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- 2026-04-04 16:37:08
- Policy
- Cushing’s syndrome drug Isturisa is approved in Korea
- by Lee, Hye-Kyung Sep 20, 2024 05:51am
- Various doses of ‘Isturisa’ (osilodrostat), a treatment for Cushing's syndrome, are expected to be approved in Korea soon. According to industry sources on the 19th, the Ministry of Food and Drug Safety concluded the safety and efficacy review of Recordati Korea's Isturisa Film Coated Tab 1mg earlier this month and recently completed the safety and efficacy review of the 5mg dose as well. Isturisa was previously approved by the US Food and Drug Administration (FDA) on March 6, 2020, the European Medicines Agency (EMA) on January 9, 2020, and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on March 23, 2021. Among the three dosage forms available – 1, 5, and 10 mg – the MFDS has completed a safety and efficacy review for the 1 and 5 mg doses. Isturisa has been under review for approval in Korea since November last year after being designated as a Global Innovative products on Fast Track (GIFT) by the MFDS. As the safety and efficacy review is the final step in the approval process, the drug’s approval is expected soon. Isturisa inhibits CYP1181, an enzyme responsible for the final step of the cortisol pathway in the adrenal glands, and was designated as a GIFT product in April 2022, when Novartis' ‘Signifor Inj’ was withdrawn from the market and the only existing treatment option was eliminated. The drug is indicated for the treatment of adult patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative and is taken orally twice a day. Cushing's syndrome occurs when the body is exposed to excessive amounts of a hormone called glucocorticoids. Glucocorticoids are produced by an endocrine organ called the adrenal glands, which are located on top of the kidneys and can occur when the adrenal glands produce more glucocorticoids than necessary, or when excessive amounts of glucocorticoids enter the body from the outside for various reasons. Cushing's syndrome is characterized by a rounded, moon-shaped face and excessive subcutaneous fat deposits on the back of the neck and shoulders, and is most common in adults between the ages of 30 and 50. It affects women three times more often than men. It can cause health issues such as hypertension, obesity, type 2 diabetes, blood clots in the legs and feet, fractures, a weakened immune system, and depression, and can be diagnosed through a blood test and a 24-hour urine test. Isturisa is also the first FDA-approved drug to work directly on cortisol overproduction by blocking an enzyme known as 11-beta-hydroxylase and preventing cortisol synthesis. The safety and effectiveness of Isturisa were demonstrated in a clinical study involving 137 adult patients, where approximately half of the patients reached cortisol levels within normal limits at Week 24. After Week 24, 71 patients who did not require further dose escalation and tolerated the drug participated in an 8-week double-blind, randomized withdrawal study in which withdrawals were treated with either Isturisa or placebo. Results showed that 86% of the patients in the Isturisa arm maintained cortisol levels within normal limits. On the other hand, only 30% of the placebo arm reached the same result.
- Company
- Samsung Bioepis recruits Lina Choi as new global head
- by Nho, Byung Chul Sep 19, 2024 05:48am
- Lina Choi named as new global head for Samsung Bioepis. Samsung Bioepis garners attention as the company recruits specialists to strengthen its global entry capacity. Sources said that Samsung Bioepis has recruited Linda Choi, Vice President, as the Head of Global Commercial in August. Choi studied pharmacy at Ohio State University College of Pharmacy and has 28 years of experience in multinational pharmaceutical companies. She is recognized as a specialist with expertise in sales and marketing. Choi started a career at Eli Lily in the United States, where she served as Senior Director. At UCB Biopharma in Belgium, Choi was the chief operating officer for global marketing. Before joining Samsung Bioepis, Choi also served as the Chief Operating Officer (COO) at AVANIR, Japan's Otsuka Holdings subsidiary, and ADESIS, a U.S.-based CDMO. By recruiting Choi, who has years of experience in the global biopharmaceutical industry, Samsung Bioepis is expected to further strengthen biosimilar product sales in the global market. Samsung Bioepis has commercialized 9 biosimilar products in immunology, oncology, ophthalmology, and hematology. Partnering with Biogen, Organon, and Sandoz, Samsung Bioepis sells 8 products in Europe and 4 in the United States. In addition to partnership deals, Samsung Bioepis focuses on establishing an independent sales network overseas. For 'Episcli,' a rare disease drug launched in Europe in July 2023, Samsung Bioepis' local subsidiary was responsible for direct sales. Samsung Bioepis has become the company with the most FDA-approved biosimilar antibody products after obtaining eight marketing authorizations from the U.S. FDA. The company expects to launch Stelara biosimilar (U.S. product name, 'Pyzchiva') in the United States. Samsung Bioepis plans to expand its product portfolio for various diseases in the global market.
