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- 2025-12-19 04:43:35
- Policy
- Directly injected gene scissor therapy receives PT3 approval
- by Lee, Hye-Kyung Oct 25, 2024 05:49am
- An in vivo gene scissor therapy that is injected directly into the body will enter Phase III clinical trials in Korea. On the 23rd, the Ministry of Food and Drug Safety (MFDS) approved a Phase III clinical trial to evaluate the efficacy and safety of ‘NTLA-2001’ in participants suffering from Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM). 'NTLA-2001,’ which is an investigational new drug by U.S. Intellia Therapeutics, works by delivering the ‘guide RNA’ that guides the gene editing ‘Cas9 mRNA’ to the target gene in a Lipid Nano Particle (LNP) to liver cells through intravenous injection to eliminate the target gene in the liver. LNPs basically bind to ApoE in the blood and enter the liver mainly through ApoE receptors on the surface of liver cells, but there have been problems with toxicity due to excessive accumulation. However, NTLA-2001 offers enhanced biodegradability by incorporating ester links into its lipid structure and achieved a half-life of one-quarter that of conventional LNPs in animal studies. NTLA-2001 has achieved positive results in a global Phase I study as a treatment for hereditary transthyretin amyloidosis (hATTR) and is on track to become the first-in-class Crispr-based therapy. ATTR is a disease in which mutations in the transthyretin gene cause the liver to produce misfolded transthyretin protein, leading to neurological damage and heart muscle abnormalities. There are reportedly around 50,000 patients worldwide, and with an average life expectancy of 2-15 years after symptom onset, there is much interest in its cure. Meanwhile, in April 2016, the US-based Regeneron signed a collaboration agreement with Intellia Therapeutics to jointly develop next-generation gene therapies using CRISPR gene editing technology. Under the agreement, Intellia received an upfront payment of USD 75 million (KRW 104.6 billion) and is eligible to receive additional payments based on future performance. In 2020, Regeneron secured the rights to commercialize NTLA-2001 through an additional USD 100 million (KRW 139.5 billion) license agreement. Current U.S. FDA-approved treatments for ATTR include Pfizer's Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis), Alnylam’s Onpattro (patisiran) and Amvuttra (vutrisiran), and Ionis' Tegsedi (inotersen).
- Policy
- Reimb discussions restart for BMS’s Camzyos in KOR
- by Lee, Tak-Sun Oct 25, 2024 05:48am
- The National Health Insurance Service was found to have restarted reimbursement discussions for the obstructive hypertrophic cardiomyopathy treatment Camzyos (mavacamten, BMS). BMS and the NHIS entered into drug price negotiations for Camzyos in August but failed to reach an agreement within the 60-day deadline. However, the deadline has been extended, and it is expected that the company may be able to list the drug for reimbursement as early as next month. In particular, there are opinions that the criticisms made during the NA audit may have accelerated the drug’s reimbursement discussions. Lorviqua, whose reimbursement progress was also criticized during the NA Audit, may likely skip the Drug Reimbursement Evaluation Committee review and re-enter into negotiations with the NHIS. According to industry sources on the 24th, during the NA Health and Welfare Committee’s Audit that ended on the 23rd, Democratic Party of Korea Rep. Hee-Seung Park inquired about the Camzyos’s reimbursement progress and requested its prompt reimbursement. Camzyos’s reimbursement has been in the drug price negotiation stage since early August. The deadline for negotiations was 60 days, so an agreement was supposed to be reached in early October. In response to Park's inquiry, the NHISon explained, “Based on DREC’s review results, we are in the process of negotiating with the company to set an appropriate drug price and expected claims amounts, and signing the risk-sharing agreement. However, during the current negotiation process, the pharmaceutical company requested a pause in the negotiation as it needed time to submit additional data, upon which the NHIS accepted the request and extended the negotiation deadline.” In other words, the period needed to submit the additional data was excluded from the negotiation period, which was why the deadline was extended. “In order to ensure access to new drugs for the active treatment of cardiomyopathy patients while minimizing the financial burden, we plan to closely review the financial impact data such as patient size and market share submitted by pharmaceutical companies and clinical literature to reflect the financial impact and clinical value of the drug under negotiation,” said the NHIS. Camzyos had been the focus of recent media coverage, as well as the NA audit, to ensure expedited access for the patients. As a result, payers are also reportedly considering expediting its reimbursement. As such, negotiations have resumed, raising the possibility of the drug being listed as early as next month. However, time is running out to finalize and report the negotiation results to the Health Insurance Policy Review Committee