mg is used for central puberty. The market for prostate cancer and precociousness treatments has grown from 118.9 billion won in 2019 to 1607 billion won in 2022, an increase of 35% over three years.
- Company
- New drug Camzyos can be prescribed at hospitals
- by Eo, Yun-Ho Nov 17, 2023 06:00am
- The new drug Camzyos for obstructive hypersatic myocardial infarction is entering the general hospital prescription. According to the related industry, Camzyos of BMS Pharmaceutical Korea has passed the Drug Committee (DC) of medical institutions such as SMC and Sinchon Severance Hospital. Camzyos is the first and only treatment that selectively inhibits the excessive cross-bining of cardiac myosine and acttin, the cause of obstructive hypertrophic cardiomyopathy (oHCM). The drug has the mechanism of separating myosine from actin and relaxing the excessively contracted heart muscle, improving occlusion with an inflated left ventricle structure and left ventricle leaks. The clinical trial based on the Camzyos permit is the EXPLORER-HCM study. In the trial, Camzyos improved more than twice as much as the patient's symptoms (NYHA grade) and motor performance (maximal oxygen intake, pVO2), which is a primary evaluation variable. Of these, 20% of the Camzyos dosing group achieved both NYHA ratings and pVO2 improvements. After exercise, the left ventricle also reduced the occlusion index more than fourfold. Seven out of 10 people treated wit Camzyos had improved their indicators to such an extent that they did not consider surgery, maintaining a consistent effect for 30 weeks. oHCM is a rare heart disease in which the left Ventil muscle of the heart is abnormally thickened and blocks blood flow through the entire body through the aorta. Symptoms of oHCM vary, including shortness of breath, dizziness, chest pain, which can increase the risk of various cardiovascular complications such as heart failure and atrial fibrillation. Currently, oHCM treatment focuses on symptom relief and management rather than underlying treatment, so there was a high demand for unfulfilled treatment. Drug treatment options such as beta blockers and calcium channel blockers could reduce heart rate and myocardial contractility, but there were limitations that existing drug treatment options alone made it difficult to expect long-term improvement. Professor Lee Sang-cheol of the Department of Circulation at Samsung Seoul Hospital said, "OHCM is a rare disease that can cause sudden heart death without warning, and there has been many difficulties in treatment because there is no effective way to treat it non-invasively. Camzyos expects to be able to restore patients' quality of life by taking oral doses once a day, with excellent symptom-improving effects from the early days of treatment."
- Company
- GC Biopharma’s Hunterase approved after 11yrs
- by Chon, Seung-Hyun Nov 17, 2023 05:59am
- GC Biopharma’s rare disease treatment ‘Hunterase’ was granted official marketing authorization 11 years after receiving conditional approval. On the 16th, GC Biopharma announced that it had completed the Phase III trial for its Hunterase on the 14th and changed its license from conditional approval to final marketing authorization. Hunterase, which was approved in 2012 in Korea, is the world’s second treatment for developed Hunter syndrome, which is also known as ‘Mucopolysaccharidosis type II (MPS II),’ is a rare disease that is known to occur in roughly 1 in 100,000 to 150,000 male live births. As a congenital, hereditary metabolic disorder, Hunter syndrome shows various unpredictable symptoms such as skeletal abnormalities decreased intelligence, and even premature death around the age of 15 in severe cases. Less than 100 patients with Hunter syndrome exist in Korea. Hunterase had received conditional approval under the condition of conducting a Phase III trial in January 2012 GC Biopharma received approval for a Phase III trial in November 2016 and conducted a clinical trial to evaluate Hunterase’s efficacy and safety in patients with Hunter syndrome who have not previously received enzyme replacement therapy. The company completed its Phase III clinical trial results report in March of this year and submitted the Phase III clinical trial results to the Ministry of Food and Drug Safety in May. Afterward, the company completed a Good Clinical Practice (GCP) inspection and received the nod for the final change in its license. In the Phase III trial, patients who received Hunterase intravenously every week for 52 weeks showed statistically significant superior results compared with the past placebo control group. Due to the nature of the rare disease, it took a longer time to agree on a Phase III trial design, including patient recruitment. In the end, it took 6 years to prepare the Phase III clinical trial results report. However, the company explained that it met the required conditions 3+ years earlier than in the plans it discussed with the MFDS. A GC Biopharma official said, “Hunter syndrome is a serious and life-threatening rare disease, and based on the fact that it is realistically difficult to conduct confirmatory clinical trials, we have supplied it to patients under conditional approval. So just the fact that we completed the Phase III trial and met the conditions we were required to meet for its formal approval has significant meaning on its own.
- Company
- Will Keytruda’s receive reimb for TNBC in KOR?
- by Eo, Yun-Ho Nov 17, 2023 05:59am
- Whether MSD Korea will be able to make progress in extending reimbursement for its cancer immunotherapy Keytruda for the 13 additional indications it had applied for is receiving attention. According to industry sources, 3 indications of MSD Korea’s PD-1 inhibitor Keytruda (pembrolizumab) are expected to be deliberated by the Health Insurance Review and Assessment Service’s Cancer Disease Deliberation Committee on the 22nd. Although the company applied for reimbursement of its 13 indications in bulk, the specific indications are expected to be deliberated separately. In October, its 3 applications for esophageal cancer, endometrial, and colorectal cancer were presented for deliberation by CDDC but received a ‘rediscussion’ decision. In the case of triple-negative breast cancer, although a new drug has received marketing authorization for the disease, no reimbursed option currently exists for TNBC as of now. Gilead Science Korea’s TNBC treatment ‘Trodelvy (sacituzumab govitecan)’ is also expected to be deliberated at the same meeting. Keytruda’s efficacy in TNBC was confirmed through 2 clinical trials. In KEYNOTE-522, a Phase III trial conducted on previously untreated patients with Stage II or III TNBC patients to evaluate the benefit of using Keytruda as neoadjuvant and adjuvant therapy, its use showed a significant 13.6% absolute increase in complete pathologic response compared with the control group. Event-free survival (EFS) rate was 84.5%, reducing the risk of disease progression and death by 37%. In KEYNOTE-355, Keytruda improved PFS regardless of the type of chemotherapy. The chemotherapy+ Keytruda arm in the study showed a PFS of 9.7 months, which was a 4.1-month improvement compared to the placebo arm, demonstrating statistical significance. MSD applied for 13 indications for Keytruda in June: ▲ early-stage triple-negative breast cancer; ▲locally recurrent or metastatic triple-negative breast cancer, ▲metastatic or with unresectable, recurrent head and neck squamous cell carcinoma, ▲ locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) carcinoma, ▲adjuvant treatment of patients with renal cell carcinoma, ▲non-muscle invasive bladder cancer,▲persistent, recurrent, or metastatic cervical cancer,▲ advanced endometrial carcinoma, ▲advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) ▲ unresectable or metastatic MSI-H or dMMR colorectal cancer ▲metastatic MSI-H or dMMR small bowel cancer, ▲ metastatic MSI-H or dMMR ovarian cancer, and ▲ metastatic MSI-H or dMMR pancreatic cancer
