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- 2026-04-04 13:41:29
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- Illegal errand companies deliver Wegovy for ₩10,000
- by Jung, Heung-Jun Oct 22, 2024 05:52am
- Amid the craze for obesity treatment drug Wegovy, errand companies are also thriving by offering pharmacy proxy pick-up and delivery services. These are illegal businesses that take advantage of the fact that consumers are looking for a low-priced source of the non-reimbursed drug, as the price varies greatly depending on the pharmacy. There are posts on diet communities that claim to have purchased Wegovy at a low price in the KRW 400,000 range. Some people said that they called the pharmacy to check the price, while others said that they found a lower price through non-face-to-face treatment venues. Some people also wrote that they received Wegovy packaged in a cooler from another region. When the reporter checked with the pharmacy that offered Wegovy delivery, they were using an errand delivery service. The pharmacy representative said, ‘We don't offer delivery services for Wegovy,” but gave me the contact details of a courier service. They explained that the pharmacy has been contacted repeatedly due to the courier service’s promotion. Although the pharmacy does not deliver directly, they are tacitly allowing the act. “They charge KRW 10,000 for 1-2 pens and KRW 12,000 for 3-5 pens,” said A, who introduced himself as an errand-running company. If you use non-face-to-face treatment services, you can send the prescription to the pharmacy and then contact them for us to receive and deliver the medicine,” A explained. After submitting the prescription to the pharmacy, the customer can exchange the detailed address and account number for the Wegovy delivery through A. A also added a condition that the customer should leave a review on online cafes and communities. “I don’t make much with the deliveries. To make a profit, we need multiple delivery orders,’ said A, adding, ’Please leave your reviews online.’ Meanwhile, pharmacies are also focusing on stocking up and selling Wegovy in line with shifting attention from Saxenda to Wegovy. Low doses are selling out quickly. Lawmakers are also posting reviews of Wegovy use on social media to promote in-patient prescriptions. The selling price of the drug, which can be checked through non-face-to-face treatment websites, varied greatly on the first day of its release, but as of today (21st), the drug’s price is set in the KRW 500,000 range.
- 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.
- Company
- "Multiple myeloma dynamics require changes to reimb strategy
- by Whang, byung-woo Oct 22, 2024 05:51am
- Youngil Koh, Professor of the Department of Hemato-Oncology at Seoul National University Hospital "Multiple myeloma is a cancer type that has a great ripple effect depending on new drugs provided in a certain environment. Reimbursement of multiple myeloma treatment requires discussion following a thorough evaluation of the impact of the treatment depending on the treatment environment." Multiple myeloma is one of the cancer types with increased treatment options following new drug entries. There are concerns about extending the relapse period and progression-free survival (PFS) as the number of treatments increases due to relapses. However, the discussion for reimbursement is hindered by cost issues. Youngil Koh, Professor of the Department of Hemato-Oncology at Seoul National University Hospital, emphasizes the importance of discussing appropriate disease-specific environments. According to Koh, the development of new drugs for blood cancers is active because of disease characteristics. Because cancer cells tend to exist in the blood and it is easy to obtain samples, the research is relatively simple. Understanding of blood cancer yields significant results compared to solid cancers. "Almost all new drugs begin from blood cancers, including TKI, therapeutic antibody, bispecific antibody, and ADC, and expand to solid cancers. Bispecific antibodies and ADCs are used as the standard treatment for blood cancers and expand their indications to solid cancers," Koh said. In South Korea, a bispecific antibody treatment, Tecvayli (teclistamab), is available for treating multiple myeloma. This drug was approved in July. The use of Tecvayli is limited because it is not reimbursed, but the expansion of market presence is expected following the announcement of long-term follow-up research results of the drug's clinical trial, MajesTEC-1, at the American Society of Clinical Oncology (ASCO 2024) meeting. The results of evaluating the efficacy of Tecvayli in 165 study participants at 30.4 months interim follow-up showed that Tecvayli reduced tumor sizes by 63% (overall response rate, ORR) in patients who had failed or were non-responsive to third-line treatments or higher. 