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- 2025-12-24 12:41:40
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- Will the leukemia ADC Mylotarg be reimb this time?
- by Eo, Yun-Ho Apr 17, 2023 05:58am
- whether the new drug for acute myeloid leukemia (AML), ‘Mylotarg’ will be reimbursed in its second attempt is gaining attention. Mylotarg (gemtuzumab ozogamicin), which submitted an application for reimbursement listing earlier this year, is likely to be presented to the Cancer Disease Review Committee of the Health Insurance Review and Assessment Service this month. Also, another ADC drug, Enhertu (trastuzumab deluxtecanis also expected to be presented for review at the CDDC meeting. Mylotarg was deliberated by the HIRA’s CDDC in May last year but was unable to pass review and set reimbursement standards at the time. Therefore, whether Mylotarg will be able to pass CDDC this time and start on its journey to receiving reimbursement in Korea remains to be seen. The drug is an antibody-drug conjugate (ADC) approved as a first-line treatment for patients with newly-diagnosed CD33-positive AML. The drug, which received marketing authorization in Korea in December 2021, is an ADC composed of a CD33-targeting monoclonal antibody linked to calicheamicin, a potent cytotoxic agent. The drug works on cells that express the CD33 antigen, which is expressed on more than 90% of AML patients. This blocks cancer cell growth and induces apoptosis. Mylotarg’s approval was based on a clinical trial (ALFA-0701) conducted on 271 patients aged between 50 to 70 with newly-diagnosed AML with no prior treatment experience. The ALFA-0701 trial was an open-label, randomly assigned, multicenter Phase III study that compared the existing standard front-line chemotherapy, daunorubicin+cytarabine combination therapy, with Mylotarg+ daunorubicin+ cytarabine combination therapy. Results showed that the median event-free survival (EFS) in the Mylotarg+daunorubicin+cytarabine combination arm was 17.3 months, a 7.8-month extension compared to the 9.5 months in the daunorubicin+cytarabine combination arm. Also, the Mylotarg combination therapy reduced the risk of induction failure, relapse, or death by 44% compared to chemotherapy alone. Also, the median relapse-free survival (RFS) was 28.0 months in the Mylotarg+daunorubicin+cytarabine combination arm and 11.4 months in the daunorubicin+cytarabine combination arm, showing a significant difference of 16.6 months. In the case of median overall survival (OS), the median OS was 27.5 months in the Mylotarg+daunorubicin+cytarabine combination arm and 21.8 months in the daunorubicin+cytarabine combination arm, and the difference was not statistically significant.
- Company
- Sales of cepha antibiotics make a rebound
- by Kim, Jin-Gu Apr 14, 2023 05:49am
- Biopharmaceutical companies that produce the so-called ‘cepha antibiotics' have taken a breather with the increased demand for related products due to the rapid increase in confirmed COVID-19 cases last year. This is in stark contrast to the situation of the previous year when many companies were contemplating whether to withdraw their cepha businesses. However, the dominant opinion is that the sales increase last year was a temporary phenomenon due to the rapid increase in confirmed COVID-19 patients. The frontline companies unanimously agree that they are still contemplating whether to reduce or discontinue their cepha business amid the recent trend of a steady decline in antibiotic prescription rates and a steady rise in production costs. ◆Sales of cepha antibiotics make a rebound...influenced by the surge in confirmed COVID-19 patients According to the Financial Supervisory Service on the 13th, Yungjin Pharm's antibiotic-related sales increased 15% in one year from KRW 59.6 billion in 2021 to KRW 68.6 billion last year. Sales of products it directly produces and sells such as Clamonex, Cefaclor, and Ceftazidime have increased 40% from KRW 30.6 billion to KRW 42.7 billion. In the same period, special sales of Cepha antibiotic APIs soared from KRW 200 million to KRW 1.8 billion. However, exports of cefcapen, cefditoren, and ceftazidime fell 16% from KRW 28.9 billion to KRW 24.2 billion. Yungjin Pharm's antibiotic-related sales have steadily declined until 2021. Its related sales, which reached KRW 102.2 billion in 2019, decreased 42% in two years to KRW 85.6 billion in 2020, then to KRW 59.6 billion in 2021. However, sales made a successful rebounded last year. It is analyzed that this is due to the increase