- Opinion
- [Reporter’s View] The new drug approval fee hike
- by Lee, Hye-Kyung Sep 19, 2024 05:48am
- The Ministry of Food and Drug Safety has announced an amendment to the “Fee Regulations for the Approval of Drugs, etc.” that will significantly increase the fee for new drug approvals from KRW 8.83 million to KRW 410 million. This is the first time in 4 years that the government has decided to increase the new drug approval fee, which is in line with the previous four-year cycle fee hike. In 2016, the fee was raised from KRW 6.82 million to KRW 8.83 million in 2020, and this time in 2024, another 4 years later, the fee is being raised to KRW 410 million. The MFDS’s fee hike announcement was received with surprise in the industry. The MFDS had been conducting a study to increase the fee for new drug approvals, so there had been a widespread assumption that the fee would be increased within the year. However, few thought that the increase, which had been 30% four years ago, would soar to 4500% and exceed KRW 400 million. No one expected the MFDS to raise the drug approval fee to a level similar to Europe and Japan at once. Most had expected the government to raise the fee in phases as before. When considering the fees charged by advanced regulatory agencies abroad, Korea's current KRW 8.83 million is unreasonable, to say the least. Even the industry acknowledged this. In Japan alone, which has a similar GDP per capita level, the PMDA KRW 430 million, the European EMA KRW 490 million, and the Canadian HC KRW 550 million. The US FDA charges KRW 5.3 billion, therefore, the MFDS’s administrative notice of raising the fee to 410 million is appropriate to align the fee to a realistic price at the global level. In particular, it has been pointed out the MFDS’s new drug approval fee is much lower than that of advanced regulatory agencies overseas, leading global pharmaceutical companies to apply for approval in a ‘testing the water’ type of manner. Before applying for approval to the FDA or EMA, the companies first apply to the MFDS, and then add the supplementary data requested by the MFDS before applying to regulatory agencies abroad. There has also been a feeling that MFDS is being regarded as a ‘consultant’ for overseas regulators. The damage caused by the understaffed reviewers reviewing the data of false applications was the delay in review, which was ultimately borne by domestic pharmaceutical companies that needed new drug approvals. The increase in the new drug approval fee seems to mean that MFDS will no longer act as a ‘consultant’ and fulfill its purpose as a proper regulator. Moreover, it seems like the government decided to foster the development of new drugs by domestic pharmaceutical companies rather than global pharmaceutical companies as the new amendment specifies the benefits provided to such by the MFDS, such as a 50% reduction in fees for domestic pharmaceutical companies and a 90% reduction in fees for modifications made to existing drugs. Although the new drug approval fee seems to have suddenly risen to KRW 410 million, this was an inevitable raise that had to be made to meet the global level sooner or later. The MFDS announced that the new drug approval period will be shortened from 420 days to 295 days with this increase. If it goes as planned, new drugs developed by domestic pharmaceutical companies will be able to enter the market a little faster. However, it would be necessary to expand the number of expert staff to accommodate this. The MFDS has set aside half of the increased fees for labor costs. The amount may seem like a lot, but it will be used as a basis for expanding the number of reviewers. If the increase in the number of expert staff can actually shorten the time it takes to approve a new drug, the fee increase would have been necessary sooner or later.