46.1% of the patients reached complete remission (CR). The study confirmed consistent, long-term clinical effectiveness and positive safety profile. "The 30-month follow-up outcome did not differ from the data presented in 2022; therefore, it is significant that we have confirmed the anticipated outcome," Koh said. "Specifically, 4 out of 10 patients showed great responses and maintained response over two years." "We have observed that the real effects observed in 30-50 patients administered with the drug at the Seoul National University Hospital did not differ greatly from the research," added Koh. "1/3 of the patients benefited from the treatment, and close to half experienced great responses." In conclusion, Koh says that the result should be highly regarded because Tecvayli's long-term follow-up data demonstrated that patients showed a significantly long response period after almost two-years of treatment. Will Tecvayli, demonstrating its long-term effects, be adminstered earlier in the treatment course? Based on the assumption that there are no differing opinions about the effect of Tecvayli, the question is 'when' to use the drug. There are concerns about using the good drug first or last, regardless of disease. However, multiple myeloma requires a more careful approach because the treatment order is particularly important. Koh says it is difficult to determine at this point, but Tecvayli is likely to be placed earlier in order since the data supporting its use of Tecvayli in earlier treatment course are now available. "Tecvayli is the only bispecific antibody to be used for the treatment of multiple myeloma in South Korea and it targets BCMA, which the conventional treatments haven't targeted," Koh said. "Based on the clinical data of Tecvayli used in combination with already effective treatments that are currently used, Tecvayli is likely to be employed earlier in the treatment and will be able to extend overall survival," Koh said. However, Koh assessed that it is too early to mention whether bispecific antibody, known as the next-generation treatment, or CAR-T therapy should be placed earlier. "The positions of bispecific antibody and CAR-T for treating multiple myeloma have not been established yet. We now know that two treatments successfully treat multiple myeloma, but it is early to determine which one is superior. Upcoming clinical data will determine their positions," Koh emphasized. "Multiple myeloma is highly dynamic…changes to reimbursement strategy should be considered" Besides the Tecvayli effect, the dilemma for new drugs is the cost. Patient burden increases when high-cost drugs are not covered by reimbursement. Since multiple myeloma treatment tends to use 2-3 treatments in combination, the government is hesitant to opening the door to reimbursement. Regarding this issue, Koh advises that rather than simply comparing the cost of the drug, a reimbursement strategy reflecting the characteristics of multiple myeloma should be discussed. "The dynamic of treatments for multiple myeloma is changing due to new drugs, and the disease is greatly influenced by the type of new drugs and the type of reimbursement. We need to discuss reimbursement after anticipating the potential impact of reimbursement on overall treatment outcomes," Koh explained. "The landscape of multiple myeloma is diverse and constantly changing; therefore, instead of simply comparing costs, the government can strategize better reimbursement plan taking into account various outcomes," Koh said. Koh stated that we need to discuss patient access to clinical trials while reimbursement for new drugs is limited. "New drugs are being developed faster than the reimbursement process. It is inevitable that patients cannot benefit from new drugs because reimbursement cannot cover all," Koh said. "In other words, drugs are available at clinical stages, so we could discuss increasing patient access to new drugs."
- 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.