in prescriptions to relieve COVID-19-related symptoms in line with the surge in confirmed COVID-19 patients in Korea. According to the market research institution UBIST, prescriptions of oral cephalosporins last year were KRW 259.6 billion, up 33.4% from the previous year. The ‘cepha antibiotics,’ or cephalosporins are widely used antibiotics for pneumonia, laryngopharyngitis, tonsillitis, and bronchitis. The situation has turned drastically from the first and second years of COVID-19. The cephalosporin antibiotic prescription market, which was worth KRW 271.1 billion in 2019, shrank to KRW 211.5 billion in 2020, then to KRW 194.6 billion in 2021. In the early phases of COVID-19, the market shrank greatly with the plummeting number of cold and flu patients, but last year, along with cough and cold preparations, their use for the purpose of relieving COVID-19 symptoms increased explosively. ◆Presciptions rise 33% in 1 year...companies that pondered market withdrawal breathe a sigh of relief Other cepha antibiotic manufacturers also experienced a similar situation. In particular, companies that had been considering withdrawing their businesses due to a steady decline in sales until the previous year were able to take a breath of relief. Boryeong's sales related to cepha antibiotics shrank from KRW 25.3 billion in 2019 to KRW 20.4 billion in 2020. However, sales rebounded to KRW 21.7 billion last year. Korus Pharm has been manufacturing cepha-class antibiotics such as Korus Cefaclor, Korus Ceftriaxone, K Axone, Cefozol, etc. The company’s combined antibiotic sales decreased 24% in 2 years from KRW 6 billion in 2019 to KRW 4.5 billion in 2021, but rebound to 5.3 billion last year. Withus Pharm’s Withus Cefaclor Cap’s sales fell to KRW 0.6 billion in 2020 from KRW 1.5 billion in 2019, then increased over threefold in 2 years to reach KRW 2.1 billion last year. Withus Pharm’s antibiotic sales, including Cefaclor Cap, increased 2.5 times in 2 years from KRW 2 billion in 2020 to KRW 5 billion last year. Daewoong Bio also saw its cepha antibiotic Ceclor’s sales increase 32% from KRW 7.4 billion in 2021 to KRW 9.8 billion last year. In addition, finished cepha antibiotic products from Kyungbo Pharmaceuticals, Kukje Pharm, and Jeil Pharm increased last year. However, Kyungbo’s sales of cepha-class antibiotic APIs decreased 4% from KRW 37.6 billion in 2021 to KRW 36.3 billion. ◆”Rise in cepha-class drugs temporary...still not profitable” This is in contrast to the years 2020 and 2021 when many companies were contemplating whether to withdraw their cepha antibiotic business. In 2020, a large pharmaceutical company A decided to discontinue consignment production of cephalosporin antibiotics. Company A has stopped manufacturing products contracted by other pharmaceutical companies and is only currently producing only its products. Another large pharmaceutical company, B, put up its cephalosporin antibiotic manufacturing plant for sale last year. A few companies considered taking over, but pharmaceutical company B withdrew its intention to sell. Company C, a mid-sized pharmaceutical company, also contemplated withdrawing from the cepha antibiotics business last year. On the surface, the company pointed to the decrease in prescriptions of cephalosporin antibiotics in the process of prolonged COVID-19, but the analysis was that the chronic deterioration of profitability caused them to consider withdrawing from the business. In line with the trend, the industry anticipates the companies’ withdrawal from the cepha antibiotics business will be repeated this year. Front-line companies all agree that the deteriorating profitability of antibiotics will not improve due to the rising cost of APIs day by day, while supply prices are unchanged under Korea’s health insurance. The price of APIs of cephalosporin antibiotics imported from China and India has risen by 10-20% during the past 2 years. On the other hand, the reimbursed product prices have stayed the same for a long period of time. The fact that the government is constantly trying to reduce antibiotic prescriptions is also cited as a reason for the deteriorating profitability. The antibiotic prescription rate for acute upper respiratory infections has decreased from 52% in 2010 to 36% in 2020. An official from a pharmaceutical company that produces cepha antibiotics said, “We have almost no profit gain from the manufacture of 1st and 2nd generation cephalosporin antibiotics. Labor costs have also risen significantly, so it is difficult to make any profit.”
