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- 2026-05-05 11:26:33
- Policy
- Chinese MM drug Xpovio reattempts reimb listing in KOR
- by Lee, Tak-Sun Feb 14, 2024 05:40am
- A new drug for multiple myeloma that was developed by the Chinese pharmaceutical company Antengene is attempting reimbursement listing again in Korea. The drug’s name is Xpovio Tab 20mg (Selinexor). The drug received a non-reimbursement decision at the Drug Reimbursement Evaluation Committee meeting that was held in November last year. According to industry sources on the 8th, Antengene applied for the reimbursement listing of its Xpovio 20mg Tab. to HIRA. The drug was approved in August 2021 for two indications: ▲ for use in combination with dexamethasone for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least 4 prior therapies and whose disease is refractory to at least 2 proteasome inhibitors (PI), at least two immunomodulatory medicinal products (IMiD), and an anti-CD38 monoclonal antibody (mAb); and ▲ as a monotherapy for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL) who have received at least two prior lines of treatment. The company has since applied and filed for reimbursement coverage, but its first reimbursement attempt was thwarted by a non-reimbursement decision at the DREC level in November last year. Established in 2017, Antengene is an anticancer drug specializing company that has received investment from the global pharmaceutical giant BMS Pharmaceuticals. Its headquarters is based in Shanghai, China. In Korea, the company obtained import authorization for the drug in 2021 and appointed Min-Young Kim, former head of Ipsen Korea, as its General Manager. Antengene successfully applied for Xpovio ‘s reimbursement from Canada in August 2022, raising expectations for its reimbursement listing in Korea. Canada is one of the 8 countries that Korea references for new drug reimbursement. The company's success in obtaining reimbursement in the US after Canada raised expectations that it would be able to set an appropriate drug price in Korea, but it failed to cross HIRA’s threshold. However, multiple myeloma patients are calling for its expedited reimbursement, explaining how Xpovio is the only drug available in the fifth line for their treatment. Therefore, the public’s eyes are on whether the authorities will fulfill the dire wishes of the patients and reimburse the drug this time. Meanwhile, the first new drug made by a Chinese pharmaceutical company to be reimbursed in Korea is Beigene Korea's blood cancer drug ‘Brukinsa’. The drug has been reimbursed for Waldenström macroglobulinemia (WM) since May last year.
- Company
- SK Chemical’s pharma biz recorded sales of 376.1 bil. won
- by Chon, Seung-Hyun Feb 14, 2024 05:40am
- SK Chemical's pharmaceutical business, which is to be sold, has reached its sales peak. The company's pharmaceuticals and new drugs under contract have demonstrated stable growth in the prescription market. On the 8th, SK Chemical reported that its pharmaceutical business generated sales of 376.1 won last year, representing an increase of 19.9% YoY. The operating profit for the same period was 31.5 billion won, showing a slight decrease of 6.3% YoY. Last year, the sales of SK Chemical’s pharmaceutical business recorded the highest amount in its history. In Q4 of last year, SK Chemical’s pharmaceutical business generated sales of 105.8 billion won, marking an increase of 32.4% YoY. As a result, the quarterly sales exceeded 100 billion won for the first time. The Quarterly sales of SK Chemical Pharmaceutical business (Left) Operating Profit (Right) trend (Unit: 100 million won, Source: SK Chemical). In the prescription market, SK Chemical’s primary products performed well. According to UBIST, a pharmaceutical market research company, the total prescription sales of ‘Joins,’ an osteoarthritis treatment, was 48.3 billion won last year, reflecting a 2.3% YoY increase. Despite being launched in 2002, Joins, a natural medicine developed by SK Chemical, continues to perform well in the market even after 20 years. The blood circulation improvement drug ‘Ginexin-F’ has generated prescription sales of 26 billion won, marking a 12.3% YoY compared to the prescription volume of the previous year. The prescription sales of Arthritis treatment Feburic in Q2 of last year amounted to 18.7 billion won, an increase by 20.2% YoY. Ultracet, a new drug introduced by SK Chemical, is showing significant growth. In February 2021, SK Chemical signed a domestic sales contract with Janssen to distribute painkiller Ultracet in Korea. The agreement included four products of Ultracet Tab, Ultracet Semi Tab, Ultracet ER Tab, and Ultracet ER Semi Tab. In the last year, Ultracet ER Tab generated outpatient prescription sales of 22.7 billion won, a 13.8% YoY increase. The analysis suggests that the surge in patients with influenza and common cold following Covid-19 endemic increased demand for painkillers, thereby resulting in the rise in prescriptions of Ultracet. SK Chemical is in talks with Korean private equity fund (PEF) Glenwood Private Equity Co (Glenwood PE) to sell its pharmaceutical business. SK Chemical is in the process of selling its pharmaceutical business. In September of last year, the company announced that “We are reviewing terms for selling the pharmaceutical division and we have signed a memorandum of understanding (MoU) before the contract.” The selling price is expected to be around 600 billion won. “We are discussing specific terms,” SK Chemical provided further explanation last month in an official announcement. If SK Chemical divests its pharmaceutical business, it will focus solely on its green chemical business, including copolyesters and functional materials. SK Chemical sold its bioenergy business in 2020. SK Chemical spun off its blood and vaccine businesses into separate entities. In May 2015, the company established SK Plasma to specialize in blood products, followed by SK Bioscience, which focuses on vaccines, in July 2018.
