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- 2025-12-22 17:07:02
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- No new news on reimb extension of Lorviqua for 4mths
- by Eo, Yun-Ho Oct 07, 2024 05:48am
- No progress has been made on the discussions on the ALK inhibitor anticancer drug ‘Lorviqua,’ which the company had reapplied for first-line reimbursement benefits immediately upon failing to successfully conclude its first drug pricing negotiations. According to industry sources, the health authorities have not decided at what stage they will initiate the process for Pfizer Korea’s ALK-positive NSCLC drug Lorviqua (lorlatinib), for which the company has now terminated the risk-sharing agreement (RSA) and applied to list it through the general reimbursement listing process. It has already been more than 4 months since Pfizer submitted the application for the drug’s general reimbursement listing in June, shortly after negotiations with the National Health Insurance Service broke down over the drug's price. At the time, the NHIS said that the drugmaker had expressed its intention to switch Lorviqua’s reimbursement listing status to general listing, which was listed through the pharmacoeconomic evaluations exemption system as an expenditure cap type RSA, but that the switching cannot be discussed as the company’s application falls under extending its reimbursed scope of use. As a result, the negotiations broke down. However, despite the company’s prompt reapplication thereafter, this delay in the simple initiation of the process itself has left patients waiting without reservation. The issue is in the regulatory process. Currently, RSA drugs can apply for reevaluation upon the expiry of their RSA term, or start their price-volume agreement negotiations from the Health Insurance Review and Assessment Service’s Drug Reimbursement Evaluation Committee’s review stage. However, for RSA drug’s reimbursement extensions, no such streamlined track is available. Moreover, since Lorviqua was originally contracted as an expenditure-cap type RSA drug through the pharmacoeconomic evaluation exemption track but is seeking reimbursement through the general listing track, the government having more difficulty shaping the direction. The problem is that the patients are left to wait without promise. Regardless of whether the drug’s reimbursement will be extended or not, the government's flexible administration and the will of the pharmaceutical companies would be needed to achieve results. Yool-Seo Cho, Director of the Korean Lung Cancer Patients' Association, who leads a community of more than 500 ALK-positive patients and caregivers, said, “It's frustrating for patients to have to wait and watch as Korea's reimbursement standards fail to keep up with treatment guidelines. In ALK-positive NSCLC, access to treatment is a matter of life and death for the patients.” Cho added, “We request the government to actively work to improve patient access to treatment, including the swift review of the reimbursement extension for lorlatinib so that patients can receive the right treatment with less mental suffering and financial burden." Lorviqua was specifically designed and developed by Pfizer to penetrate the blood-brain barrier (BBB). The drug’s high clinical value as a first-line treatment was recognized in the 5-year long-term follow-up results of the CROWN study that was presented at ASCO. Results showed that Lorviqua reduced the risk of disease progression or death by 81% compared to crizotinib, with 60% of patients surviving without disease progression at 5 years. The risk of brain metastasis progression was reduced in 94% of patients, with only 4 of 114 Lorviqua-treated patients without brain metastases developing brain metastases.
- Company
- Camzyos’s reimbursement decision imminent
- by Moon, sung-ho Oct 04, 2024 04:38am
- Obstructive hypertrophic cardiomyopathy (oHCM), has been regarded as a difficult-to-treat area due to the lack of a cure. However, with the recent introduction of new drugs and reimbursement granted for its diagnostic test, expectations are rising for a paradigm shift in oHCM treatment – the key to which will be whether or not the newly introduced treatment will be reimbursed. According to the industry sources on the 28th, the National Health Insurance Service and BMS Pharmaceutical Korea have been negotiating the drug price of the oHCM treatment Camzyos (mavacamten) since August. oHCM is a rare and potentially fatal heart disease in which the thickened left ventricular muscle blocks blood flow to the rest of the body, causing symptoms ranging from shortness of breath to heart failure, syncope, and sudden cardiac death. Camzyos was approved by the Korean Ministry of Food and Drug Safety in May 2023 for the treatment of oHCM as a treatment that selectively inhibits the excessive cross-linking of cardiac myosin and actin, which is responsible for the development of oHCM. Specifically, the drug is inidicated to improve exercise function and symptoms in patients with symptomatic (NYHA class II-III, mild and moderate) oHCM. After years of symptomatic management with off-label agents due to the lack of a cure, the introduction of Camzyos has been regarded as a paradigm shift in its treatment landscape. Last year, the European Society of Cardiology (ESC) updated its guidelines for the first time in 9 years, recommending Camzyos the highest evidence level A for the first time among treatment options. ‘Until now, the treatment of oHCM has focused on symptom relief and prevention of complications, rather than addressing the underlying cause,’ said Professor Hyung Kwan Kim, Department of Cardiology at Seoul National University Hospital. “As oHCM is a disease where many complications can occur at a young age and leave sequelae, we expect the approval of the new drug to provide a better treatment environment for patients.’ The problem is that despite the availability of the new drug, patient access to such is still low due to so-called financial toxicity and the high burden of drug cost. Furthermore, the recent reduced patient burden for oHCM’s diagnostic test, which is leading to an increase in the number of patients, further highlights the reimbursement hurdle. As of 2023, the total number of oHCM patients registered in Korea is about 20,000. However, the actual number is expected to be higher given the prevalence of oHCM. It is generally believed that oHCM occurs in 1 in 200 to 500 people in the general population, and if this prevalence rate is applied to the entire Korean population, it is estimated that approximately 100,000 to 250,000 people in Korea have oHCM. Fortunately, the diagnosis rate of oHCM in Korea is on the rise as echocardiography, which is used to diagnose hypertrophic cardiomyopathy, has been covered for people with known or suspected heart disease since 2021. As a result, the diagnosis rate of patients with oHCM, which was Camzyos’s treatment target, has also increased with the reimbursement extension of echocardiography in 2021. In 2021, 466 patients were diagnosed with oHCM, about 1.6 times more than the 291 patients diagnosed in 2020, the year the reimbursement was extended. In other words, the reimbursement of the diagnostic test has increased the number of diagnosed patients, but the therapeutic reimbursement ‘hurdle’ is limiting treatment on site. From the patient’s perspective, they are left to wait and see if the final stage of the reimbursement discussion - the ‘drug price negotiation’ - will conclude successfully. However, there has been no news regarding the conclusion of the drug price negotiation since it passed the NHIS Drug Reimbursement Evaluation Committee in July after being put on hold once. This means that the NHIS and pharmaceutical companies have yet to reach an agreement on the total expected claims amount and the level of financial sharing. “The drug passed DREC review in July, but its drug price negotiations started in August,” said a pharmaceutical industry official who requested anonymity. “Considering the 60-day drug price negotiation period, it is likely that the negotiation period will end in early October. Since it was not listed in October, the drug is expected to be applied for reimbursement at the end of this year at this timeline.”
- Company
- Expectations rise for the reimbursement of Phesgo in Korea
- by Whang, byung-woo Oct 04, 2024 04:38am
- Professor Matteo Lambertini On the 30th, Roche Korea announced that the socio-economic benefits of Phesgo (pertuzumab/trastuzumab) for early-stage and metastatic HER2-positive breast cancer were highlighted at the 2024 Korean Society of Medical Oncology (KSMO) Congress. The 2024 Korean Society of Medical Oncology (KSMO) Fall Meeting, which was held at COEX, brings together domestic and international experts from approximately 45 countries to share the latest research and innovative approaches for treating cancer. At a symposium held under the theme of Phesgo on the 26th, Kyong-Hwa Park, Professor of Medical Oncology at the Korea University Anam Hospital, chaired the session, with Professor Matteo Lambertini, Chair of the ESMO Young Oncologists Committee, participating as a speaker. Professor Lambertini emphasized the treatment effect of Phesgo through his presentation, ‘Patient Centricity with PHESGO: Achieving a Win-Win Strategy.’ The idea is that Phesgo, as a subcutaneous injection, can improve the efficiency of treatment, allowing patients with early and metastatic HER2-positive breast cancer to maintain an improved quality of life in the long term. “Phesgo is a treatment that can effectively reduce treatment and monitoring time for patients, in addition to the superior therapeutic efficacy of the trastuzumab-pertuzumab combination, which was confirmed to be non-inferior to existing intravenous agents in Phase III FeDeriCa study, with a comparable safety profile,” said Professor Lambertini. The idea is that the use of Phesgo can reduce treatment time, which will bring socioeconomic benefits for patients, hospitals, and healthcare providers, as well as improve treatment efficiency. In fact, as a maintenance therapy, Phesgo can be administered in as little as 20 minutes, reducing treatment time for dosing and observation by up to 90% compared to a total of 270 minutes required for conventional intravenous infusions. In addition, it was designated as the first biobetter for cancer in Korea in 2021, demonstrating reduced dosing time and improved patient convenience. Phesgo is a single subcutaneous injection formulation that contains trastuzumab and pertuzumab, which were previously administered intravenously and separately, The combination of trastuzumab and pertuzumab is currently the standard of care for a variety of early-stage HER2-positive breast cancers. Professor Lambertini said, “‘In fact, a study of the socioeconomic impact of Phesgo in Italy showed that switching to subcutaneous Phesgo could reduce healthcare professional involvement time per patient by 25% compared to existing intravenous formulations.” Meanwhile, the National Comprehensive Cancer Network (NCCN) guidelines recommend the combination of trastuzumab and pertuzumab as a Category 1 recommendation for the first-line treatment of HER2-positive metastatic breast cancer.