- Company
- “Reimb standard for hemophilia should align with approvals"
- by Jung, Sae-Im Apr 14, 2023 05:49am
- Young-Sil Park, Treasurer of KSH The Korean Society of Hematology has set out to narrow the gap between the approved indication and reimbursement standards for hemophilia treatments. Through the amendment, KSH aims to apply the optimal dose and dosing schedule for each patient as personalized treatment strategies have settled as the standard for maintenance and prevention therapies. Therefore, whether the government will accept the new reimbursement standard plan proposed by the KSH remains the focus of attention. Young-Sil Park, Treasurer of KSH (Department of Pediatrics, Kyung Hee University School of Medicine), met with reporters on the 13th and said “We have submitted a proposal for the amendment of the reimbursement standards for hemophilia drugs in the Fall last year and is awaiting results. Although there are several steps left to final reimbursement extensions, I heard that HIRA has shown a positive response." The amendment to the reimbursement standards that KSH proposed can largely be divided into two parts. Increasing dose amount and frequency of administration. Currently, the reimbursement standard is set narrower than the approved maximum dose and number of administrations. For this reason, it has been pointed out that the treatment effect may be reduced due to insufficient single dosage amount depending on the patient. Therefore, KSH’s argument is that the maximum level in the reimbursement standard should be set at the same level as each drug’s indication. Drug reimbursement standard improvement plan, reconstituted by Dailypharm Specifically, KSH requested the dosage allowed for administration with each reimbursement to be increased from the current 20-25 (IU/kg) to a maximum of 50. The amendment would allow the dose to be increased flexibly according to the patient's condition, with the dose for standard half-life formulations to range from 20 to 40 (20 to 50 for children under 6 years of age) and extended half-life formulations to range from 40 to 50. These are the scope of dosages specified in the label for each formulation. The KSH also suggested the reimbursed number of monthly administrations allowed for patients should be expanded. According to the current reimbursement standard, patients can visit the hospital once or twice a month and receive a total of 10 doses of standard half-life treatments. Severely ill patients are allowed up to 12 doses. A total of 7 doses (8 doses for severely ill patients) are reimbursed for extended half-life drugs during the 1-2 visits per month. Drug reimbursement standard improvement plan, reconstituted by Dailypharm This means that standard half-life drugs can be administered three times a week for severe patients. However, experts pointed out that it is often necessary to adjust the frequency of administration depending on the patient's condition. Accordingly, the proposed amendment stipulates that 'if the trough concentration level cannot be maintained at 1% or higher 48 hours after setting and administering the maximum dose allowed according to the individual pharmacokinetic test result, administration of an additional dose up to the maximum dose permitted in the label may be accepted.’ Treasurer Park said, “Currently, patients are administered treatments three times every 7 days, on Day 2, Day 4, and Day 7. In some cases, almost no clotting factors remain in the body on the third day for some patients. Our proposed improvement to the plan offers an increased number of administrations so that the optimal effect can be achieved for those whose minimum coagulation factor level is not maintained on the second day.” The KSH is requesting another revision 4 years after the reimbursement standards for hemophilia treatment were revised because the hemophilia treatment has now been expanded to maintenance and preventive therapies. In the past, treatment of hemophilia was centered around ‘replacment therapy', in which clotting factors were administered when bleeding occurred. In recent years, the treatment paradigm has evolved to 'maintenance and prevention therapy', which prevents bleeding in advance by regularly administering clotting factors even without bleeding events. Furthermore, ‘personalized treatment', where a patient’s administration cycle and dose are customized in consideration of the patient's symptoms, living environment, and pharmacokinetics, has emerged as a trend. Currently, the treatment goals for patients with severe hemophilia consist of maintenance and prophylaxis therapies. Also, the introduction of drugs with extended half-life has reduced the required number of regular administrations, and an application that allows patients to determine their pharmacokinetic levels has been introduced, customized maintenance and prevention therapy have settled as the standard of care. However, the reimbursement standards that have been set in the past are narrower than the scope approved, which limits the realization of customized maintenance and prevention therapy. Park added, “The reimbursement standards for hemophilia have improved a lot compared to the past. I believe that a change in the reimbursement standard is necessary to achieve the optimal effect for patients in line with the changes made to the domestic treatment environment and the hemophilia treatment environment. I look forward to the government's positive decision that can allow personalized treatment for each individual in consideration of each patient’s factors."