- Policy
- MFDS approves Hemgenix for Hemophilia B in Korea
- by Lee, Hye-Kyung Sep 19, 2024 05:48am
- The Ministry of Food and Drug Safety (MFDS) announced on the 13th that it has approved the orphan drug ‘Hemgenix Inj (etranacogene dezaparvovec)’ imported by CSL Behring Korea. Hemgenix is used to treat moderate-to-severe hemophilia B (congenital blood clotting factor IX deficiency) in adults without a history of Factor IX inhibitors. The drug is expected to provide new treatment opportunities for hemophilia B patients by offering a long-term, single-shot treatment option, unlike existing treatments. The MFDS explained that the quality, safety and effectiveness, manufacturing and quality control standards of Hemgenixwere thoroughly reviewed and evaluated scientifically in accordance with the review criteria outlined in the “Act on The Safety of and Support for Advanced Regenerative Medicine and Advanced Biological Products (“Advanced Regenerative Bio Act”),” which enabled the product’s expedited review and prompt introduction into the field. Hemgenix will be subject to long-term follow-up under Article 30 of the Advanced Regenerative Bio Act, which requires pharmaceutical companies to follow up on the occurrence of adverse events for 15 years from the date of administration. The MFDS said, "Based on our expertise in regulatory science, we will continue to make our best efforts to ensure that novel therapies for severe and rare diseases are promptly supplied to patients in Korea.”
- Company
- Will Columvi be reviewed for reimb by CDDC in OCT?
- by Eo, Yun-Ho Sep 19, 2024 05:48am
- Demand is rising for the reimbursement of ‘Columvi,’ the first bispecific antibody treatment option for lymphoma, in Korea. The Korea Leukemia Patients Organization has requested the CD20-CD3 bispecific antibody for diffuse large B-cell lymphoma (DLBCL) Columvi (glofitamab) be redeliberated at the Health Insurance Review and Assessment Service's Cancer Disease Deliberation Committee meeting that will be held in October. In addition, the organization urged the manufacturer, Roche Korea, to come up with a financial sharing plan to accelerate the drug’s reimbursement listing. “Columvi is a third-line treatment that can be immediately used in situations where death can occur within a few months,” said KIPO, adding, “Patients with diffuse large B-cell lymphoma who have failed two or more lines of treatment strategies are sincerely awaiting Columvi’s prompt health insurance reimbursement listing.” The drug was presented for deliberation to the CDDC last July but failed to make the cut for reimbursement. However, Roche recently submitted an application again for its reimbursement. It remains to be seen if the hopes of the medical community and patient groups for Columvi’s reimbursement listing will be fulfilled this time. Columvi was approved in Korea last December for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL), after two or more lines of systemic therapy. The drug is a third-line treatment option for DLBCL, like Novartis’s chimeric antigen receptor (CAR)-T-cell therapy Kymriah (tisagenlecleucel). The two drugs have different benefits; therefore the choice will likely be based on each patient's condition and circumstance. Columvi demonstrated efficacy in Phase I/II NP30179 trial in 155 patients with relapsed or refractory DLBCL after two or more prior systemic therapies. Results showed that Columvi achieved a complete response (CR) of 40% and an overall response rate(ORR) of 52%. The efficacy was also consistent across all subgroups. The most common adverse event was cytokine release syndrome (CRS). Adding to the encouraging data, at the 2024 Congress of the European Hematology Association (EHA 2024), the company unveiled the results of the Phase III STARGLO study, which demonstrated an improvement in overall survival (OS) with Columvi. The STARGLO study enrolled patients with relapsed or refractory (R/R) diffuse DLBCL who were not eligible to receive an autologous stem cell transplant after one or more prior systemic therapies, or who had received two or more prior systemic therapies. In the primary analysis (median follow-up 11.3 months), Columvi and gemcitabine+oxaliplatin (GemOx) combination significantly improved the primary endpoint of OS with a 41% lower risk of death compared to rituximab+GemOx. Seok Jin Kim, Professor of Hematology and Oncology at Samsung Medical Center, said, "There had been much unmet need in DLBCL for more effective third-line treatment options for patients who fail first-line or experience repeated relapses. We expect the introduction of Columvi to significantly improve the outcomes for patients with relapsed or refractory lymphoma in Korea." Although the drug is non-reimbursed, the drug can be prescribed in various general hospitals in Korea. The drug has passed the drug committees (DCs) of Samsung Medical Center, Seoul Asan Medical Center, Sinchon Severance Hospital, Seoul National University Bundang Hospital, Seoul National University Hospital, Ulsan University Hospital, Korea University Anam Hospital, Wonju Severance Christian Hospital, and Chonnam National University Hwasun Hospital.