- Company
- Market for Pulmicort and Pulmican soars 2.6 times in 2 years
- by Chon, Seung-Hyun Feb 14, 2024 05:40am
- Last year, the prescription market for asthma medications containing the ‘budesonide’ ingredient expanded to its largest size ever. There have been significant demands for the treatment due to the circumstances surrounding the pandemic and endemic. At the end of last year, the drug pricing increase led to manufacturing and the prescription market expansion. Raising drug prices during shortages yielded a virtuous cycle of resolving the imbalance between supply and demand. According to UBIST, a pharmaceutical market research agency, on the 14th, outpatient prescription sales of asthma medications with a single component of budesonide totaled 9.9 billion won last year, representing an increase of 39.1% compared to the previous year. Budesonide is a medication used to treat asthmatic bronchitis and acute bronchitis in the larynx of infants and young children. There are two available budesonide products in Korea, including AstraZeneca’s Pulmicort and Kuhnil’s Pulmican. The yearly sales of Pulmicort and Pulmican in outpatient prescriptions (Unit: 1 million won, Source: UBIST). Pulmicort and Pulmican prescription sales last year rose 163.1% in two years compared to the 3.8 billion won generated in 2021. In the same period, Pulmicort rose 155.6%, from 2 billion won to 5 billion won, and Pulmican rose 171.3%, from 1.8 billion won to 4.9 billion won. Since the end of 2021, the treatment market for budesonide has expanded significantly due to the rapid increase in the number of COVID-19 patients. There was a higher demand for Asthma medications last year due to an increased number of patients with the common cold or influenza in addition to COVID-19 patients. The surge in demand resulted in an inadequate supply of drugs due to an imbalance between supply and demand. The increase in drug pricing of Pulmicort and Pulmican in the end of last year may have contributed to market expansion. The Ministry of Health and Welfare (MOHW) raised the insurance upper price limit of Pulmican by 18.5% from 946 won to 1,121 won. Pulmicort price rose 12.5% from 1,000 won to 1,125 won. The ministry reached an agreement with pharmaceutical companies to increase drug prices to encourage production expansion. This was to address the issue of drug shortages resulting from rising demand. Pulmicort and Pulmican coproduced prescription sales of 1 billion won in November of last year. After the increase in drug pricing, prescription sales in December rose by 39.4% to 1.4 billion won compared to the previous month. Compared to the 1 billion won in December 2022, the sales rose 36.1% YoY. Pulmicort generated prescription sales of 700 million won in December last year, a 43.3% expansion compared to the previous month and an increase by 76.8% YoY. Pulmican generated prescription sales of 500 million won in November and 700 million won in December, an increase by 35.5%. The monthly sales of Pulmicort and Pulmican in outpatient prescriptions (Unit: 1 million won, Source: UBIST). Prescription volumes for both Pulmicort and Pulmican increased more than the rate of drug price increases. Pharmaceutical companies expanded supply following insurance price raises, and with supply imbalances resolved, the prescription market showed growth rates higher than those of drug price increases. The increase in drug pricing for Pulmican and Pulmicort is the fourth instance of drug pricing adjustments for drugs in short supply. The MOHW raised the insurance upper price limit of 18 items containing acetaminophen 650 mg by up to 76.5% beginning in December 2022. The insurance upper price limit of acetaminophen 650 mg used to be between 43 to 51 won, but it has now increased to 90 won. Since pharmaceutical companies were reluctant to expand production due to the low-profit cost structure, the MOHW decided to raise the prices collectively. The MOHW initially agreed to collectively adjust the prices to 70 won last December and maintain the raised price until March. The MOHW increased the drug pricing of laxatives with magnesium hydroxide as an ingredient. Magmil’s price grew by 27.8%, from 18 won to 23 won. Cho-A’s Marogel price increased from 15 won to 22 won. Sinil Pharm’s M Tab. Sinil saw an increase in price from 16 won to 22 won. In October of last year, the prices of four pseudoephedrine drugs with a single-component were raised by up to 45%. Insurance upper price limit of Shinil Pharmaceutical's Sinil Pseudoephedrine increased from 20 won to 29 won, up by 45%. Samil Pharmaceutical's Sudafed increased from 23 won to 32 won, up by 39%. Sama Pharm's Schdafen and Kolon Pharma's Cosue also saw their insurance drug prices adjusted upwards by more than 30%, from 23 won to 30 won and 31 won, respectively.
