EMA Recommends Treatment for Maple Syrup Urine Disease
MSUD is a rare genetic disorder of BCAA metabolism. In this condition, buildup of toxic metabolites from BCAAs produced from protein breakdown leads to significant and potentially irreversible developmental effects. It may cause metabolic derangements, cerebral edema, seizures, coma, and respiratory failure, and it may be fatal. It affects fewer than 0.1 in 10,000 people in the EU, equivalent to fewer than 5000 people.
This is below the ceiling for orphan drug designation (5 in 10,000). In 2020, the EMA granted orphan designation to a solution of amino acids not containing any BCAAs, intended to be given by infusion to replace other sources of amino acids. At the time, the EMA said, there were no suitable treatments authorized in the EU for MSUD, and patients were managed with strict diets to control the amount of BCAAs taken in from proteins. Some forms of the disorder also responded to vitamin B supplements. Some patients needed hospitalization for enteral feeding or procedures to filter BCAAs directly from the blood. Other patients were judged suitable for liver transplantation, which restores the ability to break down BCAAs.
Medicine Reduces Harmful Amino Acids
Maapliv, manufactured by Recordati Rare Diseases, is a combination of amino acids free of BCAA. It is used as a solution for infusion in combination with carbohydrate and lipid supplementation to prevent or reverse protein catabolism and promote anabolism in patients with MSUD decompensation, thereby reducing harmful alpha-keto acid levels.
The CHMP said that leucine normalization had been shown in patients with MSUD decompensation who are given Maapliv in five scientific publications that reported on parenteral use of BCAA-free solutions with the same formulation as Maapliv. Treatment with Maapliv should be initiated under the supervision of a physician experienced in the management of MSUD disease.
Detailed recommendations for the use of Maapliv will be described in the summary of product characteristics, which will be published on the EMA website in all official European Union languages after the marketing authorization has been granted by the European Commission.
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Novartis ianalumab Phase III trial meets primary endpoint in ITP, demonstrating statistically significant improvement in time to treatment failure
Ianalumab prolonged the duration of safe platelet levels during and after treatment in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1,2 Patients treated with ianalumab also experienced a significantly higher rate of sustained improvements in platelet count, the key secondary endpoint of the study1 Ianalumab, administered as four once-monthly doses in the ITP setting, could offer long-term disease control through a short course of treatment and potentially allow patients extended time off treatment, if approved Data expected to be presented at an upcoming medical meeting and included in future regulatory submissions in 2027 along with results from the ongoing first-line ITP trial, VAYHIT1 Basel, August 12, 2025 – Novartis today announced positive top-line results from VAYHIT2, a Phase III trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1,2. Ianalumab plus eltrombopag, compared to placebo plus eltrombopag, significantly prolonged the time to treatment failure (TTF), the primary endpoint that assesses how long patients maintain safe platelet levels during and after the treatment period1,2. Ianalumab is being investigated in other B cell-driven autoimmune diseases, including ongoing Phase III trials in first-line ITP and in second and later lines of warm autoimmune hemolytic anemia, with readouts expected in 20263,4. In VAYHIT2, patients treated with ianalumab plus eltrombopag experienced a significantly higher rate of sustained improvements in platelet count at six months, the key secondary endpoint of the study1. The safety profile of ianalumab was consistent with what was previously observed in clinical studies, with no new safety signals1. 'While current treatments for ITP are generally effective in raising platelet counts, many patients require life-long treatment to maintain safe levels, which can create a lasting treatment burden,' said Adam Cuker, M.D., Professor of Medicine and Chief, Section of Hematology, University of Pennsylvania. 'The results from VAYHIT2 are encouraging, as they suggest that ianalumab may support longer periods of disease control and reduce the need for continuous treatment.' ITP is a rare autoimmune disorder characterized by low platelet counts leading to an increased risk of bleeding, bruising and chronic fatigue5-7. Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control7. There is a need for other treatment options with novel mechanisms of action that offer durable responses while reducing the burden of long-term treatment8. 'For many people living with ITP, chronic treatment can disrupt their daily life due to the burden of regular dosing, dose adjustments and side effects,' said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. 