- Company
- “Olumiant rises as a new option for alopecia areata”
- by Jung, Sae-Im Apr 13, 2023 05:46am
- A new treatment option has emerged in the field of severe alopecia, an area where no option other than local steroid therapy had existed until now. The drug gained attention from patients with severe circular hair loss due to its safe and high therapeutic effect. However, it remains unclear whether the indication for circular hair loss will be able to rise to the right to benefit. Lilly Korea held a press conference at The Plaza Hotel in Jung-gu, Seoul on the 12th and explained the meaning of expanding the indication for severe circular hair loss of the JAK inhibitor 'Olumiant (ingredient: baricitinib)'. Oh-sang Kwon, professor of Dermatology at Seoul National University Hospital, and Bark-Lynn Lew, Professor of dermatology at Kangdong Kyunghee University Hospital, explained the current state of alopecia areata in Korea, the disease burden, and the effects of Olumient. Professor Kwon served as the vice president of the Korean Hair Research Society and Professor Lew serves as the Academic Director of her academic society. Professor Oh-sang Kwon (left) Professor Bark-Lynn Lew (right) According to Professor Lew, as of 2021, about 170,000 patients visit the hospital with alopecia areata a year in Korea. Young patients in their 20s to 40s account for about 60% of the AA population. Unlike general hair loss caused by hormones, alopecia areata is classified as one of the symptoms of an autoimmune disease. The immune system mistakenly recognizes part of one’s hair as a foreign substance, and the hair falls out. Unlike general hair loss, in which the hair gradually thins, the forehead widens in the shape of an M, and the hair loss expands, in alopecia areata, one or several circular bald areas with clear boundaries occur on the scalp. Bald spots can occur not only on the scalp, but also anywhere on the hair, such as eyebrows, beard, armpit hair, and pubic hair. Professor Lew added, “Most hair loss is naturally cured and responds well to treatment, but about 40% of patients experience recurrence within a year, and in severe cases, all hair on one’s scalp or body falls out and progresses to widespread alopecia. Alopecia areata is a chronic autoimmune disease, which has a high risk of autoimmune disease complications such as atopic dermatitis at the same time and can have a significant impact on the quality of life of patients. Its lifetime prevalence of the psychiatric disorder is up to 74%." he explained.” Before Olumiant, there was no treatment approved for severe alopecia areata. Instead, topical steroids were used off-label for patients with mild symptoms. However, the efficacy evidence was limited on its use while the risk of side effects was high from long-term steroid use. The efficacy of Olumiant was demonstrated through the Phase III BRAVE-AA1 and BRAVE-AA2 trials that were conducted on a total of 1,200 patients with severe alopecia areata. Koreans participated in both studies. The trials’ primary efficacy endpoint was a SALT score of 20 or less. SALT stands for Severity of Alopecia Tool score. 41%, and 37% in the group treated with Olumiant for 52 weeks in AA1 and AA2, respectively, achieved the primary efficacy endpoint. At the time, improvements in eyebrow and eyelash coverage were also observed. The company plans to further confirm the effectiveness and safety of Olumiant treatment for up to 200 weeks. Professor Kwon said, "What is noteworthy in the 52-week extension study is that the regrowth effect of scalp, eyebrows, and eyelashes continued to improve for up to 52 weeks when treated with Olumient 4mg. This shows that long-term treatment may be required to achieve maximum benefit in AA patients." Patients’ responses were also positive. Professor Lew said, "Although it is reimbursed yet, about 60% of patients were willing to use Olumiant when it was recommended. This is twice more than my expectations, indicating the high willingness to treat among the patients.” However, it is expected that the AA indication will not be covered for the time being. Lilly Korea said, "It's only been a month since the indication was added, so nothing has been decided or progressed related to reimbursement yet. We will make efforts to expand access to Olumiant in AA patients with high unmet needs.”