- Company
- Reimb for orphan drug 'Welireg' for cancer has been denied
- by Eo, Yun-Ho Sep 19, 2024 05:48am
- An orphan drug, 'Welireg,' for cancer still faces challenges in entering insurance reimbursement coverage. Sources said that MSD Korea is preparing to reapply for reimbursement review of its oral hypoxia-inducible factor 2 alpha (HIF-2α) inhibitor, Welireg (belzutifan), after failing to pass the Cancer Disease Review Committee (CDRC) review last month. MSD Korea filed a reimbursement application in April, and the issue was posted on a national petition platform in May. The petition had received over 50,000 signatures, but the drug did not pass the hurdle of the CDRC review. The analysis suggests that finance will be a determining factor for the outcome. Welireg received an orphan drug designation in South Korea last year for its rare indication to treat von Hippel-Lindau (VHL) disease. It won final approval in May last year. The drug is indicated to treat adult patients with VHL disease who require therapy for associated renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery. The drug has a mechanism of reducing cell proliferation, new blood vessel growth, and tumor growth-associated HIF-2α targeting gene transcription and expression. The efficacy of Welireg was demonstrated through an open-label Study-004 clinical trial, which involved 61 patients with VHL-associated RCC who had at least one measurable solid tumor localized to the kidney. Enrolled patients had other VHL-associated tumors, including CNS hemangioblastomas and/or pNET. The primary endpoint for the clinical trial was an objective response rate (ORR), evaluated radiologically by independent central review according to RECIST v1.1. Additional efficacy endpoints included duration of response (DoR) and time to response (TTR). The results showed that the ORR for patients with VHL-associated RCC treated with Welireg was 49%. All other responses were partial responses. The treatment has yet to reach the median for DoR. The percentage of patients who continued responding after 12 months was 56%. The median TTR was 8 months. In 24 patients with VHL-associated CNS hemangioblastomas, the ORR was 63%. This group had a 4% complete response rate and a 58% partial response rate. Recently, Welireg also added an indication to treat RCC in the United States. The efficacy of the drug was demonstrated through the LITESPARK-005 clinical trial, which involved advanced RCC that has progressed following PD-1 and/or PD-L1 inhibitor or VEGF-TKI therapies. Based on the results, Welireg improved progression-free survival (PFS) compared to everolimus in patients with RCC that has progressed following PD-1 or PD-L1 immune checkpoint inhibitor or VEGF receptor targeting therapies. The drug also reduced the disease progression and death risk by 25%.