meeting that will be held next week. If not next month, there is still a good chance of reimbursement by December. Also, industry eyes are on whether Lorviqua (lorlatinib, Pfizer), which had failed negotiations with them in June, will enter into negotiations again. Lorviqua is a treatment for ALK (anaplastic lymphoma kinase)-positive NSCLC that was in the process of extending its reimbursement to first-line treatment. Initially, the company submitted an application to extend the drug’s reimbursement standards as an Expenditure Cap type RSA, but after negotiations broke down, the company reapplied for the drug’s reimbursement through the general listing pathway. According to the regular procedure, the drug must be first reviewed by HIRA’s Drug Reimbursement Evaluation Committee to start negotiations with the NHIS, but as it is a special case where the drug is switching its status from RSA to general listing, there is a high possibility that the government will simplify the procedure. during the NA Audit on the 16th of this month, Dong-Kyu Lee, Director General of MOHW’s Bureau of Health Insurance Policy, said, “We are currently in negotiations with the pharmaceutical company,’ and showed a proactive stance, saying, “We will complete negotiation as soon as possible for the benefit of the patients.’ As a result, the industry expects Lorviqua’s reimbursement agenda may skip the DREC stage and restart negotiations with the NHIS.
- Policy
- MFDS says 'no procedural issues' regarding 'Leqembi'
- by Lee, Hye-Kyung Oct 24, 2024 05:52am
- Product photo of Leqembi. The Ministry of Food and Drug Safety (MFDS) stated that there was no procedural issue during the approval of 'Leqembi (lecanemab),' a dementia treatment, that omitted the review by the Central Pharmaceutical Affairs Advisory Committee (hereafter referred to as the "Advisory Committee"). Out of 33 active ingredients of new drugs that received domestic approval since 2023, only 6 active ingredients were reviewed by the Advisory Committee. The final approval of Leqembi was based on its effects, treatment options, and monitoring plans. In answering the questions from media reporters on October 22nd, the MFDS stated that regarding the procedural issue of Leqembi approval, "The specialists in the MFDS had reviewed thoroughly based on science-based expert analysis." The MFDS explained that they had made a comprehensive assessment. In particular, the drug demonstrated a reduction in cognitive impairment (27%) in patients with mild Alzheimer's disease and the potential to provide new treatment option by targeting the removal of the significant cause of the disease (amyloid beta), unlike conventional drugs. Additionally, the drug's company established a stringent monitoring method (such as MRI monitoring) to follow ARIA, a potential side effect of treatment. Leqembi's ARIA has been identified as cerebral edema and microhemorrhage confirmed by MRI imaging test. Also, the MFDS emphasized that they could seek consultation from the Advisory Committee when they decide outside consultation is needed, but it is not a mandatory procedure during the approval process. During the National Assembly's parliamentary audit of the Health and Welfare Committee, held on October 10th, Rep. Jeon Jin-sook, a member of the Democratic Party of Korea, questioned the approval process of a new dementia drug that received a recommendation for non-approval from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) due to side effects. "Although it is a new drug, approval has been granted without seeking expert opinion," Rep. Jeon said. "In the United States, this drug was approved with the required warning label statement in the prescribing information after the FDA's advisory committee meeting. Europe postponed approval," Rep. Jeon explained. In answering this question, the MFDS said, "The Pharmaceutical Affairs Act guides that the Advisory Committee shall be established to respond to inquiries from the Minister of the MFDS, but it does not mandate the Advisory Committee's review." They explained, "We request consultation from the Advisory Committee when we decide outside consultation is necessary, such as making conditional approval (requiring submission of Phase 3 clinical trial data) or marketing authorization·review." The MFDS further added that as part of Leqembi's risk-assessment plan, they will conduct a post-marketing survey (patient registration research, 6 years) and thoroughly monitor the occurrence of ARIA in Korean patients. "In the approval detail, we required management of ARIA by checking ARIA occurrences by MRI testing before and during the drug administration and suspending administration based on disease severity," the MFDS said. "Since ARIA tends to occur in the early course of treatment, we required testing before 1st, 5th, 7th, and 14th drug administration, and the administration will be suspended when the disease severity is found as moderate to severe during the testing." Meanwhile, Leqembi is intravenously administered in a sing-dose every two weeks. It is a new drug known to delay the cognitive impairment due to Alzheimer's disease by 27%. The drug eliminates 'amyloid beta' aggregates, abnormal protein in neurons, and amyloid fibrils.