'These positive top-line results from the Phase III study highlight the potential of ianalumab, if approved, to deliver long-term disease control with four once-monthly doses and enable extended time off treatment.' Data is expected to be presented at an upcoming medical meeting and included in future regulatory submissions in 2027 along with results from the ongoing first-line ITP trial, VAYHIT1. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency9,10. Recently, Novartis announced positive top-line results for ianalumab in adults with active Sjögren's disease. About ianalumab Ianalumab (VAY736) is a novel fully human monoclonal antibody being investigated for its potential to treat various B cell-driven autoimmune diseases, including Sjögren's disease, immune thrombocytopenia (ITP), systemic lupus erythematosus (SLE), lupus nephritis (LN), warm autoimmune hemolytic anemia (wAIHA) and diffuse cutaneous systemic sclerosis (dcSSc)2,4,11-16. Its mechanism of action targets B cells in two ways, namely combining B cell depletion via antibody-dependent cellular toxicity (ADCC) and interruption of BAFF-R mediated signals of B cell function and survival11. In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren's disease, systemic lupus erythematosus, and immune thrombocytopenia17-19. Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 202420. About primary immune thrombocytopenia Primary immune thrombocytopenia (ITP) is a rare, autoimmune disorder in which the immune system mistakenly targets and destroys platelets, the cells essential for blood clotting5. This can lead to symptoms such as prolonged bleeding, easy bruising and chronic fatigue, which can significantly impact daily life5,6. Despite available treatments, many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control7. Current options often focus on maintaining safe platelet levels and preventing bleeding complications and may require ongoing use7,21. The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life6,22. There is a need for therapies that offer durable response while reducing the burden of long-term treatment8. About VAYHIT2 VAYHIT2 (NCT05653219) is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of two different doses of ianalumab versus placebo, in addition to eltrombopag, in adults with primary immune thrombocytopenia (ITP) (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids2. Alongside eltrombopag, patients were randomized 1:1:1 to receive four once-monthly intravenous infusions of ianalumab at 3 mg/kg, ianalumab at 9 mg/kg or placebo2. The primary endpoint was time to treatment failure, which is defined as the time from randomization until either: a platelet count of less than 30 G/L later than 8 weeks from randomization; the need for rescue therapy later than 8 weeks from randomization; initiation of a new ITP treatment at any time; ineligibility or inability to taper/discontinue eltrombopag; or death2. The key secondary endpoint is the percentage of patients with a stable platelet count response at Month 62. Other secondary endpoints include measures of depth and duration of platelet response as well as patient-reported outcomes that measure quality of life and fatigue, among other endpoints2. DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as 'potential,' 'can,' 'will,' 'plan,' 'may,' 'could,' 'would,' 'expect,' 'anticipate,' 'look forward,' 'believe,' 'committed,' 'investigational,' 'pipeline,' 'launch,' or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Novartis. Data on file. NCT05653219. A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids (VAYHIT2). Accessed July 21, 2025. NCT05653349. Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) (VAYHIT1). Accessed July 21, 2025. NCT05648968. A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia (VAYHIA) Accessed July 21, 2025. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386-2393. doi:10.1182/blood-2008-07-162503 Kuter DJ, Mathias SD, Rummel M, et al. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012;87:558-61 Kuter DJ. The treatment of immune thrombocytopenia (ITP)—focus on thrombopoietin receptor agonists. Ann Blood. 2021;6:27. doi:10.21037/aob-2021-itp-04 Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, et al. Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022;15(7):779. doi:10.3390/ph15070779 US Food and Drug Administration. Orphan drug designation: ianalumab—treatment of primary immune thrombocytopenia. Published February 13, 2025. Accessed August 9, 2025. European Commission. Community register of orphan medicinal products: ianalumab. Updated June 30, 2025. Accessed August 9, 2025. Dörner T, Bowman SJ, Fox R, et al. Safety and Efficacy of Ianalumab in Patients With Sjögren's Disease: 52-Week Results From a Randomized, Placebo-Controlled, Phase 2b Dose-Ranging Study. Arthritis Rheumatol. 2025;77(5):560-570. doi:10.1002/art.43059 NCT05350072. Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-1). Accessed August 9, 2025. NCT05349214. Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome (NEPTUNUS-2). Accessed August 9, 2025. NCT05639114. Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1) (SIRIUS-SLE 1). Accessed August 9, 2025. NCT05126277. Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis (SIRIUS-LN). Accessed August 9, 2025. NCT06470048. A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis. Accessed August 9, 2025. Bowman SJ, Fox R, Dörner T, et al. Safety and efficacy of subcutaneous ianalumab (VAY736) in patients with primary Sjögren's syndrome: a randomised, double-blind, placebo-controlled, phase 2b dose-finding trial. Lancet. 2022;399(10320):161-171. doi:10.1016/S0140-6736(21)02251-0 Shen N, Ignatenko S, Gordienko A, et al. Phase 2 Safety and Efficacy of Subcutaneous (s.c.) Dose Ianalumab (VAY736; Anti-BAFFR mAb) Administered Monthly over 28 Weeks in Patients with Systemic Lupus Erythematosus (SLE) of Moderate-to-Severe Activity [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 9). Accessed August 9, 2025. Bradbury C, Elverdi T, Trautmann K, et al. A phase 2 study of ianalumab in patients with primary immune thrombocytopenia previously treated with at least two lines of therapy (VAYHIT3). HemaSphere. 2025;9(Suppl 1):Abstract S238. Presented at European Hematology Association (EHA) Congress, June 12‑15, 2025. Milan, Italy. Accessed August 9, 2025. Novartis. Press release. Novartis to strengthen oncology pipeline with agreement to acquire MorphoSys AG for EUR 68 per share or an aggregate of EUR 2.7bn in cash. February 5, 2024. Accessed August 9, 2025. Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875-887. doi:10.1007/s12325-015-0240-z Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021;96(2):199-207. doi:10.1002/ajh.26083 # # # Novartis Media RelationsE-mail: Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
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Annual Perseid meteor shower to reach dazzling peak on Tuesday night
Shooting stars will be visible across the UK in the annual Perseid meteor shower that reaches its dazzling peak on Tuesday night. Every summer the Earth slams into a trail of debris from the Swift-Tuttle comet that also orbits the Sun. Specks as small as a grain of dust or rice flare up under the pressure of the planet's atmosphere to create shooting stars, said Royal Observatory Greenwich astronomer Dr Ed Bloomer. Around 150 meteors will cross the sky per hour but that amount will not be visible because the horizon blocks a full view of the sky, he added. However, an estimated 100 meteors could be seen per hour in certain locations that are particularly flat and dark. The meteor shower has been running for a few weeks and will continue until around August 24, the astronomer told the PA news agency. He said this means that 'we have lots of chances to see this' but 'you have to let your eyes adjust to the dark'. Dr Bloomer recommended stargazers wait half an hour to let their eyes get used to it, adding: 'Take a camping chair or something – if you had one, you would just sit down, and you would just relax, and you would just wait. 'You want to get away from city lights, you want to get away from street lamps. 'If you're looking out from your garden – it sounds obvious – but switch the kitchen light off, give yourself time to just put the phone away.' People struggling to see the meteors can turn and watch through their peripheral vision as it is 'a little bit better with low light conditions', he said. As it is summer, viewers will also have to wait until relatively late at night for it to be dark enough to see the celestial show. The astronomer added: 'For us, it's kind of one of the best (meteor showers), it's kind of reliable, it's long lived, it's quite dense… it's pretty active. 'You don't need to really be in a very specific location, the hourly rate is fairly high, so I think even beginners will be I think satisfied having seen them.' The weather is largely clear but early in the week the almost-full waning moon could make Perseid less visible. The meteor shower will be in the north-east as the sun is going down, Dr Bloomer added. He said: 'However, it's not available to everybody, because the further south you go… Perseid is lower and lower on the horizon. 'The primary interest is for Northern Hemisphere observers – Perseid is pretty low for us here in the UK, but it is above the horizon… in fact, it's above the horizon all day, but the problem is, of course, during the day, nothing's going to be visible.' Despite it being more visible in the north 'the dominant thing is going to be, can you get yourself in a dark location', he added. 'Getting into the middle of a field in the south east of England, in London, is going to be better than being in the middle of Aberdeen.' Shooting stars generally only last a second or two and sometimes appear in flurries, the astronomer said. Rarer meteors the size of a fist or basketball will produce longer tails and are known as fireballs, he added. These can last five to 10 seconds, but Dr Bloomer said he has only ever seen one. The level up is a bolide but 'that's a sort of national emergency type thing', he added.