- Opinion
- [Reporter’s View] Benefits of the U.S. Biosecure Act
- by Son, Hyung Min Sep 13, 2024 05:50am
- The US House of Representatives recently passed the Biosecure Act, which includes the US’s biological sanctions against China. The bill will now head to the US Senate for voting and go through a presidential review process for legislation. The Biosecure Act restricts transactions with major Chinese biotech companies that the U.S. Congress deems to pose a threat to national security. The US Congress has categorized the biotechs into 3 groups, A, B, and C. Group A includes 5 companies: BGI, MGI, and Complete Genomics, which are genomic equipment manufacturers and analytical services, and Wuxi AppTec and Wuxi Biologics, which are contract development and manufacturing organizations (CDMOs). Group B includes companies that are under the control of a foreign hostile government, provide equipment or services to companies on the list of biotechnology companies of concern, or pose a risk to U.S. national security, and Group C includes subsidiaries and parent companies associated with companies in Groups A and B that are under the control of a foreign hostile government. The Biosecure Act will be implemented after a grace period until January 2032, but already expectations are rising on how Korean companies can benefit from the Act. In particular, the inclusion of Wuxi Biologics, China's largest CDMO, in the sanctions has raised attention to the opportunity it brings to other domestic CDMOs such as Samsung Biologics, Celltrion, and ST Pharm. Wuxi Biologics is present in China, the US, Ireland, Germany, and Singapore, and employs more than 12,000 people. The company occupied 10% of the global CDMO market last year. In fact, ST Pharm is believed to have benefited from the proposal of the Biosecure Act, signing contracts previously made with China. Last month, the company was also selected to supply a small molecule blockbuster drug that generates trillions of wons in annual sales. However, we cannot have blind faith that domestic companies will reap all the benefits. The market is dominated by leading global CDMOs such as Switzerland's Lonza, the US's Catalent, and Japan's Fujifilm, all of which have their eyes on Wuxi Biologics' pie. Their competitive advantage is quality. Based on their years of experience in signing contracts with global pharmaceutical companies, the companies have been expanding their market. Global CDMOs have strong technical capabilities in early research, manufacturing, and commercial finishing processes. Indian companies that own price competitiveness are also emerging as viable competitors. Currently, India has more than 15,000 drug manufacturing facilities owned by around 3,000 pharmaceutical companies. Of these, it is estimated that at least 100 of which are specialized CDMOs. India has 40% lower drug manufacturing costs than the US and Europe, and has the highest number of US Food and Drug Administration (FDA)-approved pharmaceutical plants outside the US. In other words, to survive, domestic companies need to secure production capacity, quality, and technology, not settle for just a slice of Wuxi Biologics' pie. Global CDMOs have invested in cell gene therapy (CGT) and antibody-drug conjugates (ADCs) to win orders. Currently, domestic CDMOs are still in the investment stage for ADCs and CGTs. In addition, there is also a possibility that the U.S. will extend the protection of its industry and sanction more companies. In 2022, the U.S. administration has implied restricting overseas companies by proposing an executive order to increase domestic production of biopharmaceuticals. Policies aimed at increasing domestic production and protecting its own biotech sector are bound to be a blow to companies looking to enter the US. In this sense, it is not impossible that the knife pointed at China could be extended to other countries. This is where the government’s support comes into play. While the current government has signaled that it sees the bio-industry as a future growth engine and is committed to implementing relevant policies, there is a consensus that the industry has not felt the impact so far. Experts believe that the government needs to continue to support the industry by increasing the scale of government support and providing tax incentives. In the current state, The US Biosecure Act is clearly an opportunity for domestic companies. If the companies continue to seek growth based on their investment and technological capabilities and are supported by the government, they will be able to enter the global market.