- Policy
- 'Preferential drug pricing for K-made new drugs' revision
- by Lee, Jeong-Hwan Oct 24, 2024 05:52am
- Rep. Baek Jong-heon It was pointed out that upon receiving criticism that the recently announced amendment plan for drug pricing system lacked provision to provide preferential drug pricing for new drugs made by Korean pharmaceutical companies, the Ministry of Health and Welfare (MOHW) posted an administrative notice detailing amendment to be made for 'Pharmaceutical Approval and Adjustment Criteria.' Previously, the amendment plan had been criticized for making a reverse discrimination against Korean pharmaceutical companies. According to the MOHW's notice, the amendment plan included implementing a 68% drug pricing increase applied to national essential medicines for up to 10 years (5+5 years), preferential drug pricing of new drugs manufactured by pharmaceutical companies with significant R&D contribution, and a double pricing system to support exports. On October 23rd, Rep. Baek Jong-heon, a member of the People Power Party, stated that during the parliamentary audit held on October 8th, he raised an issue with the Health Insurance Review and Assessment Service (HIRA)'s amendment plan for the pharmaceutical pricing system. The criticism concerned that the amendment plan excluded the core clause to benefit Korean pharmaceutical companies. Baek emphasized that the amendment plan could reverse discrimination against Korean pharmaceutical companies because most of the revision details for the drug pricing system favored multinational companies. In response to this criticism, the MOHW's Pharmaceutical Benefits division proceeded with revising the drug pricing system, aiming to reflect the new drug's innovative value and public health security. They have notified an administrative action for 'Pharmaceutical Approval and Adjustment Criteria.' MOHW's plan will be officially announced in 2025 after being reviewed by the Ministry of Government Legislation and evaluated by the Regulatory Reform Committee. Earilier, Baek questioned Cho Kyoo-hong, Minister of Health and Welfare, about the rationale for excluding a measure that provides practical benefits to Korean pharmaceutical companies from the revised plan, despite Korean pharmaceutical companies requesting improvements to the current policy related to new drug development and exports. "As part of the 120th national agenda, the Yoon Suk Yeol government has decided to foster bio-health business as the core export business, aiming to leap as a leading global country in the bio·digital health field," Baek said. "However, the MOHW announced the revised plan without considering practical supporting measures." "MOHW's role is to lead the growth and development of the Korean biopharmaceutical industry and enhance international competitiveness, thereby contributing to the nation's health and welfare improvement," Beak emphasized.
- Policy
- Will non-face-to-face prescriptions of Wegovy be banned?
