Latest news with #MHLW
Tokyo Weekender
11 hours ago
- Politics
- Tokyo Weekender
A Young Man Took a Part-Time Job. It Cost His Grandparents Their Welfare.
On June 12 , Japan's Supreme Court issued an unanimous ruling that sparked a perhaps unexpectedly huge reaction: It upheld the decision to cut off welfare benefits for an elderly couple in Kumamoto Prefecture. This wasn't because the pair's financial situation had improved, but rather because their cohabiting grandson — a vocational nursing student — had begun earning more from a part-time job he'd taken on to pay his tuition. Never mind that this grandson wasn't using a yen of it for family support. Never mind that existing law explicitly allowed for 'household separation,' a provision protecting the income of students from being lumped into the family's welfare calculation. In the eyes of the court, because the grandson's income had risen past a certain level, the prefectural government was right to treat the household as a single unit — and cut off the grandparents' access to much-needed aid. Though it came down earlier this month, the ruling has gone viral and garnered blistering reactions in recent days. To many in the Japanese public, the ruling feels cold and unforgiving, a cruelly rigid interpretation of the law that sends a bleak message to low-income families trying to break the cycle of poverty. One viral tweet with over 60,000 likes reads : 'The message from the ivory tower is clear: Poor people have no business dreaming.' Another rages : 'Why the hell are young people being forced to bankroll their entire families? The grandson has his own damn life. What kind of broken system punishes the ones actually trying to get out?' List of Contents: The Case: Bureaucratic Logic Meets Lived Reality A System Designed to Break the Poor The Backlash Related Posts The Case: Bureaucratic Logic Meets Lived Reality The case began in 2014, when a man living in Nagasu Town, Kumamoto Prefecture, began receiving welfare with his wife. At the time, their grandson, who lived with them, was studying at a vocational nursing school and working part-time to cover his tuition. Under Article 10 of the Public Assistance Act and related Ministry of Health, Labour and Welfare (MHLW) guidance, such students are eligible for setai bunri , or household separation, a legal mechanism meant to prevent a student's income from affecting the eligibility of their cohabiting relatives. Initially, the family qualified for this protection. But then the prefectural government abruptly changed course, citing an increase in the grandson's income and reclassifying the family as a single household. The elderly couple's welfare benefits were terminated. The grandfather sued; according to Yahoo News, he said that his grandson's income was being used for his own tuition, with no money left over for living expenses. In 2022, the Kumamoto District Court ruled in his favor, warning that 'terminating welfare would cause financial hardship for the couple and likely interfere with the grandson's efforts to become self-reliant.' But in 2024, the Fukuoka High Court overturned the decision, declaring the move 'not illegal.' The Supreme Court unanimously upheld that logic. A System Designed to Break the Poor Japan's welfare system has always been grudging at best, and openly punitive at worst. While Article 25 of Japan's Constitution guarantees all citizens 'the right to maintain the minimum standards of wholesome and cultured living,' in practice the system is riddled with stigma and bureaucratic thresholds. Only about 1.6 percent of the population receives welfare because applying for assistance often triggers social ostracism, surveillance and even coercive family contact. Under a policy known as fuyo shokai , caseworkers routinely contact an applicant's relatives — parents, siblings, even grandchildren — to ask if they can provide financial support. In theory, it's about upholding family responsibility. In reality, it functions as a deterrent. Some survivors of domestic violence have had their safety compromised after being exposed to abusers. In 2021, over 35,000 people petitioned to abolish the practice altogether. That same year, the government admitted that out of 460,000 inquiries, only 1.45 percent led to actual familial aid. The household separation system was one of the rare progressive compromises within this landscape. Introduced in the 1970s, the policy allowed children from welfare households to attend high school or vocational school without penalizing their families. It acknowledged that education is not indulgence — it is lifeline. But this ruling shows just how difficult it is to forge a path forward in Japan, given its harsh, uncompromising bureaucratic landscape. The Backlash The ruling is a particularly cruel irony in Japan, where demographic collapse and caregiving crises have turned multigenerational households into an economic necessity. The government needs young people to stay home, take care of aging