- Company
- JAK inhibitor 'Jyseleca' approved for use at 'Big 5'
- by Eo, Yun-Ho Sep 13, 2024 05:50am
- Product photo of Jyseleca. The JAK inhibitor 'Jyseleca' has been successfully introduced to general hospitals. Sources said Jyseleca, the fifth JAK inhibitor in South Korea, has passed the drug committees (DC) of 'Big 5' tertiary hospitals, including Samsung Medical Center, Seoul University Hospital, Seoul St. Mary's Hospital, Seoul Asan Medical Center, and Sinchon Severance Hospital. The drug also passed DC of medical centers, including Kangbuk Samsung Hospital, Korea University Anam Hospital, Cha University Bundang Medical Center, Sejong Chungnam National University Hospital, Ulsan University Hospital, Konkuk University Hospital Chungju Hospital, and Hanyang University Medical Center, as well as national university hospitals in major cities. Considering that it is the fifth approved JAK inhibitor in South Korea, it has quickly become available for prescription at hospitals following reimbursement listing in November last year. Jyseleca’s initial indication for reimbursement was for the treatment of rheumatoid arthritis and moderately to severely active ulcerative colitis. Its reimbursement criteria are set for individuals who have had an inadequate response to conventional therapies or have no drug tolerance to each disease. For those who are over 65 years old, the criteria are set for individuals who have had an inadequate response to TNF-α inhibitors or have no drug tolerance. In South Korea, JAK inhibitors, such as 'Xeljanz (tofacitinib),' 'Olumiant (baricitinib),' and 'Rinvoq (upadacitinib),' are being prescribed. It is to be watched whether Jyseleca would have a competitive advantage over these drugs. Since their launch, these drugs have been expanding indications and reimbursement criteria. Xeljanz additionally secured indications for ulcerative colitis and psoriatic arthritis, and latecomers, such as Rinvoq, are also expanding prescription areas in autoimmune diseases, including atopic dermatitis, Chron’s disease, and ankylosing spondylitis. Since their launch, these drugs have been expanding indications and reimbursement criteria. Xeljanz additionally secured indications for ulcerative colitis and psoriatic arthritis, and latecomers, such as Rinvoq, are also expanding prescription areas in autoimmune diseases, including atopic dermatitis, Chron’s disease, and ankylosing spondylitis. Consequently, patients are expected to have improved treatment options for rheumatoid arthritis. Of course, the use of JAK inhibitors, including Jyseleca, will substantially increase. According to the market research firm UBIST, the JAK inhibitor outpatient prescription market size for the first half of 2023 was KRW 27.5 billion, up by 54% from KRW 17.8 billion year over year. Meanwhile, Jyseleca is a selective ATP-competitive and reversible JAK1 inhibitor. JAK1 transmits signals from a cytokine, and it is regarded as the key target for the treatment of rheumatoid arthritis. Recently launched treatments inhibit JAK2 or JAK3, depending on their mechanism. However, there are concerns that adverse reactions may occur, as two signaling pathways regulate immune cell proliferation and homeostasis. The FINCH1, FINCH2, and FINCH3 Phase 3 trials have demonstrated the effectiveness of Jyseleca. In the FINCH1 trial, Jyseleca 200 mg treatment in patients with moderately to severely active rheumatoid arthritis reached ACR20 at 20 weeks more quickly despite continued treatment with MTX.
- Policy
- Moderna’s COVID-19 vaccine is approved in Korea
- by Lee, Hye-Kyung Sep 13, 2024 05:50am
- A new COVID-19 variant vaccine, which is the only mRNA-based COVID-19 vaccine that will be manufactured domestically (by Samsung Biologics) has been approved in Korea. The Ministry of Food and Drug Safety (MFDS, Minister: Yu-Kyoung Oh) announced on the 11th that it has authorized the manufacturing and sale of ‘Spikevax JN.1’ that Moderna Korea applied for. Like Pfizer's COVID-19 vaccine, which was approved on August 30, Spikevax JN.1 is a vaccine that contains an mRNA designed to express the JN.1 variant antigen as the active ingredient. It is indicated for the prevention of COVID-19 in persons 12 years of age and older and is administered as a single intramuscular injection of 0.5mL. Those who have previously received a COVID-19 vaccine should administer Spikevax JN.1 at least 3 months after his or her most recent COVID-19 vaccination. Samsung Biologics will receive the active pharmaceutical ingredient supplied by Moderna and manufacture the finished drug through filling and labeling, and its domestic manufacture is expected to ensure a smooth supply of the vaccine. The Ministry of Food and Drug Safety said, ‘While strictly examining the safety, effectiveness, and quality of this vaccine, we worked to promptly approve the drug in 2 months so that there would be no disruption to the National Immunization Program for high-risk groups that are scheduled to begin next month.”