- Company
- Biologics seek to expand indications…is approved for COPD
- by Son, Hyung Min Oct 04, 2024 04:38am
- Biological agents are expanding their reach beyond inflammatory diseases such as atopic dermatitis, oesophagitis, and asthma to lung disease. Recently, Sanofi and Regeneron's biologic Dupixent was approved by the US Food and Drug Administration (FDA) for the treatment of chronic obstructive pulmonary disease (COPD). Other biologics, such as GSK's Nucala and AstraZeneca's Fasenra, have also shown progress in treating COPD in clinical trials and are looking to expand their indications. Biologics are manufactured from pathogenic microorganisms, such as proteins, antibodies, nucleotides, and cells. They have specific effects on specific proteins or cells, which enables them to treat diseases, and are characterized by low side effects compared to chemical drugs. Dupixent secures first COPD indication among biologics Sanofi and Regeneron According to industry sources on the 3rd, the FDA approved Dupixent for the treatment of COPD. This is the first COPD indication secured by a biologic. Dupixent is the first biologic to target the signaling of interleukin (IL)-4 and IL-13, which are key drivers of type 2 inflammation. It has been shown to be effective in inflammatory conditions such as asthma, atopic dermatitis, and eosinophilic esophagitis. The expansion of Dupixent’s indication to COPD was based on the Phase III BOREAS and NOTUS trials. The trials included 1,874 COPD patients. Patients were randomly assigned to receive Dupixent or placebo every 2 weeks in addition to triple therapy with an inhaled corticosteroid (ICS), long-acting beta-agonist (LABA), and long-acting muscarinic antagonist (LAMA) or dual LABA and LAMA dual therapy. Results showed that Dupixent reduced the annualized rate of moderate or severe COPD exacerbations by up to 34% compared to placebo over 52 weeks, which was the primary endpoint. In addition to Dupixent, Sanofi, and Regeneron are developing the IL-33 inhibitor itepekimab for COPD. The companies are currently conducting 2 clinical studies on itepekimab and expect to present new data next year. Biologics continue to look to expand COPD indications GSKIn addition to Dupixent, other biologics are also looking to expand their indications to COPD. GSK recently announced positive clinical trial results for its IL-5 inhibiting biologic, Nucala. The trial was conducted in 806 COPD patients aged 40 years and older without asthma who required triple therapy with an ICS, LABA, or LAMA to determine the efficacy of Nucala in moderate/severe exacerbations. Results showed that Nucala reduced the primary endpoint of moderate/severe annualized exacerbation rate. Patients treated with Nucala for up to 104 weeks demonstrated a statistically and clinically significant reduction in the annualized exacerbation rate compared to placebo. AstraZeneca is investigating the biologic Fasenra for several diseases, including COPD, chronic rhinosinusitis, and hypereosinophilic syndrome (HES). Fasera is a biologic that binds directly to the IL-5 receptor alpha on eosinophils, leading to rapid depletion of blood and tissue eosinophils. AstraZeneca However, Fasenra did not demonstrate an effect in COPD in 2 clinical studies - GALATHEA and TERRANOVA, which were published in 2019. AstraZeneca is conducting a third Phase III study on Fasenra in patients with high eosinophil levels, as it has confirmed the drug’s benefit in this population. The ongoing Phase III RESOLUTE trial, which is studying Fasenra in more than 600 COPD patients, is expected to be completed by next June. AstraZeneca is also seeking to expand the indication for Tezspire, a biologic the company had co-developed with Amgen, to COPD as well. Tezspire is an anti-thymic stromal lymphopoietin (TSLP) monoclonal antibody therapy that binds to TSLP, which causes airway inflammation. Other biologics inhibit IL-5, lgE, and others, and Tezspire is the first to target this mechanism. Currently, Tezspire is only approved for the treatment of severe asthma.