- Product
- Consumers use tricks to receive Wegovy prescriptions
- by Kang, Hye-Kyung Oct 22, 2024 05:51am
- With heating interest in the obesity drug Wegovy, the way-around measures the consumers are taking to receive prescriptions are causing controversy. From how to receive Wegovy through non-face-to-face treatment venues without the legwork to how to split the high-dose formulations being shared online, concerns are growing over the drug’s misuse. The first issue is non-qualified prescriptions. Prescriptions that ignore the prescribing criteria, such as obese patients with a body mass index (BMI) of 30 kg/m2 or more, are becoming widespread, and one of the outlets for such is the non-face-to-face treatment platforms. As influencers with tens of thousands to hundreds of thousands of followers are revealing the fact that they have been prescribed Wegovy, non-face-to-face treatment platforms such as ‘Dr Now’ and ‘My Doctor’ are being mentioned as venues for such prescriptions. You can receive a prescription for up to 5 pens of Wegovy through non-face-to-face treatment, and can also compare drug prices at pharmacies. The reason for choosing non-face-to-face treatment is that it is easier to receive prescriptions and dispense compared to face-to-face treatment. Although some prescribing medical institutions have been promoting Wegovy prescription through press releases, blogs, and social media including Instagram, hospitals and pharmacies have not yet been able to secure enough supplies, which is why patients are turning their attention to non-face-to-face treatment, which is relatively easier to receive prescriptions and dispensing. These platforms allow users to check the information, contact details, and prices of Wegovy in pharmacies with stock at a glance, making it easy to receive Wegovy without having to make many phone calls. Looking at the non-face-to-face treatment platforms, the price of Wegovy prescriptions by pen ranges from KRW 5,000 for 1 pen, KRW 7500 for 2 pens, KRW 10,000 for 3 pens, and KRW 10,000 to KRW 15,000 for 4 pens. For dispensing, a pharmacy in Gwangju had the lowest price in the country at KRW 419,000. In addition, some blogs and other websites have been sharing advice on how to get prescriptions easily, with tips such as ‘raising your weight.’ Another problem is the indiscriminate spread of information on its off-label use, such as ‘how to use Wegovy at half the price’. The correct way to start from the 0.25mg dose, then increase to 0.5mg, 1.0mg, 1.7mg, and 2.4mg over a four-week titration period, but many people have been prescribed 2.4mg and have been sharing tips such as splitting the dose into multiple doses. In fact, one doctor described it as ‘using the same drug at half the price’ and said, ‘I just use a 2.4mg syringe from the start. Then you can use a single syringe for four 0.25mg doses = 1/4 syringe, and four 0.5mg doses = 1/2 syringe, and so on.’ The argument is that since the price per dose is the same, it's more cost-effective to take one pen of 0.25mg over 4 doses than one pen of 0.5mg over 8 doses. It's also easier to stock the higher doses than the starter doses of 0.25mg and 0.5mg. Pharmacists are wary that their fears are being realized. Pharmacist A pointed out that “non-face-to-face treatment is promoting medical shopping in conjunction with the Wegovy craze. Shouldn't there be sanctions against random prescriptions and off-label use that are being conducted with non-face-to-face treatment?” Pharmacist B also said, “I am concerned that the Wegovy craze is spreading like a fad on social media without any warnings or side effects.” In particular, regarding dividing the high-dose formulation, B added, “Wegovy can be stored for up to 6 weeks after opening. If you split the dose, you'll be forced to take it beyond that period. I don't think I can give such advice from a professional perspective.” ‘The role of the government, pharmaceutical companies, and healthcare professionals will be crucial in tackling this Wegovy craze,’ he added. The Korea Pharmaceutical Association also said it plans to respond strongly to concerns such as the abuse of non-face-to-face Wegovy prescriptions and the delivery by documenting cases. Meanwhile, Novo Nordisk has warned patients in its patient instructions not to use Wegovy Prefilled Pen if they are hypersensitive (allergic) to semaglutide or any of the drug's excipients, or if they are pregnant, nursing mothers, etc., and emphasized patients to refrain from using the drug unless indicated.
- Company
- Will combination therapies for cancer be reimbursed in KOR?