relatives and fill in the gaps left by a crumbling welfare state. But it offers them no meaningful support to do so. There's an extra layer of irony in the fact that this young man wanted to become a nurse — exactly the kind of profession Japan desperately needs amid a ballooning healthcare crisis. He studied, he worked, he paid his way. And for that, the state punished his grandparents. After the ruling, Kumamoto Prefecture commented : 'Under the current system, we believe this ruling is correct and validates the prefecture's position. However, if future reforms are made to support young people who are working hard, we would support that.' The ruling may technically be correct under the current system. But that doesn't make it just. Related Posts Drunk American Tourist Damages Kyoto Temple Tied to Samurai Legend 'What Do You Mean It's Not Free?' The Otoshi Trap Confusing Tokyo's Tourists Inside Japan's Kurdish Refugee Crisis
Yahoo
13 hours ago
- Business
- Yahoo
Amicus Therapeutics Announces Approval of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) in Japan
Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Now Approved in Japan for Adults with Late-onset Pompe Disease (LOPD) PRINCETON, N.J., and TOKYO, June 25, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) for the treatment of adult patients with late-onset Pompe disease (LOPD). "We are delighted that we will now be able to offer a compelling new treatment option to patients living with late-onset Pompe disease in Japan. We are grateful to the MHLW and to Japan's Pompe community, including the patients, families, and physicians who participated in our clinical studies, for their collaboration,' said Bradley Campbell, President and Chief Executive Officer, Amicus Therapeutics, Inc. Pombiliti + Opfolda is a two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. The MHLW approval for Pombiliti + Opfolda was based on clinical data from the Phase 3 pivotal study (PROPEL). PROPEL is the only trial in LOPD to study the real-world population of both ERT-naïve and ERT-experienced participants in a controlled setting. With this approval, Pombiliti + Opfolda is now approved in the U.S., E.U., U.K., Canada, Australia, Switzerland and Japan. About Pombiliti + OpfoldaPombiliti® + Opfolda®, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONS AND USAGEPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). SAFETY INFORMATIONHYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK. About Late-Onset Pompe DiseaseLate-onset Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Late-onset Pompe disease can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly skeletal muscles and muscles that control breathing. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company's website at and follow on X and LinkedIn. CONTACT: Investors: Amicus Therapeutics Andrew Faughnan Vice President, Investor Relations afaughnan@ 662-3809 Media: Amicus Therapeutics Diana Moore Vice President, Corporate Communications dmoore@ 662-5079 FOLD-G
Yahoo
21-04-2025
- Business
- Yahoo
Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1
Del-desiran first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). "This decision by MHLW further reinforces the significant potential of del-desiran to address the root cause of DM1 and the urgent need to bring an approved therapy to the many people impacted by this devastating rare disease in Japan and around the world," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by del-desiran data reported from the MARINA and MARINA-OLE studies thus far, demonstrating favorable long-term safety and tolerability, reversal of disease progression, and consistent and durable improvements in multiple clinical endpoints. Looking ahead, there are multiple key milestones this year as we complete enrollment in the phase 3 HARBOR trial and continue to advance our global commercialization preparations to potentially deliver the first globally approved drug for people living with DM1." Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan. Japan's MHLW grants Orphan Drug designation to drugs in development for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need. An investigational therapy is eligible to qualify for Orphan Drug designation if there is no approved alternative treatment option or if there is high efficacy or safety expected compared to existing treatment options. Orphan Drug designation provides certain benefits, including prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period. About the Phase 3 HARBOR™ TrialThe global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. Patients will be administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the HARBOR trial, visit the HARBOR study website or visit and search for NCT06411288. About the Phase 2 MARINA-OLE™ StudyMARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit and search for NCT05479981. About Del-desiranDel-desiran, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). About Myotonic Dystrophy Type 1Myotonic dystrophy type 1 (DM1) is an underrecognized, autosomal dominantly inherited, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people living with DM1. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking StatementsAvidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with del-desiran in the MARINA and MARINA-OLE studies, the conclusions drawn therefrom, and the impact of such data on the advancement of del-desiran and its ability to treat DM1; the designs, goals, statuses and enrollment levels of, and plans for, the MARINA-OLE and HARBOR studies; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: Avidity may not realize any benefit from Orphan Drug designation of del-desiran by global health authorities; the data and results produced in Avidity's ongoing studies of del-desiran as of the most recent respective cutoff dates may not be indicative of final results, may not support a BLA submission, may not be satisfactory to the MHLW and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the MHLW and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of the MARINA-OLE and HARBOR studies; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact:Kat Lange(619) 837-5014investors@ Media Contact:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
Yahoo
17-03-2025
- Business
- Yahoo
HanAll Biopharma Announces Orphan Drug Designation for Batoclimab in Japan for Active Thyroid Eye Disease
Batoclimab receives Orphan Drug Designation in Japan, advancing treatment for active Thyroid Eye Disease (TED). Batoclimab, subcutaneous formulation, offers the potential for at-home administration, improving patient convenience and accessibility. Phase 3 study to confirm the potential of batoclimab to address unmet needs of patients with TED in process, with top-line results expected in the 2H 2025. SEOUL, South Korea, March 17, 2025 /PRNewswire/ -- HanAll Biopharma Co., Ltd. (KRX: a global biopharmaceutical company committed to discovering and developing innovative medicines for patients, announced that Batoclimab, an anti-FcRn treatment being developed for a range of autoimmune diseases, has received Orphan Drug Designation (ODD) from the Ministry of Health, Labor and Welfare (MHLW) in Japan for active Thyroid Eye Disease (TED). The Orphan Drug Designation (ODD), granted by the MHLW, is awarded to drugs and biologics intended to treat rare diseases affecting fewer than 50,000 people in Japan, with eligibility based on criteria such as patient population size, medical needs, and the feasibility of development, and possibility of development[1]. Currently, it is estimated that approximately 35,000 people in Japan are affected by TED.[2] "We are thrilled to have received Orphan Drug Designation for Batoclimab in Japan, marking an important milestone in our efforts to bring this promising treatment to patients in need," said Sean Jeong, MD, MBA, CEO of HanAll Biopharma. "This designation highlights the potential impact Batoclimab could have on the lives of patients with TED. We remain dedicated to advancing the development of this treatment and are focused on bringing it closer to the market." Batoclimab is a monoclonal antibody designed to target and inhibit FcRn, which plays a crucial role in recycling IgG antibodies. By selectively binding to FcRn, Batoclimab reduces the levels of harmful IgG antibodies, offering the potential to treat a variety of IgG-mediated autoimmune diseases. Being developed as a subcutaneous (SC) formulation, Batoclimab is expected to allow patients to administer the treatment at home, improving convenience and accessibility. Currently, Batoclimab is being investigated globally for conditions such as generalized myasthenia gravis (gMG), thyroid eye disease (TED), chronic inflammatory demyelinating polyneuropathy (CIDP), and Graves' disease. HanAll, with its licensee, is conducting a Phase 3 study of Batoclimab in active TED. The study, which includes patient enrollment in Japan, aims to confirm the efficacy and safety of Batoclimab as a potential new treatment for individuals affected by TED. Thyroid Eye Disease (TED), also known as Graves' orbitopathy, is a rare and debilitating autoimmune disorder primarily affecting individuals with hyperthyroidism or Graves' disease. TED is characterized by a range of severe symptoms, including eye bulging, pain, double vision, and, in some cases, vision loss. TED can severely limit daily activities such as reading, driving, and working. In addition, many individuals face significant social and psychological challenges, including concerns about their appearance, anxiety, low mood, and social withdrawal. Currently, treatment options for those with moderate to severe cases of TED remain limited, highlighting the urgent need for more effective therapies to improve patient outcomes and quality of life. About HanAll Biopharma HanAll Biopharma (KRX: is a global biopharmaceutical company with presence in Korea, the USA, Japan, and Indonesia with the mission of