- Company
- "Importance of fungal infections TX amid COVID-19 spike"
- by Son, Hyung Min Oct 04, 2024 04:38am
- Sung-Yeon Cho, a professor of at the Catholic Univ. of Korea Seoul St. Mary "Fungal infections usually occur in immunocompromised patients and have a high fatality rate. Because fungal infections can be fatal to patients, especially during the resurgence of infectious diseases, such as COVID-19, it is of paramount importance to secure various treatment options." During a recent meeting with Daily Pharm, Sung-Yeon Cho, a professor in the Department of Infectious Diseases at the Catholic University of Korea Seoul St. Mary's Hospital, suggested potential improvements to treatment settings for fungal infections in South Korea. In August, the number of patients hospitalized for severe COVID-19 symptoms was a record high for this year, and concerns about the pandemic increased, posing threats to public healthcare due to fungal infections. The U.S. Center for Disease Control and Prevention (CDC) has designated September 16-20, 2024, as 'Fungal Disease Awareness Week' and notifies the importance of managing fungal infections. The most reported fungal infections are COVID-19 associated pulmonary aspergillosis (CAPA), COVID-19 associated mucormycosis (CAM), and Candida auris. These diseases worsen the prognosis of immunocompromised patients, leading to death. As infectious diseases resurge, the emphasis on fungal disease management and treatment becomes increasingly important. However, there are unresolved issues regarding the timely treatment of Korean patients, as the focus of governmental support has been on antibiotics among antimicrobials, and patient access to treatments is limited. European and U.S. guidelines suggest Vfend (voriconazole) and Cresemba (isavuconazonium) as first-line treatments for invasive CAPA. Cresemba won the Ministry of Food and Drug Safety (MFDS) approval in 2020 and it has been listed as the 'National Essential Drug.' However, it still has unmet medical needs because it is not reimbursed by national health insurance. This is in contrast with Cresemba's reimbursable status in Europe, the UK, Hong Kong, Taiwan, and China. Cho strongly suggested that fungal infections can be fatal to immunocompromised patients, and thus, patient access to treatments must be improved. Fungal infections, the fatality rate↑ in patients with COVID-19 CAPA is categorized as the highest-ranked, 'Critical Priority Group,' in the WHO-published 'Fungal Priority Pathogen List.' It shows the highest antifungal tolerability, mortality, and occurrence rate. CAPA is a variety of fungal infections induced by the 'aspergillosis' fungus, which exists in nature. Patients with respiratory diseases or are immunocompromised can commonly contract 'invasive CAPA.' Invasive CAPA is the most serious form of CAPA, usually occuring in immunocompromised patients. It commonly invades the lungs but can induce infections in any part of the body. "Fungus exists naturally, but infections do not happen when the immune system normally works. Patients receiving anticancer therapies, with a lowered number of neutrophils in white blood cells or taking extended steroids are vulnerable to fungal infections, " Cho said. "COVID-19-associated infections reduce normally working protective mechanism of upper/lower respiratory airways and make the body susceptible to all secondary infections related to the respiratory system," Cho added. Based on the results of a study involving 2427 patients with severe COVID-19 symptoms, 4.6% of all hospitalized patients and 11.2% of intensive care unit patients had CAPA infections. The median value of time it takes until CAPA diagnosis was 9.5 days. In particular, corticosteroid use in throughout the body has increased the risk of CAPA infections in COVID-19 patients. 30-day mortality was found to be higher in CAPA-infected patients (30%) than in patients with severe COVID-19 symptoms who are CAPA-negative (7.2%). "Fungal infections have a high mortality rate because they commonly occur in patients with lowered white blood cell neutrophils, who have undergone anticancer therapies, who are taking extended steroids, and who are immunocompromised. The mortality rate for CAPA is 30%. It is known to be up to 100% when not diagnosed," Cho said. "The rate of contracting CAPA is reported to be approximately 15-25% of COVID-19 patients who are hospitalized in intensive care units in South Korea. Since there are cases where CAPA is not diagnosed, depending on institutes and patient sizes, the incidence rate is reported to be as low as 5% and up to 50%. The CAPA mortality rate differs on diagnosis," Cho added. A previous study indicated the seriousness of fungal infections, showing that CAPA has a similar incidence rate and mortality rate in patients with viral infections, including severe influenza infections, besides COVID-19. WHO recommends physicians always consider the possibility of fungal infections when treating patients. Effective treatments have emerged, but are 'pie in the sky' The importance of fungal infection treatment has been emphasized, but patient access to new drugs is limited in South Korea. Voriconazole, a class of Azole, and Cresemba are prescribed as a first-line treatment of CAPA. Antifungal agents are used in patients who are likely to have COVID-19-associated CAM, besides CAPA. However, discussions about the reimbursable status of those agents are still challenging. Antibiotics include antimicrobials effective for treating germs, antifungals effective for treating fungus, and antivirals effective for treating virus. Among these, antifungals and antiviral agents are not included in the scope of the 'Cost-Effectiveness Evaluation Waiver System.' During the COVID-19 pandemic, the government included antimicrobials in the 'Cost-Effectiveness Evaluation Waiver System.' However, it sees that expanding the scope to antifungals does not meet the gravity and urgency standards. As a result, some are advocating for reimbursement of new antifungal drugs, citing the increasing incidence rate of fungal infections is on the rise worldwide, including in South Korea. However, reimbursement for new antifungals has not been made lately. "Voriconazole is a treatment with extended proof of superior effects in treating CAPA than other antifungals, based on accumulated data since the 1990s. However, there are concerns of extended treatments depending on the patient's immune conditions and refractory due to antifungal tolerance," Cho said. "Cresemba is in high demand among patients likely to have multiple infections as it is effective in treating broad spectrum fungal infections as well as improved safety profiles," Cho added, "Drug interactions that may occur in antifungals of Azole class is 2-10, whereas in Cresemba is as low as 1-1.5. It has the advantage of administering to patients with severe symptoms who are taking multiple drugs." Cresemba proved non-equivalence to the first-line treatment, voriconazole, and it is regarded as having a broader antifungal spectrum. Additionally, Cresemba significantly reduced the most typical adverse reactions of antifungals, such as liver toxicity and photosensitivity, to almost none. "The biggest reason for delayed reimbursement listing of antifungals is finance, but low awareness of infectious diseases can also be attributed. Although antifungals may be crucial to patients, they are not on the priority list," Cho said, "Reimbursement listing of antifungals is often being delayed because people think it is substitutable despite the fact that none are substitutable." "There should be no cases where good new drugs with confirmed clinical data and recommended by global guidelines cannot be prescribed in 2024 due to systemic issues," Cho emphasized, "The reimbursement criteria must be improved considering that patients who need antifungals have life-threatening severe infections."