- Policy
- Will Enhertu·Ilaris·morning sickness drugs be reimbursed
- by Lee, Tak-Sun Feb 14, 2024 05:40am
- Whether Enhertu Inj 100mg, Ilaris Injection Sol, and the morning sickness drugs that had passed the Health Insurance Review and Assessment Service's Drug Reimbursement Evaluation Committee review on the 1st, will be listed for reimbursement in April is gaining attention. As all three drugs are highly sought-after by patients and the public, the government is trying to get them on the reimbursement list ahead of the 22nd National Assembly election scheduled on April 10. According to industry sources on April 13, the companies for Enhertu Inj 100 mg (trastuzumab deruxtecan), Ilaris Injection Sol (canakinumab), and morning sickness drugs (doxylamine succinate + pyridoxine hydrochloride) I are expected to enter drug pricing negotiations with the National Health Insurance Service soon. Enhertu Inj is used to treat HER2-positive breast cancer and HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. It is considered to be one of the most potent breast cancer drugs ever developed. Ilaris is used for rare diseases such as criophorin-associated periodic fever syndrome, which affects only 13 patients in Korea. Also, morning sickness drugs have been sold off-label at a price of KRW 100,000 per month ever since its launch in 2016. Due to its high price, voices had been rising even among the general public, requesting its reimbursement listing. At the DREC meeting that was held on the 1st, the committee members acknowledged the reimbursement adequacy of Enhertu and Ilaris, and granted conditional approval for the 7 morning sickness drugs including Diclectin Enteric Coated Tab, on their reimbursement adequacy if the companies accept a price below the assessed amount. Accordingly, the NHIS and the companies immediately started the drug pricing negotiation process for Enhertu and Ilaris, and the companies of the morning sickness drugs can also start negotiations if they accept the assessed price within 30 days. The government had expressed determination to expedite the reimbursement listing of these drugs. Also, the National Health Insurance Service had reportedly held preliminary discussions with AstraZeneca to expedite the pricing process. Furthermore, as Enhertu had been reviewed by DREC twice to reach the appropriate drug price level, its pricing negotiations with the NHIS are expected to be conducted relatively smoothly. The company had faced difficulties passing the economic evaluation process for Enhertu, and although the drug’s ICER ICER (incremental cost-effectiveness ratio) value was reportedly slightly higher than the threshold, the government decided to flexibly apply the threshold and accept its reimbursement adequacy. Ilaris is also expected to be reimbursed quickly as the National Assembly and patient organizations are calling for its prompt reimbursement. The NHIS has 60 days to negotiate the drug price, but it aims to reach an agreement sooner and submit it to the Health Insurance Policy Deliberation Committee by late March. Although a price acceptance process remains for the morning sickness drugs, the government's willingness to move forward with the process is raising the possibility of their speedy reimbursement as well. Through the imbuement listing, the health authorities are hoping to create a favorable public opinion for the ruling party before the general election. In response, the Ministry of Health and Welfare is expected to hold a meeting with the 8 morning sickness treatment companies around next week to clarify the government’s stance on their speedy reimbursement. An industry official said, "The fact that the MOHW is in talks with companies even before the NHIS negotiations have started is a positive sign for reimbursement. We believe the government will also hold an open ear to the company’s voices for prompt reimbursement listing.”