- InterView
- ‘Era of once-weekly growth hormones…adherence is key’
- by Hwang, Byung-woo Sep 13, 2024 05:49am
- "The frequency of drug administration is an important factor in the treatment of growth hormone deficiency. The emergence of a once-weekly dosing option is significant in terms of treatment adherence.’ With the market for growth hormone injections rising rapidly, the diversified treatment options have been increasing market interest. The introduction of once-weekly treatment options to the once-daily regimen has emphasized the importance of choosing the right treatment option for patients. Professor Masanobu Kawai (Osaka Women's and Children's Hospital( focused on the effect of the once-weekly growth hormone deficiency treatment. Professor Masanobu Kawai, Osaka Women Growth hormone deficiency is a condition in which the pituitary gland, which is responsible for the secretion of hormones in the body, does not produce enough growth hormone, and is defined as a serum peak growth hormone concentration of less than 10 ng/mL in two or more growth hormone provocation tests. According to the Health Insurance Review and Assessment Service, the number of Korean patients under the age of 19 diagnosed with growth hormone deficiency has nearly doubled over the past 5 years, from 9,746 in 2019 to 19,363 in 2023. The main treatment method is the administration of growth hormone therapy, and once-daily administration has been the main option. In September last year, Pfizer's once-weekly growth hormone deficiency treatment Ngenla (somatrogon) was approved for reimbursement, expanding the options available. Professor Masanobu, who has more experience prescribing Angela as the drug was first released in Japan than in Korea, explained that Ngenla offers a big difference in terms of treatment adherence. Professor Masanobu said, “As a once-daily growth hormone therapy, Ngenla’s approved dose is higher in Korea than in Japan. In Japan, we had concerns about the efficacy of the approved daily treatments due to their low dose, but the once-weekly treatments with higher doses have shown a clear effect.” He added, “Ngenla has strengths in terms of treatment adherence, which is an important factor in growth hormone therapy. Patients with growth hormone deficiency are typically prescribed every 3 months, and patients who are prescribed daily growth hormone therapy have more injections left over at the time of their visit.’ This means that the convenience of a once-weekly treatment, compared to a daily treatment, leads to higher adherence. This convenience is expected to reduce the burden of administration on patients and caregivers. In fact, according to a survey on growth hormone deficiency patients and their parents in Japan, 44% responded that the frequency of dosing was the most important aspect they consider when using growth hormone therapy. “This finding, which was identified before the introduction of Ngenla, demonstrates the inconvenience patients felt due to the frequent administration of existing therapies. A once-weekly dosing regimen is convenient and could ease the burden of treatment for patients and their parents,” explained Professor Masanobu The hurdle of the once-weekly dose is ‘pain’...‘Most patients would prefer less frequent dosing’ However, there is also a hurdle for once-weekly treatments. While they are less burdensome to administer than their competitors that require daily injections, there is still the issue of pain management that follows the use of injectables. On this, Professor Masanobu said that the long-acting injectables may bring a higher pain burden due to the higher dose, but this can be addressed by adjusting the injection site. “Once-daily treatments use thin, short needles, but Ngenla uses thicker, longer needles because of the higher dose. If the needle is injected deep enough to reach the subcutis, the injection pain can be absorbed by the surrounding tissues, reducing the burden.’ However, South Korea's approach to pain management differs from Japan's in that it uses a single injection. Nevertheless, Professor Masanobu believed that Ngenla’s less frequent administration is likely to be preferred by most patients other than the younger patients for whom pain management is a priority. In addition, Professor Masanobu believes that the emergence of a once-weekly dosing option for growth hormone deficiency treatment could lead to improvements in the patient and parents’ quality of life (QOL) beyond therapeutic effect. Professor Masanobu said, “In terms of efficacy, the once-daily and once-weekly doses may have similar effects, however, the burden of daily dosing can affect a person's life in general. Considering the pressure felt by patients and parents, as well as treatment adherence, the ability to alleviate this burden is one of Ngenla’s greatest advantages.’