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- Supreme Court dismissed again
- by Chon, Seung-Hyun Apr 13, 2023 05:44am
- Pharmaceutical companies won a complete victory in the 2nd round suspension of execution of the lawsuit to cancel the reduction of benefits for the brain function improving Choline alfoscerate. Following Daewoong Bio Group, Chong Kun Dang Group also issued a ruling in the Supreme Court to suspend the implementation of wage reduction until the main lawsuit in the second trial is over. According to the industry on the 7th, the 3rd Special Division of the Supreme Court dismissed the decision of the Ministry of Health and Welfare to suspend the enforcement of choline drug benefit reduction. It is a decision to suspend the effect until the 30th day from the date of adjudication of the cancellation case of the ‘Announcement of Partial Revision of Details on the Application Criteria and Methods of Medical Care Benefit’, which contains the reduction of health insurance benefits for choline preparations. This means that 26 pharmaceutical companies including Chong Kun Dang and 8 individuals will not be able to reduce benefits until the end of the second trial in progress with the Ministry of Health and Welfare to cancel benefit reduction for choline drugs. This means that the benefit reduction will not be implemented until the end of the second trial of the ongoing lawsuit against the Ministry of Health and Welfare between 24 pharmaceutical companies such as Daewoong Bio and one individual to cancel the benefit reduction of choline drugs. In August 2020, the Ministry of Health and Welfare issued a partial revision notice of ‘Details on the Application Criteria and Methods of Medical Care Benefits’, which contains the content of reducing the scope of benefits for choline preparations. It is content that increases the cost burden rate from 30% to 80% when patients who have not been diagnosed with dementia use cholinergic drugs. After the Ministry of Health and Welfare issued a notice, a lawsuit began all at once. Pharmaceutical companies filed a lawsuit to cancel the notice of reduction in benefits. The lawsuit was filed in two groups according to the legal representative. Law firm Shin & Kim filed a lawsuit on behalf of 39 companies, including Chong Kun Dang, and eight individuals, while Lee & Ko, a law firm, took on the lawsuit for 39 companies, including Daewoong Bio, and one person. However, the Chong Kun Dang Group lost in July of last year, and Daewoong Bio also received a ruling in November of last year. The pharmaceutical companies filed an appeal and also requested a suspension of execution of the reduction in benefits. The suspension of execution requested by Daewoong Bio Group was cited in December of last year. The Ministry of Health and Welfare requested an appeal for suspension of execution, but the Supreme Court also sided with the pharmaceutical companies last month. The Chong Kun Dang Group’s request for suspension of execution also received a ruling in November of last year, “Suspend the enforcement of the public notice until the 30th day from the date of the second trial judge.” Pharmaceutical companies have all led to the suspension of execution in the first trial of the lawsuit for cancellation of benefit reduction. Pharmaceutical companies filed a lawsuit to cancel the reduction in benefits in 2020 and requested suspension of execution to suspend the implementation of the reduction notice until the main lawsuit. The lawsuit for suspension of execution was also filed in two groups according to the legal representative. The suspension of payroll reduction enforcement requested by Chong Kun Dang and others was completed until the Supreme Court ruling in April 2021. In September 2020, the Seoul Administrative Court made a decision to suspend execution, and in the appeal trial in December of the same year, the court sided with the pharmaceutical companies. The Supreme Court upheld the decision of the lower court even in the appeal of the suspension of execution. The suspension of execution of choline drugs, which was raised by Daewoong Bio and others, was cited in October 2020, and nine months after the Ministry of Health and Welfare appealed, the second trial also issued a decision citing the suspension of execution. In October of last year, a ruling was dismissed in an appeal filed by the Ministry of Health and Welfare. From the pharmaceutical company's point of view, it means that they have won all 10 cases of suspension of execution of choline drug benefit reduction. Looking at the ruling in the case of suspension of execution, the court judged that "there is no reason to admit that there is a concern that the suspension of choline drugs may have a significant impact on public welfare." The court pointed out that due to the reduced reimbursement for cholinergic drugs, patients may find themselves in a situation where they have to continue to be prescribed the drug or give up taking it while bearing a considerably higher co-payment than before. The court judged that if benefits were reduced because the clinical usefulness of choline preparations was not proven, the trust and reputation of choline preparations and the reputation of pharmaceutical companies could be damaged, and the related market could suffer a major blow.