making meaningful contributions to patients' lives by introducing innovative, impactful medicines to address severe unmet medical needs. HanAll has been operating a portfolio of pharmaceutical products in the therapeutic areas of endocrine, circulatory, and urologic diseases for over 50 years. HanAll has also expanded its focus to immunology, oncology, neurology, and ophthalmology to discover and develop innovative medicines for patients with diseases for which there are no effective treatments. One of its lead pipeline assets, HL161 (INN: batoclimab), an anti-FcRn antibody, is being developed in Phase 3 and Phase 2 trials across the world for the treatment of autoimmune diseases including generalized myasthenia gravis (gMG), thyroid eye disease (TED), chronic inflammatory demyelinating polyneuropathy (CIDP). HL161ANS (IMVT-1402), another anti-FcRn antibody from HanAll, is being evaluated in Graves' Disease (GD) and Rheumatoid arthritis (RA). Another lead asset, HL036 (INN: tanfanercept), a TNF inhibitor protein, has commenced a Phase 3 VELOS-4 study in the US and is also being evaluated in China for the treatment of dry eye disease. Results from the Phase 1 study, HL192 (ATH-399A), a Nurr1 activator targeting Parkinson's Disease (PD), have been released, and the preparations to initiate a next study in patients with PD is progressing. For further information, visit our website and connect with us on LinkedIn. For any media inquiries, please contact HanAll PR/IR (pr@ ir@ Disclaimer Statement The contents of this announcement include statements that are, or may be deemed to be, "forward-looking statements." These forward-looking statements can be identified by the use of forward-looking terminology, including the terms "believes," "estimates," "anticipates," "expects," "intends," "may," "will," or "should," and include statements HANALL (the company, we) makes concerning its 2025 business and financial outlook and related plans; the therapeutic potential of its product candidates; the intended results of its strategy and the company, and its collaboration partners', advancement of, and anticipated clinical development, data readouts and regulatory milestones and plans, including the timing of planned clinical trials and expected data readouts; the design of future clinical trials and the timing and outcome of regulatory filings and regulatory approvals. By their nature, forward-looking statements involve risks and uncertainties, and readers are cautioned that any such forward-looking statements are not guarantees of future performance. The company's actual results may differ materially from those predicted by the forward-looking statements. These may include various significant factors, such as our expectations regarding the inherent uncertainties associated with competitive developments, preclinical and clinical trial and product development activities, and regulatory approval requirements. In addition, performance may be affected by our reliance on collaborations with third parties, estimating the commercial potential of our product candidates, our ability to obtain and maintain protection of intellectual property of technologies and drugs, our limited operating history, and our ability to obtain additional funding for operations and to complete the development and commercialization of product candidates. A further list and description of these risks, uncertainties, and other risks can be found in Korea Stock Exchange (KRX) filings and reports, including in our most recent annual report as well as subsequent filings and reports filed by the company with the KRX. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. We undertake no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by Korean law and regulations. [1] Regulation by MHLW: [2] Natsuko W et al. J Endocr Soc. 2023 Nov 27;8(1):bvad148. View original content to download multimedia: SOURCE HanAll Biopharma Sign in to access your portfolio
Yahoo
25-02-2025
- Business
- Yahoo
Press Release: Sarclisa approved in Japan for patients with newly diagnosed multiple myeloma
Sarclisa approved in Japan for patients with newly diagnosed multiple myeloma Approval offers access to new treatment options for newly diagnosed MM patients Approval based on positive results from the IMROZ phase 3 study that demonstrated Sarclisa in combination with VRd significantly improved progression-free survival, compared to VRd alone in transplant-ineligible newly diagnosed multiple myeloma Paris, February 25, 2025. The Ministry of Health, Labour and Welfare (MHLW) in Japan has approved Sarclisa, in combination with bortezomib, lenalidomide, and dexamethasone (VRd), for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM) based on data from the IMROZ phase 3 study. Global Head, Oncology'In recent years, new multiple myeloma cases have increased steadily in Japan and other Asian-Pacific nations, creating a need for new treatment approaches, particularly in the front-line setting. While Sarclisa-based