- Company
- Unstoppable sales growth of Entresto
- by Kim, Jin-Gu Feb 13, 2024 06:18am
- Entresto. Novartis Korea’s Entresto, a heart failure treatment, repeatedly shows significant growth. Entresto has consistently achieved over 30% growth in sales each year since its release in October 2017, having passed six years. Since its release, Entresto has undergone five price reductions to suppress the steep increase in prescription sales. However, as Entresto’s simultaneous reimbursement range expanded, efforts to suppress sales were ineffective. Prescription sales of Entresto exceeds 50 billion won. The drug has shown a steep growth of more than 30% annually IQVIA, a drug market research agency, reported on the 8th that Entresto’s outpatient prescription sales reached 57.5 billion won last year, showing a 35% year-over-year (YoY) increase from 42.5 billion won in 2022. Entresto is a new class of drugs called angiotensin receptor neprilysin inhibitor (ARNI), a combination of Valsartan, an angiotensin II receptor blocker (ARB) inhibitor, and Sacubitril, a neprilysin inhibitor. After receiving reimbursement approval, Entresto was released in October 2017. The annual prescription performance of Entresto (Unit 100 million won, Source: UBIST). After its release, Entresto continued to experience rapid growth. Entresto’s prescription sales grew from 5.5 billion won in 2018 to 14.3 billion won the following year, a 2.6-fold increase. Despite the exacerbation of Covid-19 in 2020, Entresto continue to experience an upward trend in sales. Annual prescription sales increased by more than 30%, from 20 billion won in 2020 to 30 billion won in 2021 and further to 40 billion won in 2022. Last year, its sales exceeded 50 billion won and are now approaching 60 billion won. Entresto underwent pricing reductions five times until last year, a 21% ↓ from 2243 won to 1774 won Entresto has undergone five price reductions, including adjustments made through the price-volume agreement and voluntary price reduction. The price of Entresto was initially listed at 2243 won for 500 mg, 1,000 mg, and 2,000 mg in October 2017. It was subsequently reduced by 1.9% reduction to 2,200 won in September 2019, by 7.0% reduction to 2,046 won in June the following year, and by an additional 6.6% reduction to 1,910 won in February 2022. In January 2023, the price was reduced by 6.2% to 1,792 won due to the price-volume agreement. In July of the same year, it was further reduced by 1.0% to 1,774 won due to price adjustments related to expansion of reimbursement. During this period, the total reduction in drug pricing was 20.9%. The trend of Entresto's drug pricing reductions (Source: HIRA). Although it hasn’t been updated to last year’s prescription, Entresto saw two additional price reductions this year. In January last year, it saw a 5% reduction from 1,774 won to 1,690 won due to the volume-price agreement. In January, Novartis voluntarily reduced the price of Entresto by 0.4% to 1,683 won as Luxturna was reimbursement listed. The reduction in drug pricing was nullified by additional expansion applications The analysis suggests that despite consistent decreases in drug prices, there has been a significant increase in prescription volume due to expansions in reimbursements. In 2017, Entresto got reimbursed as a ‘treatment for patients with heart failure with reduced ejection fraction (HFrEF) and decreased heart rate.’ The reimbursement was limited to patients who underwent a combination therapy of standard treatment and stable doses of ACE inhibitor or ARB inhibitor for over four weeks. In March 2022, Entresto was approved as a first-line treatment, making it available for patients who had not previously been treated with ACE inhibitor or ARB inhibitor. In July of the following year, Entresto was approved for prescription to both hospitalized patients and outpatients. There have also been suggestions for the possibility of additional expansion. In 2021, the United States Food and Drug Administration (FDA) approved Entresto for the indication of treating patients with heart failure with preserved ejection fraction (HFpEF). Analysis suggests Korea can expect Entresto’s reimbursement to be expanded to include HFpEF, a condition affecting half of the heart failure patients. Will the upward growth in sales continue? Factors such as generic companies challenging patents and competing drugs affect this trend The pharmaceutical industry anticipates that Entresto will likely continue its upward trend in sales, especially given the possibility of additional expansion. There could be two factors affecting this trend. The first is the outcome of patent challenges by Korean pharmaceutical companies. A patent dispute related to Entresto is awaiting the Supreme Court’s ruling. If the Supreme Court rules in favor of the patent-challenging companies, it could lead to the early release of generic versions. In this case, Entresto’s drug pricing could potentially see a 30% reduction in the first year. Since 2021, companies like Elyson Pharm have filed successive challenges to Entresto patents, with generic companies winning in all first-instance rulings. Novartis has appealed three of these cases. The verdict for one of the appeals was reached in the second instance, ruled in favor of generics as in the first-instance. Subsequently, Novartis has submitted an appeal to the Supreme Court. Entresto's potential competing drugs: Verquvo, Forxiga, and Jardiance. As for Forxiga, AstraZeneca Korea has decided to withdraw Forxiga from the Korean market. Another factor is the emergence of competing drugs. Last September, Bayer released Verquvo as a reimbursed treatment for chronic heart failure. This therapy, containing the active ingredient vericiguat, promotes the synthesis of cyclic guanosine monophosphate within cells, regulating heart contraction, vascular tension, and cardiac remodeling. It represents a novel mechanism for improving myocardial and vascular function. By the end of last year, prescription sales were approximately 20 million won. With approval from major hospital pharmacy committees last year, Bayer expects significant prescriptions starting this year. Additionally, SGLT-2 inhibitor drugs are considered potential competing products for Entresto. Last year, Forxiga (dapagliflozin) and Jardiance (empagliflozin) received approval for the indication of chronic heart failure. However, their reimbursements are currently limited to patients with heart failure accompanied by diabetes. Industry experts anticipate that these two drugs will receive expanded reimbursement for treating patients with chronic heart failure and reduced ejection fraction regardless of having diabetes. The government is analyzing the financial effects related to expanding reimbursement for SGLT-inhibitor drugs.