- by Lee, Jeong-Hwan Oct 24, 2024 05:51am
- Yu-Kyoung Oh, Minister of the Ministry of Food and Drug Safety, said the ministry will discuss the issue of Wegovy, a new obesity drug, being prescribed through non-face-to-face treatment with the Ministry of Health and Welfare. The MOHW Minister Kyoo-hong Cho also responded that he would actively engage in Oh's request for discussions. On the 23rd, Rep. Joo Young Lee, a lawmaker from the Reform Party, asked Oh about the misuse of obesity drugs through non-face-to-face prescriptions. Rep Lee said, ‘The anticipated concerns regarding obesity treatment have become a reality. After the release of Wegovy on the 15th, in addition to the illegal sales and advertisements of the drug online, but also the issue of the drug being prescribed through non-face-to-face treatment platforms even if they are normal or underweight arose. The MFDS’s promotion is not enough, and I believe it is necessary to make it institutionally difficult to make the inappropriate access itself.” “There is a precedent of post-partum contraceptives being excluded from the list of possible prescriptions from non-face-to-face treatment platforms,” said Lee, adding, “Obesity management should start with lifestyle improvement. Adding Wegovy prescriptions to the list of allowed non-face-to-face prescription servrequiresquire more sensitivity and expertise. Bad practices such as using them in smaller doses are arising. Is there a countermeasure?” Oh replied that the ministry would seek measures to address the misuse of Wegovy and limit non-face-to-face treatment prescriptions. “The purchase of Wegovy online is a problem, and the cyber investigation team has been intensively monitoring this for a month,’ Oh said, adding, ’Overseas direct sales are also a concern due to poor temperature control. Since yesterday, we have been cooperating with the Korea Customs Service to block overseas direct sales and purchases of Wegovy.’ “Non-face-to-face treatment prescriptions are also a problem. We sent a letter asking them to refrain from excessively advertising the prescription of obesity drugs,” said Oh, adding, ’We will discuss whether to include prescriptions for non-face-to-face treatment (such as Wigobi) as it is the responsibility of the Ministry of Welfare.’ Minister Cho Kyu-hong also briefly responded to Oh's comment, saying that the MOHW would “cooperate.”
- Policy
- Overseas direct purchases of GLP-1 obesity drugs banned
- by Lee, Hye-Kyung Oct 23, 2024 05:50am
- The government has begun blocking direct purchases of GLP-1 obesity drugs overseas in the wake of the ‘Wegovy’ craze. The Ministry of Food and Drug Safety (Minister: Yu-Kyung Oh) and Korea Customs Service (Comissioner: Kwanghyo Ko) announced on the 22nd that they would block the direct purchase of recently launched GLP-1-based obesity drugs via overseas online platforms and bringing them into Korea. They also said they are actively cracking down on illegal sales and advertisements online and via social media. The MFDS has asked major online shopping malls to set ‘Wegovy,’ ‘Saxenda,’ ‘diet pills,’ and ‘slimming pills’ as prohibited words and requested their cooperation in self-monitoring and regulating the illegal imports. GLP-1-class obesity drugs such as Wegovy must be administered following a doctor’s prescription at a hospital and dispensing and medication guidance of a pharmacist at a pharmacy, according to the set dosage and administration method. If purchased directly from overseas, it is difficult to check the authenticity of the drug due to unclear manufacturing and distribution routes, and if it is an illegal counterfeit product, its safety cannot be guaranteed as it may contain harmful ingredients and is dangerous due to the possibility of misuse. The Ministry of Food and Drug Safety has requested major online shopping malls to prohibit the sales of obesity treatment drugs and conduct self-monitoring and requested the Korea Communications Commission to block access to posts on social media and online shopping malls that illegally promote or advertise sales of such drugs. Since Wegovy was released on the 15th to the 21st of this month, 12 violative posts were detected and taken action against. The Ministry of Food and Drug Safety and the Korea Customs Service will continue to share relevant information to prevent consumer damage caused by illegal direct purchases and advertisement of obesity treatment drugs overseas. They also warned that the obesity drugs sold on online platforms should never be purchased or administered and that they will do their best to strengthen the monitoring system for illegal sales and bait advertising in the future for the safe use of drugs by people in Korea.