- Company
- SK Bioscience declares its vision
- by Kim, Jin-Gu Apr 13, 2023 05:43am
- SK Bioscience announced on the 12th that it held a vision proclamation ceremony at Andong L House on the 11th and pledged to become a 'global bio hub' through 'One Goal, One Team'. About 450 executives and employees attended the vision proclamation ceremony, including Ahn Jae-yong, CEO of SK Bioscience, Kim Hoon, CEO of Global R&BD, and Lee Sang-gyun, plant manager (vice president) of L House. The vision proclamation ceremony consists of a time to look back on the achievements and major milestones that L House has achieved during the COVID-19 pandemic and to share future strategies and goals at the time of transition to the endemic era. SK Bioscience shared the future strategy of 'SKBS 3.0', which consists of specific action tasks and action plans to grow into a global company in the endemic era, and a 'digital establishment plan'. In addition, to achieve the One Goal of 'Global Innovative Partner of Vaccine and Biotech', the members of L House were asked to join forces as one team in the future. In particular, the acquisition of cGMP (Current Good Manufacturing Practice) from the U.S. Food and Drug Administration (FDA) was selected as a key task for L House to achieve SK Bioscience's vision. Along with this, SK Bioscience plans to solidify its position as a global vaccine production hub by expanding facilities of about 1,067,616 sqft in L House. In addition, it plans to build a global top-tier R&D network through the 325.7 billion won Global R&PD Center being established in Songdo, Incheon with the goal of completion in 2025. Ahn Jae-yong, CEO of SK Bioscience, said, "Today's vision proclamation ceremony is a place where the members of House L announce their will to achieve 'One Goal' by gathering strength through the 'One Team' culture." We will secure a vaccine portfolio and promote sustainable growth.” Lee Sang-gyun, Plant Manager, said, “SK bioscience has been able to stand at the center of the response to the COVID-19 pandemic over the past three years because of the valuable efforts of L House members.” I am grateful to the staff," he said.
- Company
- LSK's contracts for early-phase clinical trials increase
- by Lee, Tak-Sun Apr 12, 2023 05:54am
- After analyzing Korea’s clinical trial trend through the number of its contracted research, LSK Global Pharma Services Co., Ltd. (LSK Global PS) announced that the number of early-phase clinical trials increased significantly recently. As of March 2023, the company’s number of contracted clinical research was 1,503, including 164 global clinical trials and 1,339 domestic clinical trials. Since its establishment in 2000, the company’s number of contracted research cases reached 1,000 in 2018 and then increased by 50% in 5 years thereafter. By research type, the number of Phase III clinical trials was the highest at 372 cases, and the rate of increase or decrease in the trials compared to 2018 showed that early phase trials such as Phase II clinical trials (▲ 62%) and Phase I clinical trials (▲ 49%) increased the most. A similar trend can be observed when dividing the domestic clinical trial approval status by phases. In the past, late-stage clinical trials were more actively conducted than early-stage clinical trials in Korea. However, with various domestic clinical trials being conducted by CROs and accumulated experience, early clinical trials are now being actively conducted, and related infrastructure has also been established. To accommodate the increase in demand, in 2021, LSK Global PS explained that it has been providing differentiated services by operating a team dedicated to early-phase trials to respond to the increasing demand for early-phase clinical trials. This also aligns with the global drug development trend. Anticancer drugs are still the most actively researched area in new drug development due to high unmet demand, and 10 to 20 new drugs are approved by the US FDA every year in the area. In Korea, R&D investment and recruitment of research personnel in the field of anticancer drugs is being actively carried out recently. Young-Jack Lee, CEO of LSK Global PS, said, “Unlike treatments for other diseases that are highly prevalent, there are many factors to consider when developing an anti-cancer drug, such as disease complexity, tumor heterogeneity, toxicity, and resistance, and require huge time and monetary investments. LSK