- Policy
- HIRA to revise guidelines on new drugs utility assessment
- by Lee, Tak-Sun Feb 13, 2024 05:52am
- The Health Insurance Review and Assessment Service (HIRA) will revise the guidelines on indirect comparison for assessing the clinical utility of new drugs. For this purpose, HIRA is currently recruiting researchers for contract research work. On the 1st, the HIRA initiated public bidding for ‘Research services to revise guidelines on indirect comparison for assessing the clinical utility of new drugs.’ “When used as a resource for evaluating clinical utility of new drugs, direct comparison with substitute drugs through randomization are prioritized to assess their efficacy improvements. However, in cases where the submitted product was the sole treatment group in a clinical study or where no direct comparison data with a substitute drug is available, an evaluation basis through a valid indirect comparison becomes necessary,” the HIRA explained. “Based on the findings of the ‘Research on developing the guidelines on indirect comparison for assessing the clinical utility of new drugs (2010),’ the guidelines on the required documentation for new drugs will be formulated. The guidelines will help applicants submit objective effect comparison data for new drugs, and they will also serve as a resource for assessing the suitability and cost-effectiveness of new drugs. However, revisions to domestic guidelines may be necessary to accommodate improved methods, particularly when overseas countries have revised their guidelines,” the HIRA added. Furthermore, once the system updates based on last year’s 'Research on the improvement of the cost-effectiveness evaluation waiver system for drugs (current status and evaluation)’ are implemented, the guideline will serve various uses. According to findings from last year’s research on improving the cost-effectiveness evaluation waiver system for drugs, the existing waiver system will change into a ‘deferred’ system. Drugs that are granted deferred status will have their clinical utility assessed during the post-reimbursement management evaluation. The post-reimbursement evaluation is expected to be important for the drugs that are granted cost-effectiveness deferment. “There are increasing cases of drugs that cannot produce direct comparison clinical data, such as in cases of anti-cancer drugs and orphan drug treatments featuring a single treatment group in clinical research. Consequently, the indirect comparison method is expected to become a valuable source following the revision to the cost-effectiveness evaluation waiver system for drugs,” the HIRA explained. The HIRA plans to sign a contract with external services in February and begin research in March. The deadline for submitting the final report is set for December.
- Policy
- Ulcerative colitis drug Omvoh is approved in Korea
- by Lee, Hye-Kyung Feb 13, 2024 05:51am
- The Ministry of Food and Drug Safety (MFDS, Minister: Yu-Kyoung Oh) announced that it has approved Lilly Korea's new drug Omvoh Inj (mirikizumab-mrkz, recombinant) for the treatment of ulcerative colitis on the 7th. Omvoh Inj. 20 mg/ml is a monoclonal antibody drug that binds to the p19 subunit of interleukin (IL)-23, providing a new therapeutic opportunity for patients with ulcerative colitis by inhibiting interleukin receptor downstream signaling that causes inflammatory responses. Omvoh Inj. is indicated for the treatment of adult patients (aged 18 years or older) with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or biologic treatment. It binds to interleukin (IL)-23, which plays a central role in the pathogenesis of several immune-mediated and chronic inflammatory diseases, including ulcerative colitis, to help maintain resistance to bacterial and viral infections in the gut while supporting clinical improvement of inflammation. The MFDS added, "We will continue to make our best efforts to ensure that treatments with sufficiently verified safety and efficacy are promptly supplied based on regulatory science.”