- by Whang, byung-woo Oct 21, 2024 05:49am
- As the government has begun to prepare the principles for the reimbursement review of combination therapies that use new anticancer drugs, attention is being paid to whether the discussion will progress further. According to industry sources, the Health Insurance Review and Assessment Service recently held a Cancer Disease Deliberation Committee meeting and decided to prepare deliberation principles to discuss whether to approve benefits for major combination therapies. The decision is in line with the growing calls for a change in the reimbursement paradigm for prescribing anticancer drugs based on accumulated evidence on ‘combination therapies’ between treatments from various clinical studies. Recently, multinational pharmaceutical companies have been seeking the reimbursement of combination therapies using new anticancer drugs, calling for an institutionalized process. When considering reimbursement for combinations of non-reimbursed new drugs and reimbursed chemotherapy drugs, the industry has suggested that discussions on previously reimbursed drugs should be reserved and only the non-reimbursed drugs should be discussed. A case in point is AstraZeneca's immuno-oncology drug Imfinzi (durvalumab), the reimbursement of which had been discussed for biliary tract cancer last year. At the time, CDDC only approved gemcitabine and cisplatin in combination with chemotherapy (the GemCis regimen) for first-line treatment of biliary tract cancer, while leaving Imfinzi non-reimbursed. The government will discuss setting a deliberation principle where a drug, which is already reimbursed, is used with a non-reimbursed new drug as part of combination therapy and should continue to be reimbursed without further discussions. This can be observed in the results of the 7th CDDC Review, which reviewed the combination of ‘endocrine therapy’ and ‘Verzenio (abemaciclib)’ for the treatment of early breast cancer. The outcome of the deliberations was to keep the previously reimbursed adjuvant endocrine therapy as is and make the newly added Verzenio available on a ‘100% coinsurance’ basis, reducing the cost burden on the patients. In other words, it means that the process will be simplified by allowing the reimbursement of existing treatments within the combination to be recognized. However, these principles are not automatically applicable to all new combination therapies, and as it leaves the existing framework in place, it is far from the improvements to the new drug approval approaches the industry had hoped for. Nevertheless, it is positive as it has opened the door to further discussions. There are also expectations that such discussions will lead to solutions for ‘new drug+new drug’ combination therapies. A pharmaceutical industry official said, “It is still a drug-by-drug approach, so it's not a one-size-fits-all approach, but it's positive that the discussions have been officially made. We understand that KRPIA and others are also having discussions on what kind of processes will be required for the reimbursement of new drugs for combination therapies in the future,’ “With the number of combination therapy cases increasing, there is currently no set process for how applications should be submitted for listing and how they should be reviewed. We are looking forward to the various approaches that may be developed through discussions as individual cases have come to light.”
- 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.
- Company
- Therapeutic device 'improves OS for lung cancer patients'
- by Son, Hyung Min Oct 21, 2024 05:48am
- The first therapeutic device has been approved for use in non-small cell lung cancer (NSCLC). Novocure's therapeutic device, in combination with an NSCLC medication, demonstrated to improve patients' overall survival and won the approval of the U.S. regulatory authority. In South Korea, Nu Eyne is researching the potential of oncology therapeutic devices through clinical studies. According to industry sources on October 19th, the U.S. Food and Drug Administration (FDA) granted approval for Novocure's Optune Lua for the treatment of metastatic non-small cell lung cancer (NSCLC). Optune Lua is approved for use in combination with PD-1/PD-L1 immunotherapy for cancer or docetaxel. Optune Lua is a device that delivers 'Tumor Treating Fields (TTFields),' which exert physical forces on the electrically charged components of dividing cancer cells, resulting in cell death. Novocure says that TTFields do not affect healthy cells because they have different properties (including division characteristics, morphology, and electrical properties). The basis of approval was the Phase 3 LUNAR study. The clinical trial involved 301 patients with NSCLC who had failed during or after platinum-based chemotherapy. The patients were randomly assigned in a 1:1 ratio to the group receiving Optune Lua plus a PD-1/PD-L1 inhibitor or docetaxel or to the single-treatment group receiving a PD-1/PD-L1 inhibitor or docetaxel. Clinical results showed Optune Lua combination therapy recorded a median overall survival (OS) of 13.2 months, set as the primary endpoint. This was 3 months longer than 9.9 months in the control group. Patients randomly administered Optune Lua plus immunotherapy had a median OS of 19.0 months, and patients administered a PD-1/PD-L1 inhibitor alone had a median OS of 10.8 months. The Optune Lua combination therapy group had an OS extension of over 8 months compared to patients treated with immunotherapy alone. Patients randomly administered Optune Lua plus docetaxel had a median OS of 11.1 months, whereas patients treated with docetaxel had a median OS of 8.9 months. However, this figure did not show a statistically significant difference. Device-related adverse events (AE) occurred in 63.1% of patients treated with Optune Lua combination therapy. Most cases were Grade 1-2 and only 4% (6 patients) experienced Grade 3 skin toxicity and discontinued treatment. Optune Lua did not show Grade 4-5 toxicity, and no deaths occurred. Therapeutic device is being developed in South Korea…Nu Eyne challenges the field A Korean company, Nu Eyne, has confirmed the effects of immunotherapy and is conducting therapeutic device development. To confirm treatment effects on cancer, Nu Eyne conducted a cell-based clinical study to compare the stimulated group to the non-stimulated group of cultured cells after 48 hours of electrical stimulation. The results showed that the electrically stimulated group had an up to 80% reduction in tumor size. Nu Eyne's technology for its oncology therapeutic device has been developed to deliver a high-intensity electrical field that responds particularly to cancer cells, thereby inducing cell death. The technology is known to have a low risk of causing side effects on surrounding organs. Nu Eyne is developing advanced systems and materials to maximize oncology therapeutic effects. Using its technology, Nu Eyne aims to develop therapeutic devices to secure indications for treating lung cancer and bone metastatic cancer. Nu Eyne aims to complete the development of a clinical therapeutic device prototype in the second half of 2026. After that, the company plans to conduct clinical trials to secure the FDA regulatory approval.