- Company
- Imfinzi builds evidence for biliary tract cancer reimb
- by Whang, byung-woo Oct 02, 2024 05:49am
- AstraZeneca is reattempting to extend reimbursement of its immuno-oncology drug imfinzi (durvalumab) after announcing significant clinical outcomes in biliary tract cancer. Amid the rising interest in Imfinzi’s reimbursement reapplication review, with patient petitions being posted on the National Assembly's e-petition website, the outcome of the Korean subgroup analysis has been recently announced, gaining attention. 임핀지 제품사진According to industry sources, the Health Insurance Review and Assessment Service will hold the 7th Cancer Disease Deliberation Committee (CDDC) meeting on the 2nd of this month. This time, AstraZeneca is knowingly reattempting to extend Imfinzi’s reimbursement after failing to extend its reimbursement as a first-line treatment for biliary tract cancer last year. Last year, the company was not able to receive full coverage for the Imfinzi and GemCis (gemcitabine plus cisplatin) combination, and only GemCis was granted reimbursement at the time. AstraZeneca has now reapplied to CDDC for full coverage based on several additional grounds. Highlights include a Korean subgroup analysis of TOPAZ-1, Imfinzi’s Phase III study in patients with locally advanced or metastatic biliary tract cancer, which was presented at the Korean Society of Medical Oncology (KSMO) International Conference on October September 27th. The 3-year follow-up of 120 South Korean patients who were enrolled in the TOPAZ-1 study showed that the Imfinzi combination improved median overall survival (mOS), 3-year OS rate, and clinical safety compared to conventional therapy. Korean patients treated with the Imfinzi combination showed an mOS of 16.6 months, compared to the 11.3 months found in Korean patients treated with conventional therapy, which is a 5.3-month extension in overall survival. The OS rate at 3 years also improved over twofold, being 21.0% in the Imfinzi combination arm and 8.8% in the conventional therapy arm. Another factor that can also influence the decision is patient demand. A petition had been uploaded to the National Assembly on September 13 called for prompt insurance reimbursement of Imfinzi. The petitioner, who described herself as the child of a patient with stage IV intrahepatic biliary tract cancer, said she had been paying more than KRW 10 million a month for Imfinzi as out-of-pocket cost because of its non-reimbursement, and that she was now unable to afford the treatment because she had exhausted her insured actual expense limit. Despite Imfinzi’s proven effect, the petitioners believe that many patients have difficulty accessing the treatment due to financial reasons. As of September 30, the petition had received 5,114 signatures. Imfinzi is now recommended as a first-line standard of care in major biliary tract cancer treatment guidelines, including the National Comprehensive Cancer Network (NCCN) and the European Society for Medical Oncology (ESMO), and is reimbursed in major countries, including the United States and the United Kingdom. However, there is also a view that the cost of Imfinzi, an immuno-oncology drug, is burdensome for Korea’s health insurance finances and that the company’s proportion of financial sharing will determine whether it passes CDDC review, regardless of the drug’s clinical performance. The industry's view is that the fact that only GemCis was reimbursed in the Imfinzi combination regimen illustrates such government concerns. The government had decided to not fully reimburse the Imfinzi-GemCis combination because this would require significant additional health insurance funding to cover its use as first-line treatment for biliary tract cancer.
- Company
- Will the Imfinzi·Imjudo combo be reimb within the year?