- Policy
- New drug expenditure is 13.5% of NHI finance's drug spending
- by Whang, byung-woo Oct 23, 2024 05:49am
- It was found that the total expenditure on new drugs listed in the past six years since the Positive List System policy was implemented amounted to 13.5% of the National Health Insurance pharmaceutical spending, which is the lowest compared to the averages of A8 countries and OECD countries. The Korean Research-based Pharmaceutical Industry Association (KRPIA) announced the research outcome of 'Analysis of pharmaceutical spending on new drugs by disease,' conducted by Seung-Rae Yu, a Professor at Dongduk Women's University's College of Pharmacy, on October 22nd. The research evaluated new drugs listed from 2007 to 2022 when the Positive List System policy was implemented to allocate financial resources to drugs. The document compared the performance to 25 OECD countries besides South Korea. The research aimed to set the analysis period to the past 6 years (2017-2022) and to investigate financial analysis reflecting the outcome of the government's policy implementation to strengthen patient access to new drugs. It researched disease types contributing to patient deaths and compared domestic and overseas new drug expenditures in diseases with the highest financial burden, by analyzing pharmaceutical costs by disease. The percentage of new drug expenditures in South Korea (red), OECD countries (green), and A8 countries (blue). The percentage of new drug expenditure in South Korea Based on the research, the percentage of new drug expenditure in South Korea's total pharmaceutical cost amounted to 13.5%, which was below half of those in A8 countries, 38.0% on average, and in OECD countries, 33.9% on average. The analysis suggests that the figure would be the lowest among 26 countries. The analysis of the trend in the percentage of new drug expenditure from 2017 to 2022 indicates that the differences between Korea and the average for A8 countries grew significantly, amounting to almost a threefold difference in 2022. In particular, the total expenditure on new drugs was merely 15-25% of those in A8 countries, including the U.K., France, Italy, and Canada, which are similar in population and GDP per capita. The research analyzed the financial cost of disease by comparing a weighted index of disease·injury·risk factor, including death·disorder, by country, and new drug expenditure through the analysis of pharmaceutical cost by disease. The percentage of expenditure on new drugs for treating tumors was 46.2% for South Korea, not significantly different from 54.4% in the OECD on average. However, the percentage of expenditure on new drugs for treating cardiovascular diseases was 20.2% for the OECD on average and 2.4% for South Korea. Furthermore, the neurological disease category was 30.1% and 4%, respectively. The respiratory system category was 43% and 6.7%, respectively. They showed that Korea's percentages were merely 1/10th of the OECD countries' average, indicating relatively poor patient access to treatments. In other words, Korea showed significantly lower expenditures in the top-costing disease category, including the cardiovascular system, neurological system, and respiratory system, compared to those of the OECD and A8 countries. The analysis of the trend in the percentage of new drug expenditures in South Korea (red) and the average for A8 countries (blue). The research pointed out that despite a continuous increase in National Health Insurance reimbursement in the past, reimbursement for severe·high cost diseases started to decrease since 2022. The expenditure index of the number of new drugs and pharmaceutical cost by reimbursement category shows that the number of new drugs listed under the economic evaluation exemption program or essential program amounts to merely 11.6% and 3.6%, respectively. These drugs are needed to enhance patient access to severe disease treatments. The percentages of these drugs in the total pharmaceutical cost were 0.6% and 0.3%, which is relatively low. Additionally, the percentage of new drugs listed after undergoing economic evaluation out of the total number of listed new drugs was 26.8%. Excluding drugs in the antitumor category, it is merely 14.5%, indicating that the percentage of listed new drugs that are found to have values for improving clinical effectiveness is relatively small. "After implementing the Positive List System policy, the percentage of pharmaceutical cost in the total medical cost was aimed at about 24%, and it has been maintained. However, it seems that setting the new drug expenditure in the total pharmaceutical cost lacked specific aim and direction," said Yu. "From a financial viewpoint, we need to consider differences in the percentage of new drugs in Korea to other major countries and set the priority to strengthen reimbursement of treatments, including reimbursement of innovative new drugs, for diseases with high cost." "The percentage of new drugs in National Health Insurance-supported pharmaceutical cost is significantly lower than those of overseas, indicating that Korean patients are not fully benefiting from new drug treatments," Lee Young-shin, vice chairman of the KRPIA, said. "The system may need improvements, including the economic evaluation and expanding economic evaluation exemption and risk sharing agreement system, to enhance patient access to innovative new drugs and to allow them to have a better lifestyle."