- Company
- Oxlumo receives orphan drug designation in Korea
- by Eo, Yun-Ho Oct 21, 2024 05:48am
- The primary hyperoxaluria treatment Oxlumo received orphan drug designation in Korea. The Ministry of Food and Drug Safety (MFDS) recently announced the designation through an orphan drug designation notice. Oxlumo (lumasiran) was also recently designated a Global Innovative products on Fast Track (GIFT) by the MFDS. The drug is an RNAi therapeutic for primary hyperoxaluria (PH1), a rare kidney disease that was approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2020. RNAi is recognized as a next-generation gene therapy that has the advantage of providing specific access to disease-causing human genes. PH1 is a rare disease caused by excessive production of oxalate in the liver. Symptoms include the deposition of oxalate crystals or potassium oxalate crystals in the kidneys and urinary tract. As the disease progresses, kidney damage occurs and dialysis is required. Eventually, a liver or kidney transplant is required to cure the disease. The option to treat PH1 with a therapeutic agent became available with the approval of Oxlumo in 2020. Oxlumo is an RNAi therapy that targets hydroxy acid oxidase 1 (HAO1), which encodes glycolate oxidase (GO), the enzyme that produces oxalate. The mechanism of action reduces oxalate by inhibiting HAO1, which reduces GO production. Oxlumo’s efficacy was confirmed through a Phase III clinical trial in 39 PH1 patients aged 6 years and older. Oxlumo reduced oxalate levels in the urine by 65.4% compared to the placebo group. In addition, 84% of patients treated with Oxlumo had urinary oxalate levels near normal. 52% achieved urinary oxalate levels within the normal range.
- Company
- RNAi therapeutic 'Givlaari' receives the ODD in KOR
- by Eo, Yun-Ho Oct 21, 2024 05:48am
- Product photo of Givlaari (givosiran) RNAi therapeutic 'Givlaari' has been designated as an orphan drug following its designation as the GIFT. The Ministry of Food and Drug Safety (MFDS) recently announced this through the posting of the Orphan Drug Designation (ODD). Givlaari (givosiran) had previously been designated as the 'Global Innovative products on Fast Track (GIFT)' by the MFDS. This drug won U.S. FDA approval in 2019 and also received the accelerated approval from Europe's EMA. Givlaari is a subcutaneous injection that targets the enzyme aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. AHP is caused by a genetic deficiency due to the depletion of the enzyme needed for heme biosynthesis in the liver. It is a rare genetic disorder with abnormal accumulation of porphyrins in the body. Patients with AHP experience debilitating symptoms, such as severe abdominal pain, vomiting, and seizures, and chronic pain. The efficacy of Givlaari has been demonstrated through the Phase 3 ENVISION clinical trial. Givosiran was shown to reduce the occurrence of annual porphyria-associated seizures by 74% compared to the placebo. The research outcome was published in The New England Journal of Medicine (NEJM) on June 11th, 2020. During the six months of treatment, the percentage of patients who had not experienced porphyria-associated seizures was 50% for the givosiran-treatment group and 16.3% for the placebo group.