- Policy
- Janssen’s Spravato required to submit domestic trial data
- by Lee, Hye-Kyung Feb 13, 2024 05:51am
- With Janssen’s novel drug for treatment-resistant depression (TRD), ‘Spravato Nasal Spray (esketamine hydrochloride)’ failed to demonstrate efficacy in Japanese and Chinese patients, the Korean authorities also held discussions on whether to maintain its marketing authorization status in Korea as well. The drug was approved in June 2020. As a result, the authorities decided that no immediate action such as suspension of sales should be taken as no results have shown that the drug was ineffective in Korea. However, the company will be required to submit Phase III clinical trial-level data to further prove its efficacy. For now, the product has been kept on the shelves, but attention will continue to be focused on whether or not it will remain on the shelves by demonstrating its efficacy in Koreans. According to the industry on the 8th, according to the minutes of the Central Pharmaceutical Affairs Council that was released by the Ministry of Food and Drug Safety, the CPAC held an advisory meeting on whether to maintain the marketing authorization (sales) of the medical narcotic drug Stravato and whether it would need to further demonstrate its efficacy. Janssen representatives that had been present at the CPAC meeting, explained “The trials in Chinese and Japanese patients failed due to the lack of statistical significance in the efficacy endpoint. The failure was due to an unexpectedly high placebo effect, which resulted in a lack of significant difference between the Stravato and placebo group, and it is difficult to judge this as a lack of efficacy because the MADRS (Montgomery-Asberg Depression Rating Scale) score did fall with the use of Stravato. In particular, the initial clinical design, which was designed based on FDA guidelines, did not include Korean patients, as Korea does not require data from its own population for approval, so no efficacy data for Koreans are available. Regarding the CPAC member’s question on whether the company can demonstrate Stravato’s efficacy in Koreans at the level of the Phase III clinical trial conducted at the time of initial approval, Janssen said, "The number of subjects had been about 200 to 300, so it may be difficult to recruit the same with Koreans alone, but we are currently working hard on post-marketing surveillance, and we have collected about 70 safety stories and efficacy assessment statements from patients. We will add more number of subjects by the end of the 6-year period." Janssen added that the results of a one-year, single-arm, open-label clinical trial involving 26 Koreans and an investigator-initiated multicenter trial are underway. After Janssen’s explanation, the CPAC chair said, "The CPAC acknowledges that the drug cannot be said to be effective in Japan and China, that is, Asia. We need to decide whether to go through the process of receiving and reviewing the use status, clinical efficacy, and additional supplementation plans through the company or make a decision here and now." After discussions, the CPAC has decided to wait for further clinical data to be submitted rather than make an immediate decision, such as the suspension of sales. In the process, the CPAC concluded that the changes to the labeling information (the drug has not been proven effective in Chinese and Japanese people) should be shared with experts and that it is necessary to receive opinions from doctors organizations, and mental health associations that actually prescribe the drug on-site.
- Company
- ‘Biobetter Nexviazyme is a better treatment option'
- by Eo, Yun-Ho Feb 13, 2024 05:51am
- Professor Hyunjoo Lee There are times when a single treatment has a significant impact on the overall management of the disease. This is especially true for rare and incurable diseases. In the field of metabolic diseases, a paradigm shift occurred when researchers found that certain rare patients lacked a single enzyme, which led to the development of therapies to introduce a substitute into the body to fulfill the enzyme’s role. As a result, patients suffering from these conditions, which are referred to as ‘LSDs (Lysosomal Storage Diseases),’ include Hunter syndrome, Gaucher disease, Fabry disease, and Pompe disease, are currently being treated with enzyme replacement therapy (ERT). Among these diseases, recent advances have been made in the treatment landscape of Pompe disease. About a decade after the ERT drug Myozyme (alglucosidase alfa) was introduced, the biobetter Nexviazyme (avalglucosidase alfa-ngpt) has been granted reimbursement coverage in Korea. Nexviazyme, which was added to the reimbursement list in September last year, became the first drug to receive pricing benefits as a biobetter in Korea. Dailypharm met with Hyunjoo Lee, Professor of Pediatrics at Gangnam Severance Hospital, to learn more about the significance of Nexviazyme’s arrival in Korea. -Nexviazyme can now be prescribed with reimbursement in Korea. What is the benefit of this new treatment option? Pompe disease is a progressive, genetic neuromuscular disease. Despite the many advances in the treatment landscape, there still remained an unmet need as patients were unable to achieve 100% improvement with existing treatments. Nexviazyme was developed to address this unmet need. Nexviazyme is a biobetter drug that was approved for its improved formulation and advancements over the existing Myozyme. It increases intracellular drug uptake by expressing approximately 15 times more mannose 6-phosphate (M6P) on the surface of the therapeutic enzyme than existing therapies. As a result, it can reduce muscle cell damage through effective glycogenolysis by improving glucosidase activity, and it is also beneficial in terms of safety due to improved immunogenicity. -Could you elaborate on the unmet needs that had existed in Korea? Pompe disease is divided into 2 types: infantile-onset Pompe Disease (IOPD), and late-onset