combinations have been approved for relapsed or refractory patients in Japan, this approval represents the first indication for certain newly diagnosed patients. We are pleased to offer physicians an important new option for their patients earlier in the treatment journey, building upon our continued commitment to advancing innovative oncology treatments in difficult-to-treat hematologic malignancies around the world.' In Japan, Sarclisa was launched in August 2020 and has been approved for four different treatment regimens (in combination with pomalidomide and dexamethasone, as monotherapy, in combination with carfilzomib and dexamethasone, or in combination with dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma). In addition, Sarclisa has front-line approvals in the EU and the US. In the Asia Pacific region, Sarclisa combination regimens were also recently approved by the National Medical Products Administration in China, specifically Sarclisa-VRd in NDMM patients who are not eligible for autologous stem cell transplant, as well as Sarclisa in combination with pomalidomide and dexamethasone (Pd) for the treatment of adult patients with relapsed or refractory MM who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. About SarclisaSarclisa (isatuximab) is a CD38 monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a target for antibody-based therapeutics such as Sarclisa. In the US, the non-proprietary name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of biological products guidance for industry issued by the US Food and Drug Administration. Currently, Sarclisa is approved in more than 50 countries, including in the US, EU, Japan, and China, across multiple indications. Based on the ICARIA-MM phase 3 study, Sarclisa is approved in the US, EU and Japan in combination with Pd for the treatment of patients with R/R MM who have received ≥two prior therapies, including lenalidomide and a proteasome inhibitor; this combination is also approved in China for patients who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. Based on the IKEMA phase 3 study, Sarclisa is also approved in more than 50 countries in combination with carfilzomib and dexamethasone, including in the US for the treatment of patients with R/R MM who have received one to three prior lines of therapy and in the EU for patients with MM who have received at least one prior therapy. In the US, EU, UK, and China, Sarclisa is approved in combination with VRd as a front-line treatment option in transplant-ineligible NDMM patients, based on the IMROZ phase 3 study. This combination is also approved in Japan for patients with NDMM. Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, which includes several phase 2 and phase 3 studies across the MM treatment continuum spanning six potential indications. In addition, the company is evaluating a subcutaneous (SC) administration method for Sarclisa in clinical studies. In January 2024, Sanofi reported positive results from the IRAKLIA phase 3 study evaluating Sarclisa SC formulation administered via an on-body delivery system (OBDS) in combination with Pd compared to intravenous (IV) Sarclisa in patients with R/R MM. In December 2024, additional positive results from the program, including the GMMG-HD7 phase 3 study evaluating Sarclisa-RVd induction therapy in transplant-eligible NDMM patients, were also presented at the 66th American Society of Hematology Annual Meeting and Exposition. The safety and efficacy of Sarclisa has not been evaluated by any regulatory authority outside of its approved indications and methods of delivery. In striving to become the number one immunoscience company globally, Sanofi remains committed to advancing oncology innovation. Through focused strategic decisions the company has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive progress. Efforts are centered on difficult-to-treat often rare cancers such as select hematologic malignancies and solid tumors with critical unmet needs, including multiple myeloma, acute myeloid leukemia, certain types of lymphomas, as well as gastroenteropancreatic neuroendocrine tumors and other gastrointestinal and lung cancers. For more information on Sarclisa clinical studies, please visit About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Evan Berland | +1 215 432 0234 | Nicolas Obrist | +33 6 77 21 27 55 | Léo Le Bourhis | +33 6 75 06 43 81 | Victor Rouault | +33 6 70 93 71 40 | Gilbert | +1 516 521 2929 | Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Lauscher | +1 908 612 7239 | Keita Browne | +1 781 249 1766 | Pham | +33 7 85 93 30 17 | Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Sanofi forward-looking statementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans', and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under 'Risk Factors' and 'Cautionary Statement Regarding Forward-Looking Statements' in Sanofi's annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press ReleaseSign in to access your portfolio