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11 minutes ago
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Researchers raise alarm after extreme heat triggers bizarre animal behaviors: 'Can have negative consequences'
Researchers raise alarm after extreme heat triggers bizarre animal behaviors: 'Can have negative consequences' Across the United Kingdom, animals are displaying strange behaviors as another heat wave rolls in. Birds have been fluttering their throats and squirrels are "splooting" on the ground, but unfortunately, these cooling tactics are signs of distress and desperation. As the country weathers its third heat wave of the summer, researchers are warning that the effects on animals could carry long-term consequences for ecosystems, farms, and people. What's happening? According to the Guardian, unusual behaviors are being observed across the animal kingdom as temperatures rise. Birds like pigeons and herons are vibrating their throat muscles in a behavior known as gular fluttering to help them release body heat. Blackbirds are panting as squirrels are flattening themselves against shaded ground to reduce heat. Earthworms and snails are entering dormant states called aestivation to ride out the extreme heat. Farm animals are also showing distress. Cows have been bunching together, which actually worsens the heat for them and creates risks of mastitis, an udder infection, and hoof issues. Horses are struggling to stay cool despite sweating and are showing signs of heat exhaustion and electrolyte imbalances. "Because they don't use the barn area fully, the areas that they lie in can become wetter and dirtier, which increases their chance of getting mastitis. They also spend more time standing, which has consequences for their feet, legs, and lameness scores," said Dr. Zoe Barker, an agricultural scientist, per the Guardian. These responses may help animals survive short-term heat, but the cost is impaired cognition, delayed development, reduced feeding, and lower fertility rates. "Lots of animals will go and hide in the shade, which might save them from some of the bad effects of the heat, but it also means they're not out finding food or mates, which can have negative consequences if that heat is prolonged," said University of Exeter professor Alex Thornton, per the Guardian. Why is extreme heat concerning? Extreme heat is disrupting how animals live, reproduce, and interact with their environments. That leads to population declines, fewer pollinators, and less food security for people. A separate Guardian report said most young bees don't survive when nest temperatures climb above 36 degrees Celsius (96.8 degrees Fahrenheit), with the ideal range being closer to 28-32 degrees Celsius (82.4 to 89.6 degrees Fahrenheit). Worker bees try to cool things down by fanning their wings, but that doesn't always work when it's extremely hot. Fewer baby bees and pollinators mean a disruption in the growth of our food. What's being done to help animals stay cool? To help wildlife thrive, consider planting native species and leaving shady areas in your garden. Another good idea is making sure to leave shallow water dishes for birds, insects, and other animals. Letting some grass grow long can also offer shelter from the sun. For pet owners, make sure you're using pet-safe sunscreen, giving your pet lots of water, and keeping them indoors during the hottest hours of the day. According to the Guardian, agricultural scientists are focusing on more resilient infrastructure and revisiting building designs that are conducive to animal welfare. A cleaner and safer future is dependent on individual action, protecting green spaces, and cutting planet-warming gases so we can all better withstand the heat. Do you worry about air pollution in your town? All the time Often Only sometimes Never Click your choice to see results and speak your mind. Join our free newsletter for good news and useful tips, and don't miss this cool list of easy ways to help yourself while helping the planet. Solve the daily Crossword