Pompe Disease (LOPD), which is characterized by irreversible muscle damage. Myozyme use in patients with infantile-onset Pompe disease has been associated with rapid improvement in heart-related symptoms, such as enlargement of the heart, but there remained room for improvement in muscle-related symptoms, which did not improve 100% and progressed over time. Patients with Pompe disease experience ongoing issues, such as difficulty walking due to arm and leg muscle damage and breathing difficulties due to respiratory muscle damage, but they had limited options as there was only one treatment available for their use. - I understand that forced vital capacity (FVC) and the ability to walk were key endpoints in the Nexviazyme trial. That is correct. In addition to cardiac symptoms, improvements in respiratory and skeletal muscles are important in Pompe disease, to the extent that researchers seek to understand which muscles are most affected. This is most often determined by a six-minute walk test or a forced vital capacity test, but since the walk test cannot be conducted on pediatric patients, symptom improvement in these patients is determined by respiratory and infectious disease susceptibility, and muscle strength for daily activities. The FDA approval in late-onset Pompe disease was granted first for Nexviazyme because of the drug’s effect in improving skeletal muscle, respiratory failure, and muscle-related symptoms. - Are there any risks associated with long-term treatment with Nexviazyme? Because it is an injectable, an infusion-related reaction may occur. Also, regular hospital visits are needed for the injections, which can be challenging. Younger children may have difficulty with vascularization, but there are no other risks. - I remember a few years ago, there was a lot of talk on the need to improve the diagnostic system for genetic diseases such as Pompe disease. What is the current diagnostic landscape like for Pompe disease in Korea? Early diagnosis is important in infantile-onset Pompe disease because of its rapid symptom progression, which can to severe cardiorespiratory disorders and death within a year if the patient does not start treatment immediately. In Korea, diagnostic testing itself is not difficult due to good medical access, and since January this year, lysosomal storage diseases have been included in the neonatal screening test, which means that many patients will be diagnosed more quickly. This is expected to increase the diagnosis rate of lysosomal storage diseases and enable better collection of accurate data about the disease, including the prevalence of Pompe disease patients in Korea. - Lastly, what area do you think needs improvement in the Pompe disease treatment environment? Despite much support from the government, relevant organizations, and pharmaceutical companies, there still are some neglected areas that require attention. For example, in adults, we can evaluate the patient's improvement through FVC and 6-minute walk tests, etc. but it is difficult to do these tests on children. Urine tests are a good option, but these tests are often performed overseas, making it difficult to find out the results immediately. I hope that national or rare disease organizations will recognize this need and create a system to enable efficient testing in Korea. False positives can occur in newborn babies that are born too small or prematurely, depending on their condition. In such cases, I believe the additional tests that are performed at the discretion of the doctor on these patients should also be covered. Also, screening newborns after 28 days of age or those with late-onset Pompe disease is not reimbursed in Korea, and I would like the initial screening process for these patients to be covered so that those with late-onset Pompe disease can be more proactively diagnosed.
- Policy
- Good pharma companies will receive better drug price
- by Lee, Jeong-Hwan Feb 08, 2024 05:49am
- The government decided to further enhance the preferential drug pricing benefits for pharmaceutical companies that have contributed to public health and economic development in Korea, such as by increasing R&D investment in new and incrementally modified drugs, creating many jobs, and supplying essential medicines. The government will promote innovation in the pharmaceutical industry by overhauling the existing system that only provides preferential treatment – a drug price at the highest level among its substitutes - for companies that supply essential drugs or receive a new drug approval for the drug for the first time in Korea. It will also continue expanding insurance coverage through measures such as reflecting the value of innovative new drugs that demonstrated high therapeutic effects in severe and rare diseases in the drug price, and expediting and expanding the number of targets that benefit from health insurance coverage. Furthermore, the government plans to prepare measures for drug price reevaluations, high-priced drug management, and advancing the generic drug pricing structure. These are the contents of the 2nd Comprehensive National Health Insurance Plan (2024-2028) that was released by the Ministry of Health and Welfare on February 4th. Basically, the ministry will strengthen economic support using health insurance finances for pharmaceutical companies that have made achievements in innovating healthcare, health security, and national economic development. The changes were made as part of the government’s efforts to create a system that reflects the growing public and social demand for innovative medical technologies that provide new treatment opportunities for patients with severe and incurable diseases or are more cost-effective than existing technologies. In terms of health security, the government plans to support the establishment of a basic infrastructure to enable the domestic supply of essential medicines that are directly related to the life and health of the people so that the drugs can