- by Eo, Yun-Ho Oct 02, 2024 05:49am
- Whether the combination of the immuno-oncology drugs Imfinzi and Imjudo will be reimbursed for liver cancer in Korea is gaining attention. In June, AstraZeneca Korea submitted an application for the reimbursement of the PD-L1 inhibitor Imfinzi (durvalumab) and CTLA-4 inhibitor Imjudo (tremelimumab) combination for liver cancer. Therefore, it remains to be seen if it will be presented to the Health Insurance Review and Assessment Service’s Cancer Disease Review Committee within this year. Imjudo received approval in combination with Imfinzi from the Ministry of Food and Drug Safety in June last year. The first target indication for the combination was liver cancer, and the drug may be prescribed as a first-line treatment for adult patients with advanced or unresectable hepatocellular carcinoma (liver cancer). Specifically, patients are treated with the STRIDE (Single Tremelimumab Regular Interval Durvalumab) regimen, which consists of a single dose of Imfinzi 1,500 mg plus 300 mg of Imjudo, followed by an additional dose of Imfinzi at regular intervals every 4 weeks. At the recent European Society for Medical Oncology (ESMO) Congress 2024, the 5-year overall survival data from the Phase III HIMALAYA trial that demonstrated the efficacy of the Imfinzi and Imjudo combination in hepatocellular carcinoma was presented. In the HIMALAYA trial, patients with inoperable HCC were treated with STRIDE (single dose of Imjudo followed by Imfinzi maintenance therapy), Imfinzi monotherapy, and sorafenib monotherapy. When comparing the results of the Imfinzi and Imjudo combination with sorafenib combination therapy in patients with unresectable HCC, patients who received the STRIDE regimen had a 5-year overall survival (OS) rate of 19.6%, compared with the 9.4% for patients who received sorafenib. The median overall survival was 16.43 months and 13.77 months, respectively, showing a 24% lower risk of death in the Imfinzi-Imjudo combination arm. “ The Imfinzi-Imjudo combination therapy has significant advantages in that it has a much lower risk of bleeding than conventional therapies and does not worsen liver function," said Hong Jae Chon, Professor of Hemato-Oncology at CHA Bundang Medical Center. “In particular, the combination shows potential for longer survival than existing therapies."
- Company
- "Expanded scope for the newborn screening test"
- by Whang, byung-woo Oct 02, 2024 05:48am
- "Conducting genetic testing on family members and related families when a baby is found to be a carrier from a newborn screening test can identify more individuals with carriers. For rare diseases, such as Gaucher's disease, early diagnosis improves the patient's quality of life and saves the societal cost." Starting this year, the items for the newborn screening test, which is conducted in newborns within 48-72 hours of birth regardless of any symptoms shown, has expanded. As a result, early diagnosis of rare diseases is more likely. For example, reimbursable items now include Lysosome storage disorder (LSD). Many hope that circumstances surrounding the treatment of diseases with a 'diagnostic odyssey,' like Gaucher's disease and Fabry's disease, will improve. Beom Hee Lee, Professor of the Department of Pediatrics at Asan Medical CenterDaily Pharm met with Beom Hee Lee, a Professor of the Department of Pediatrics at Asan Medical Center's Medical Genetics Center, to discuss the significance of newborn screening testing and treatment options. LSD is estimated to occur in 1 in 7,000 to 9,000 people. There are approximately 50 known LSDs, depending on specific enzyme deficiencies. Among these, six LSDs, including Pompe disease, mucopolysaccharidosis (Type 1·2), Gaucher's disease, and Fabry's disease, have treatments and are manageable. "LSD is a disease in which lysosomal degrading enzyme, which eliminates unwanted materials within the cell, is genetically deficient. Most prominent diseases include Gaucher's disease, Fabry's disease, and Pompe disease," said Lee, adding, "However, the causes and symptoms are disease-specific. For example, in Gaucher's disease, the risk of leukemia increases, and neurological symptoms are life-threatening." The government's decision to include LSD in reimbursable items for newborn screening tests this year is crucial because it could lead to early diagnosis and early treatment. Approximately 70 patients are being treated for Gaucher's disease, one of LSDs, in South Korea. Because there are only a few patients in South Asia, it is difficult to assess the prevalence rate. Gaucher's disease is also a rare disease with the 'diagnostic odyssey,' taking around 13 years on average to diagnose. Lee said that the expanded reimbursement scope of the screening test is expected to be beneficial. "Patients often visit several hospitals until getting diagnosed because rare diseases are not acknowledged well and there are only a few doctors with specialties," Lee said, adding, "There are cases of patients diagnosed with Gaucher's disease in their last years, and a teen patient was diagnosed with Gaucher's disease with hepatosplenomegaly after 10-years of testing despite reduced platelet levels." "As six types of enzyme activity tests have been included in reimbursable items of newborn screening test, starting January 2024, potential for diagnosing Gaucher's disease has opened up," Lee said, adding, "It provides patient access and advantages for reducing complications by early disease diagnosis and treatment, suggesting that circumstances for treatment are improving." Lee explained that six months had passed since the testing was implemented, and no patients were reported to have abnormal findings diagnosed with Gaucher's disease. However, a positive effect is