Global PS offers medical consulting throughout clinical trials for anticancer drugs from setting target patient groups, study design, and efficacy endpoint suggestions to help establish and conduct strategic clinical trials.” A total of 129 clinical trials (54 global, 75 domestic) related to Investigational New Drug Applications (IND) that are conducted to receive approval from regulatory authorities were found to be in progress. By clinical stage, Phase I and Phase II clinical trials, and by disease group, anticancer drugs increased the most, showing a similar trend with the change in the total number of contracted clinical research. In addition to the continuous rise in the number of contracted clinical research related to new drug development, marked growth has also been observed in the fields of digital therapeutics and medical devices. Compared to 2018, the number of contract clinical research cases for medical devices had increased around threefold, 10 of which trials are related to digital therapeutics. LSK Global PS newly established a medical device clinical team in January of last year to provide more specialized services.
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- PharmaEssentia attempts to reimb its first new drug BESREMi
- by Eo, Yun-Ho Apr 12, 2023 05:53am
- The Taiwanese pharmaceutical company PharmaEssentia is attempting to list its first new drug ‘Besremi’ for reimbursement. According to industry sources, PharmaEssentia submitted an application for the reimbursement of its polycythemia vera treatment, Besremi (Ropeginterferon alfa-2b) on March 28th. Polycythemia vera is a rare blood disorder where a somatic cell mutation in the bone marrow abnormally activates bone marrow function and produces excessive red blood cells. It has a short survival period and is so fatal that 10~15% of patients with polycythemia vera develop myelofibrosis or leukemia within 10 years. Although hydroxyurea had been used as the standard of care, it was difficult to fundamentally cure the disease with hydroxyurea, and patients who could not be treated with hydroxyurea had limitations as there were practically no drugs available for them in Korea’s domestic reimbursement environment. Besremi is an interferon treatment that selectively removes JAK2 mutations that cause polycythemia vera. In Korea, the drug received approval in October 2020 to treat low-risk and high-risk patients with polycythemia vera without symptomatic splenomegaly. The drug demonstrated its potential as a radical treatment for polycythemia vera in patients who had not received cytoreduction therapy or received less than 3 years of treatment with hydroxyurea. Therefore, whether the only interferon treatment option approved for polycythemia vera will be reimbursed is gaining attention. Besremi demonstrated its efficacy and safety in the Phase III PROUD/CONTINUATION-PV trial that was conducted on polycythemia vera patients. Trial results showed that 53% of the patients in the Besremi arm achieved a complete hematological response, an improvement compared with the hydroxyurea patient arm (38%). The response rates at 72 months were high at 80.4% and 65.3% in low-risk and high-risk patients, respectively, and showed high hematologic and molecular responses. Regardless of their risk, patients treated with Besremi did not require phlebotomy even 6 years after administration. Sung-Soo Yoon, Professor of Hemato-Oncology at Seoul National University Hospital, said, “Polycythemia vera is currently left unattended in terms of reimbursement in Korea. Patients that show no response to hydroxyurea, the current standard of care, had no appropriate treatment options available for use and had no option but to wait for their condition to progress further. He added, “Korea’s clinical practice guidelines recommend interferon and ruxolitinib as second-line treatment for patients who show intolerance or are refractory to hydroxyurea, but both drugs are currently unreimbursed, and other interferon treatments have withdrawn from the Korean market. Therefore, as the only treatment option available, Besremi is in urgent need of reimbursement.” Besremi is recommended as a first-line or second-line treatment for polycythemia vera by the National Comprehensive Cancer Network (NCCN) and European Leukemia Network (ELN) guidelines, regardless of previous treatment experience.
- Company
- 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.