be stably produced in Korea even if there is a disruption in the overseas supply chain. Pharmaceutical companies that contribute through drug innovation, or drive national development, etc. will benefit from higher drug prices The Ministry of Health and Welfare will improve policies to strengthen access to innovative new drugs. First, it will continue to expand coverage for highly effective severe and rare disease treatments. The order of the reimbursement listing will be prioritized based on disease severity, availability of alternative drugs, superiority of treatment, cost-effectiveness, and financial burden. It will also continue to expedite health insurance listing of new drugs for life-threatening diseases. The approval, evaluation, and negotiation linkage system that has been implemented will reduce the time required for health insurance reimbursement from 330 days to 150 days. To be eligible for the parallel review system, the drug has to have no alternative and be a treatment for a life-threatening disease, demonstrate a significant improvement in clinical effect compared to existing drugs, and be approved for fast-track review by the MFDS. The MOHW will also expand eligibility for expediting listings. Currently, the expedited listing system is being piloted in 2 rare pediatric disease groups - neuroblastoma and hereditary cholestatic stasis – based on which the scope of diseases eligible for fast-track registration will be expanded from 2025. New drugs that are recognized as innovative will be able to receive expedited listing support by recognizing their economic feasibility even if their incremental cost-effectiveness ratio (ICER) exceeds a certain level. The drug pricing premiums provided for each pharmaceutical company will also be expanded. The MOHW believes that it is necessary to calculate drug prices differently based on the company’s degree of contribution to improving public health, health sustainability, and national economic development. Therefore, the ministry will prepare a mechanism that provides preferential drug prices to pharmaceutical companies that have led healthcare innovation and contributed to building a stable supply chain by investing in R&D for new and incrementally modified drugs, supplying essential drugs, and creating jobs. Currently, only companies that supply essential medicines that received a new drug approval for the drug for the first time in Korea receive a preferential price at the highest price level of its alternatives. The government will improve patient access to drugs by expanding risk-sharing system (RSA) treatments for serious diseases that cause significant and irreversible deterioration in quality of life. Reinforce the system for the stable supply of essential medicines In terms of health security, the MOHW will monitor the supply and demand of national essential medicines as well as frequently prescribed drugs such as cold medicines to immediately respond to any abnormalities that may arise. The price of national essential drugs made with domestic ingredients will receive preferential treatment. First, in the case of newly designated national essential medicines, this drug will be given preferential drug prices over other generics when the main ingredient of the generic drug is produced domestically. Drug prices will also be raised quickly to resolve the issue of unstable drug supply. The MOHW is preparing a procedure to quickly raise drug prices of drugs that have become difficult to produce due to rising costs that arose due to their unstable supply and demand after COVID-19. By simplifying the review process based on the criteria for adjusting the insurance price ceiling and concurrently progressing with the NHIS drug pricing negotiations, the MOHW plans to reduce the time required for drug price increases from '210 days+@' to '30 days+@.’ In addition, the MOHW will continue to improve the adequacy of compensation for drug shortage prevention drugs by reflecting manufacturing costs. It will expand the scope of national essential medicines that are eligible for drug price increases and differentiate support to specific classifications. The upper limit for Korean herbal medicine formulations will also be adjusted. Considering the rising cost of APIs and strengthened manufacturing and quality control regulations, the government will consider raising the insurance price ceiling for herbal medicines based on the results of a field survey. Will reduce drug expenditures through drug price reevaluations, management of high-priced drugs, restructuring of generics, etc. The MOHW will establish an integrated price adjustment mechanism for reasonable drug cost management. First, it will prepare a mid to long-term strategic plan to unify the currently fragmented mechanisms for adjusting the price ceiling. Starting this year, the ministry will conduct policy research on establishing a mid- to long-term strategy. The MOHW will also review patent-expired drugs and adjust the price if their domestic price is higher than their highest price in foreign countries. In the case of reevaluating patent-expired drugs, the government plans to start with chronic disease drugs that have a large number of generics. However, drugs that require a stable supply, such as antidepressants, will not be subject to reevaluations. In addition, the management of high-priced drugs for serious diseases will be strengthened. To reduce the patient’s burden of medical expenses as well as the government’s health insurance finances, various types of risk-sharing systems, including outcome-based reimbursement, will be applied to newly listed drugs. The MOHW will also strengthen follow-up management of high-priced drugs for serious diseases based on patient safety and medical effects. It will also rationalize the price-volume agreement system, improving such as increasing the price cut rate for drugs that have a high claims amount or expanding the subjects that can be excluded from being subject to PVA.