expected because the testing could expand the genetic disease LSD diagnosis to family members and related families. Gaucher disease requires early diagnosis and early treatment…unmet needs for treating neurological symptoms The standard therapy for Gaucher's disease involves receiving a bi-weekly intravenous injection of a deficient enzyme protein as part of an enzyme replacement therapy (ERT). Lee believes that different ERTs yield similar treatment effects. Since the treatment effects and safety profile have been confirmed, ERT may effectively lessen liver and spleen enlargement. "Neurological symptoms of Gaucher's disease occur regardless of age, typically found in infancy, teenage years, and early-20s, despite different timing of neurological symptom occurrence and diagnosis," Lee said, adding, "Even the patients who have improved symptoms with ERT can experience sudden neurological symptoms, significantly burdening patients and caregivers." Although there are no treatment options for effectively managing neurological symptoms, studies are being conducted with chaperone therapy using ambroxol. Ambroxol is an active ingredient used for treating respiratory diseases. In a study of ambroxol in combination with ERT, it has been reported to reduce the number of seizures and alleviate or prevent symptom worsening after around 10 years of treatment in patients with Gaucher's disease who have neurological symptoms. "Although many patients have improved neurological symptoms using ERTs, patients had the inconvenience of having to take many pills in a day. Moreover, ambroxol is not officially approved," Lee said, adding, "A treatment development is needed to fulfill unmet patient medical needs for effective neurological symptoms." Despite receiving positive review for newborn screening test, a concern for increased screening cost arises Lee expected that discussions about potential unnecessary screening increases due to the expanded scope of the newborn screening test might be needed. "When tests are being done in a broad term, an initial purpose for conducting screening may not be achieved and it may result in unwanted outcomes," Lee said, adding, "Caregivers tend to independently receive genetic testing with low accuracy in addition to the government-funded tests. As a result, the cost of conducting tests with out-of-pocket fees may be a burden." Some diseases may require immediate treatments upon the identification of a carrier. However, several diseases, within the six types of LSD testing category, do not require immediate treatment. "Testing with fees conducted in newborns may not be for diseases that must be identified at the newborn stage, and the rate of the diagnosis for certain disease at further tests are low," Lee mentioned, "It is a common issue that nations conducting similar screening program face." Regarding this issue, Lee emphasized the importance of accurate final diagnosis and treatment through enzyme analysis testing or genetic testing rather than excessive analysis at the screening stage. "For Gaucher's disease, treatments are available, and patients can maintain typical lifestyles if treated and managed well. It does not affect marriage or daily lives," Lee said, "The most crucial factor is early diagnosis and early treatment. Because Gaucher's disease can cause irreversible damages to the body, treatment must begin before symptom manifests."
- Company
- Jardiance generics prepare for final launch in Korea
- by Kim, Jin-Gu Sep 30, 2024 05:47am
- Generic companies are in the midst of last-minute preparations to enter the market for the SGLT inhibitor diabetes drug Jardiance (empagliflozin), which is set to expire in just about half a year. With Forxiga’s market withdrawal expected to leave an annual sales gap of KRW 50 billion, the race to fill this gap is expected to intensify among the generic companies. Generic companies seek to avoid Jardiance’s crystalline form patent 8 years later…are they eyeing Forxiga’s gap? According to industry sources on the 26th, Hanmi Pharmacuetical and KyungDong Pharm recently won the passive trial to confirm the scope of Jardiance’s crystalline form patent (10-1249711). In the case of Hanmi Pharmaceutical, it succeeded in avoiding Jardiance’s crystalline patent a step later than other companies. More than 50 companies, including Chong Kun Dang, have challenged Jardiance’s crystalline form patent since 2018 and won their first trial between 2019 to 2021. Hanmi Pharmacuetical had also filed to invalidate the same Jardiance’s patent in 2015 but hadn’t made much action since losing the first trial the following year. Even when the other companies filed a series of challenges to avoid the crystalline patent, Hanmi Pharmaceutical chose not to join in the race. However, in March this year, the company changed its course and again filed to avoid the crystalline patent. The company’s challenge comes 8 years later than the other companies. Like the other companies, Hanmi Pharmaceutical plans to launch its generic early, when the patent for Jadian expires in March next year. Some speculate that AstraZeneca’s decision to withdraw another SGLT-2 inhibitor, Forxiga, from South Korea, may have played a role in this. AstraZeneca Korea decided to withdraw Forxiga from the Korean market last December. Forxiga was the leading SGLT-2 inhibitor in the market until recently. With the market leader withdrawing from the market, industry insiders believe that the generic companies are seeking to target this void created by the withdrawal. In fact, prior to Hanmi Pharmaceutical, Daehwa Pharmaceutical, Medica Korea, Korea Prime Pharmaceutical, and Aprogen Biologics filed challenges to avoid the crystalline patent and launch their generic versions of Jardiance in