- Policy
- Dongkook receives reimb for s-amlodipine triple-drug combo
- by Lee, Tak-Sun Oct 22, 2024 05:52am
- Olmevikar, a combination of s-amlodipine and olmesartan currently sold by Dongguk. The company will sell Olmevikar HCT from November, which contains Olmevikar and hydrochlorothiazide Dongkook Pharmaceutical has succeeded in becoming the first Korean company to receive reimbursement for a three-drug combination drug for hypertension that combines the CCB ‘s-amlodipine,’ ARB ‘olmesartan’ and diuretic ‘hydrochlorothiazide’. The combination therapy is expected to be useful for patients whose blood pressure is not adequately controlled with the company's combination product, s-amlodipine+olmesartan. Only Dongkook Pharmaceutical and Ahngook Pharm owns an s-amlodipine+olmesartan combination drug. According to industry sources on the 21st, Dongkook Pharmaceutical will start selling three types of Olmevikar HCT Tab beginning November, 1 with reimbursement. Olmevikar HCT Tab is a three-drug combination of S-Amlodipine Besylate 2.5 Hydrate, Olmesartan Medoxomil, and Hydrochlorothiazide. Compared to Daiichi Sankyo's existing Sevikar HCT, the only difference is the amlodipine and s-amlodipine ingredients. S-amlodipine is a component derived from only the S-enantiomer of amlodipine, which is the active isomer of amlodipine. Therefore, it is equally effective with half the dose of amlodipine with no difference in terms of stability. In Korea, there are 28 amlodipine 3-drug combination drugs like Sevikar HCT. However, Dongkook Pharmaceutical is the first to develop a 3-drug combination containing s-amlodipine. Olmevikar HCT is a combination of s-amlodipine besylate 2.5 hydrate and olmesartan medoxomil that was approved in late May for the treatment of essential hypertension, a condition in which blood pressure is not adequately controlled. It is indicated as a second-line treatment following amlodipine+olmesartan combination therapy as well as s-amlodipine+olmesartan. There are only two as s-amlodipine+Olmesartan combination drugs on the market, Dongkook Pharmaceutical ‘Olmevikar Tab’ and Ahngook Pharm’s ‘Levomos Tab.’ Last year, Olmevikar generated KRW 3.8 billion in outpatient prescriptions and Levomos KRW 1.2 billion, according to UBIST. “Looking at the indications, Olmevikar HCT tablets are expected to be useful for patients who are not sufficiently treated with Olmevikar Tab,” said a pharmaceutical industry insider. “Dongkook’s product competitivity by strengthening its hypertension combination product lineup.” The price has been set in line with the original Sevikar HCT. The company has applied for the 5 (s-amlodipine)/40 (olmesartan)/12.5 mg (hydrochlorothiazide) dose at KRW 821 per tablet, the 2.5/40/12.5 mg dose at KRW 765, and 2.5/20/12.5 mg dose at KRW 703 for, which is lower than the calculated price. This is the same as the price of the same dose Sevikar HCT.