March this year. All of these challenges were filed after AstraZeneca decided to withdraw Forxiga from South Korea. According to the market research institution UBIST, Forxiga’s prescription sales amounted to KRW 51 billion in 2022. Last year, its prescriptions grew 9% to KRW 55.5 billion, despite the launch of its generics upon Forxiga’s patent expiry. The rise in prescription sales was slightly slower this year, earning KRW 21.7 billion in the first half of the year. In Q2, the drug’s sales were overtaken by the combined prescription volume of its generics. As a result, the drug’s prescriptions are expected to decline in earnest in the second half of this year. AstraZeneca Korea plans to supply Forxiga domestically only until the first half of this year, and then distribute only the stockpiled amount. This means that from next year, a KRW 50 billion gap will remain unfilled in the market in earnest. Last-minute generic approvals also in full swing...companies expected to push ahead with launch despite unregistered patent risk Due to such circumstances, the domestic pharmaceutical companies' expectations for Jardiance’s generic market have also been growing ahead of the expiration of the product patent. Companies that have previously succeeded in avoiding Jardiance’s crystalline patent are in the midst of last-minute preparations, including receiving authorizations for their generics one after another. Only this month, GC Cross and Dongkwang Pharm received approval for their generic versions of Jardiance and Jardiance Duo. Including the two, there are now 53 companies that have authorized generic versions of Jardiance and Jardiance Duo. If Hanmi Pharmaceutical and Daehwa Pharmaceutical, which succeeded in avoiding Jardiance’s crystalline patent, also receive approval for their respective generic versions, the number of approved generic companies is expected to increase to 60. In other words, fierce competition among Jardiance generics is expected from March next year. The product patent for Jardiance will expire in March next year. Other than the product patent, the crystalline patent is the only patent registered in MFDS’s green list. Generic companies plan to avoid the crystalline patent and release generics early in line with the date of Jardiance’s substance patent expiry. Although there are still patents that have not been registered in Korea’s green list, most companies are expected to push ahead and launch their respective generics. In the case of Trajenta generics, most companies pushed ahead with their generic launches without fully addressing the risk of its unlisted patents.
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- New myelodysplastic syndrome drug 'Reblozyl' lands Big 5 DC
- by Eo, Yun-Ho Sep 30, 2024 05:46am
- Product photo of Reblozyl. The treatment for myelodysplastic syndrome (MDS), 'Reblozyl,' is available for prescription at tertiary general hospitals. Sources said that Bristol Myers Squibb (BMS) Pharmaceutical Korea's Reblozyl (luspatercept) has passed all drug committees (DC) of the 'Big 5' medical centers, including Samsung Medical Center, Seoul National University Hospital, Seoul St. Mary's Hospital, Seoul Asan Medical Center, and Sinchon Severance Hospital. However, Reblozyl is still non-reimbursable. The drug was not approved for reimbursement appropriateness at the Drug Reimbursement Evaluation Committee (DREC) of the Health Insurance Review and Assessment Service (HIRA), held August last year. It has not been updated since then. Reblozyl can be prescribed to treating ▲patients with very-low risk, low-to intermediate-risk MDS or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis ▲anemia in adult patients with myeloproliferative neoplasms ▲patients with Beta Thalassemia who may need RBC transfusions. Reblozyl is for adults who have not responded well to an Erythropoiesis-Stimulation Agent (ESA) or may need red blood cell (RBC) transfusions. Reblozyl can be administered once every 3 weeks in patients with MDS or Beta Thalassemia at an initial dosage of 1.0 mg/kg. Reblozyl's mechanism works by binding to TGF-β superfamily ligands, thereby diminishing the overactivation of the Smad 2/3 pathway. The drug promotes erythroid maturation. The efficacy of Reblozyl was demonstrated through the Phase 3 MEDALIST study. During the 24-week follow-up period, the study results showed that 13% of the placebo group reached consecutive transfusion-free periods, whereas 38% reported in the Reblozyl group. During the same period, the percentages of patients who reached a transfusion-free period over 12 weeks were 8% for the placebo group and 28% for the Reblozyl group. Those for over 16 weeks were 4% for the placebo group and 19% for the Reblozyl group. Extending the follow-up period to 48 weeks, the percentages of patients who reached a transfusion-free period over 16 weeks were 7% for the patient group, whereas 28% for the Reblozyl group. Meanwhile, MDS is a type of malignant disease affecting blood stem cells in the bone marrow. It is a disease of the elderly, with higher occurrence in elderly over 60 years of age. MDS is characterized by immature blood cells and low counts of healthy white blood cells, red blood cells, or platelets in the peripheral blood. The most common adverse reactions are fatigue from anemia, systemic weakness, and loss of motor ability. Worsened anemia could cause palpitation, trouble breathing, chest pain, and the possibility of advancing to acute myeloid leukemia (AML). The clinical outcomes and the progress were found to be categorically diverse. There were cases of a stable life with slight anemia, but a case of death within a few months from complications associated with low counts of red blood cells or acute leukemia was reported.