- Policy
- MFDS grants EUA to COVID-19 variant vaccine for infants
- by Lee, Hye-Kyung Oct 22, 2024 05:51am
- The Ministry of Food and Drug Safety (MFDS) announces. The Ministry of Food and Drug Safety (Minister Oh Yu-kyoung, MFDS) announced on October 21st that it has granted Emergency Use Authorization for 'Comirnaty JN.1 Inj 0.033 mg/mL (bretovameran),' a SARS-coV variant (JN.1) vaccine for infants aged 6 months-4 years. Emergency Use Authorization is a system designed to respond appropriately to a public health crisis, including infectious disease pandemics. In this system, the MFDS minister enables the supply of medical products that do not have domestic approval by ordering manufacturers and importers to manufacture or import upon requests from the central administrative agency. The Korea Disease Control and Prevention Agency (KDCA) requested the Emergency Use Authorization to provide immunization for infants (6 months-4 years old), and the MFDS quickly reviewed it and granted approval. For Emergency Use Authorization, MFDS gathers expert opinions by reviewing company's submitted clinical and quality documents through the official posting of the emergency use authorization upon request from the central administrative agency. Then, after review and voting from the committee for the 'Public Health Emergency Preparedness and Responses for a Medicinal Product,' the MFDS makes a final decision. "We will continue to put efforts in enhancing public health by quickly supplying medical products that are effective in overcoming COVID-19, based on our scientific knowledge and regulatory expertise," the MFDS stated.
- Policy
- 2nd GIFT drug Nefecon’s approval imminent in Korea
- by Lee, Hye-Kyung Oct 21, 2024 05:48am
- Meditip's Nefecon (budesonide), which was designated as the 2nd Global Innovative Products on Fast Track (GIFT) drug last year, is close to receiving approval in Korea. According to industry sources on the 21st, the Ministry of Food and Drug Safety recently completed the safety and efficacy review for Nefecon. The completion of the safety and efficacy review in general leads to approval in Korea. Nefecon has been designated as a GIFT item as a drug that developed a new efficacy and effectiveness using the already marketed budesonide ingredient. A GIFT designation can also be given to a previously approved ingredient that has no treatment for a disease but can be used to treat a new patient population. Nefecon is a drug used to treat primary IgA nephropathy in adults with a urinary protein-to-creatinine ratio of 1.5 or greater who are at risk of rapid progression. In a presentation made at Kidney International, Nefecon was associated with a 27% lower urinary protein-to-creatinine ratio at 9 months of treatment compared to placebo. Glomerular filtration rate remained stable, with a difference of 3.87 ml/min/1.73 m2 compared to placebo. The drug was approved by the US FDA last year under the brand name Tarpeyo and by the European EMA under the trade name Kinpeygo. In China, the National Medical Products Administration (NMPA) designated Nefecon as a Breakthrough Therapy Designation (BTD) in 2020, and the Taiwan Food and Drug Administration granted it an Accelerated Drug Designation (ADD). IgA nephropathy is a disease caused by the deposition of immune complexes, including IgA, in the glomeruli of the kidneys, causing an inflammatory response. About 9,000 patients are known to be affected by IgA nephropathy in Korea. In clinical practice, antihypertensive drugs such as ARBs and ACEIs, immunosuppressants, and diuretics are used to treat IgA nephropathy. However, these drugs are symptomatic treatments that prevent the worsening of symptoms, and no drug fundamentally treats the condition. Meanwhile, drugs are given the GIFT designation if it is a ▲ drug aimed at treating serious diseases such as life-threatening cancer or rare diseases ▲ drug aimed at preventing or treating infectious diseases that may cause serious harm to public health, such as bioterrorism infectious diseases or pandemics of infectious diseases ▲ new drug developed by a Korea Innovative Pharmaceutical Company as designated by the Ministry of Health and Welfare ▲ a combination of a drug and medical device subject to fast-track review ▲ a case where there is no existing treatment or a drug showed clinically significant improvement in efficacy over existing treatments. The MFDS reviews the eligibility for the GIFT designation when a pharmaceutical company applies for expedited review and, if necessary, designates a drug as a GIFT item after consulting with the Medical Product Expedited Review Expert Council. Once designated, the review period is reduced by at least 25% (e.g. from 120 working days to 90 working days). Applicants will receive a variety of support for rapid commercialization, including rolling review, where the application data is reviewed on a rolling basis, close communication between the reviewer and the developer, such as item briefings and supplementary briefings, and expert consulting on regulatory matters.
