Medroxyprogesterone Acetate

Company plans to submit for regulatory approval later this year SAN RAFAEL, Calif., April 2, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. Safety results were consistent with the known profile of the medicine. PALYNZIQ is the first and only enzyme therapy approved to treat adults with PKU. Detailed results from the PEGASUS study will be presented at an upcoming medical meeting and submitted to global health authorities starting later this year to request a label expansion for PALYNZIQ to include adolescents. "For more than two decades, BioMarin has made strides for people with PKU – pioneering the first treatment, bringing a meaningful new option in PALYNZIQ, and continuing to innovate as we advance our research pipeline. We are encouraged to see these positive data that build on that legacy and show how PALYNZIQ can make an impact for adolescents as they begin their transition to adult living," said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. "We look forward to sharing these data with the scientific community and to submitting them to global regulators with the goal of making PALYNZIQ available for younger people living with PKU." About PEGASUS PEGASUS is a Phase 3 multi-center open-label randomized controlled study evaluating the safety and efficacy of PALYNZIQ compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria. The primary endpoints are change in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include change in total dietary protein intake and pharmacokinetics. The study is being conducted in two parts: the primary treatment phase ranging from weeks 1-73 (Part 1), and the extension phase (Part 2), which lasts for up to an additional 80 weeks of monitoring for the PALYNZIQ arm and allows for crossover for those in the diet-only arm. For more information, please visit clinicaltrials.biomarin.com. About PALYNZIQ PALYNZIQ substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe. PALYNZIQ is administered using a dosing regimen designed to facilitate tolerability; PALYNZIQ's safety profile consists primarily of immune-mediated responses, which can include anaphylaxis, for which robust risk management measures effective in clinical trials are in place. PALYNZIQ is approved to reduce blood Phe concentrations for adults in the U.S., for people 16 and older in the EU, Canada and Brazil, and for people 15 and older in Japan with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Patient Support Accessing PALYNZIQ To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-866-906-6100 or e-mail [email protected]. For more information about PALYNZIQ, please visit . For additional information regarding this product, please contact BioMarin Medical Information at [email protected]. About Phenylketonuria PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic condition affecting approximately 70,000 people in the regions of the world where BioMarin operates. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If functional enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU born after this period in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most individuals to adhere to the lifelong strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact. PALYNZIQ U.S. Indication and Important Safety Information PALYNZIQ® (pegvaliase-pqpz) is a phenylalanine (Phe)-metabolizing enzyme indicated to reduce blood Phe levels in adult patients with phenylketonuria who have uncontrolled blood Phe levels greater than 600 micromol/L on existing management. BOXED WARNING: RISK OF ANAPHYLAXIS Anaphylaxis has been reported after administration of PALYNZIQ and may occur at any time during treatment Administer the initial dose of PALYNZIQ under the supervision of a healthcare provider equipped to manage anaphylaxis, and closely observe patients for at least 60 minutes following injection. Prior to self-injection, confirm patient competency with self-administration, and patient's and observer's (if applicable) ability to recognize signs and symptoms of anaphylaxis and to administer auto-injectable epinephrine, if needed Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use Prescribe auto-injectable epinephrine. Prior to the first dose, instruct the patient and observer (if applicable) on its appropriate use. Instruct the patient to seek immediate medical care upon its use. Instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment PALYNZIQ is available only through a restricted program called PALYNZIQ REMS (Risk Evaluation and Mitigation Strategy). Further information, including a list of qualified pharmacies, is available at or by telephone at 1-855-758-REMS (1-855-758-7367) WARNINGS AND PRECAUTIONS Anaphylaxis Signs and symptoms of anaphylaxis reported include syncope, hypotension, hypoxia, dyspnea, wheezing, chest discomfort/chest tightness, tachycardia, angioedema (swelling of face, lips, eyes, tongue), throat tightness, skin flushing, rash, urticaria, pruritus, and gastrointestinal symptoms (vomiting, nausea, diarrhea) Anaphylaxis generally occurred within 1 hour after injection; however, delayed episodes occurred up to 48 hours after PALYNZIQ administration Consider having an adult observer for patients who may need assistance in recognizing and managing anaphylaxis during PALYNZIQ treatment. If an adult observer is needed, the observer should be present during and for at least 60 minutes after PALYNZIQ administration, should be able to administer auto-injectable epinephrine, and call for emergency medical support upon its use Anaphylaxis requires immediate treatment with auto-injectable epinephrine. Prescribe auto-injectable epinephrine to all patients receiving PALYNZIQ and instruct patients to carry auto-injectable epinephrine with them at all times during PALYNZIQ treatment. Prior to the first dose, instruct the patient and observer (if applicable) on how to recognize the signs and symptoms of anaphylaxis, how to properly administer auto-injectable epinephrine, and to seek immediate medical care upon its use. Consider the risks associated with auto-injectable epinephrine use when prescribing PALYNZIQ. Refer to the auto-injectable epinephrine prescribing information for complete information Consider the risks and benefits of readministering PALYNZIQ following an episode of anaphylaxis. If the decision is made to readminister PALYNZIQ, administer the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose. Subsequent PALYNZIQ dose titration should be based on patient tolerability and therapeutic response Consider premedication with an H1-receptor antagonist, H2-receptor antagonist, and/or antipyretic prior to PALYNZIQ administration based upon individual patient tolerability Other Hypersensitivity Reactions Hypersensitivity reactions other than anaphylaxis have been reported in 204 of 285 (72%) patients treated with PALYNZIQ in clinical trials Management of hypersensitivity reactions should be based on the severity of the reaction, recurrence of the reaction, and the clinical judgment of the healthcare provider, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and/or corticosteroids ADVERSE REACTIONS The most common adverse reactions (at least 20% of patients in either treatment phase) were injection site reactions, arthralgia, hypersensitivity reactions, headache, generalized skin reactions lasting at least 14 days, nausea, abdominal pain, vomiting, cough, oropharyngeal pain, pruritus, diarrhea, nasal congestion, fatigue, dizziness, and anxiety Of the 285 patients exposed to PALYNZIQ in an induction/titration/maintenance regimen in clinical trials, 44 (15%) patients discontinued treatment due to adverse reactions. The most common adverse reactions leading to treatment discontinuation were hypersensitivity reactions (6% of patients) including anaphylaxis (3% of patients), angioedema (1% of patients), arthralgia (4% of patients), generalized skin reactions lasting at least 14 days (2% of patients), and injection site reactions (1% of patients) The most common adverse reactions leading to dosage reduction were arthralgia (15% of patients), hypersensitivity reactions (9% of patients), injection site reactions (4% of patients), alopecia (3% of patients), and generalized skin reactions lasting at least 14 days (2% of patients) The most common adverse reactions leading to temporary drug interruption were hypersensitivity reactions (14% of patients), arthralgia (13% of patients), anaphylaxis (4% of patients), and injection site reactions (4% of patients) Angioedema and serum sickness: In clinical trials, 22 out of 285 (8%) patients experienced 45 episodes of angioedema (symptoms included: pharyngeal edema, swollen tongue, lip swelling, mouth swelling, eyelid edema, and face edema) occurring independent of anaphylaxis. In clinical trials, serum sickness was reported in 7 out of 285 (2%) patients Blood Phenylalanine Monitoring and Diet Obtain blood Phe levels every 4 weeks until a maintenance dosage is established. Periodically monitor blood Phe levels during maintenance therapy Counsel patients to monitor dietary protein and Phe intake, and adjust as directed by their healthcare provider DRUG INTERACTIONS Effect of PALYNZIQ on Other PEGylated Products In a single-dose study of PALYNZIQ in adult patients with PKU, two patients receiving concomitant injections of medroxyprogesterone acetate suspension (a formulation containing PEG 3350) experienced a hypersensitivity reaction. One of the two patients experienced anaphylaxis The clinical effects of concomitant treatment with different PEGylated products is unknown. Monitor patients treated with PALYNZIQ and concomitantly with other PEGylated products for hypersensitivity reactions including anaphylaxis USE IN SPECIFIC POPULATIONS Pregnancy and Lactation PALYNZIQ may cause fetal harm when administered to a pregnant woman Advise women who are exposed to PALYNZIQ during pregnancy or who become pregnant within one month following the last dose of PALYNZIQ that there is a pregnancy surveillance program that monitors pregnancy outcomes. Healthcare providers should report PALYNZIQ exposure and encourage these patients to report their pregnancy to BioMarin (1-866-906-6100) Monitor blood Phe levels in breastfeeding women treated with PALYNZIQ Pediatric Use The safety and effectiveness of PALYNZIQ in pediatric patients have not been established Geriatric Use Clinical studies of PALYNZIQ did not include patients aged 65 years and older You are encouraged to report suspected adverse reactions to BioMarin at 1-866-906-6100, or to FDA at 1-800-FDA-1088 or . Please see accompanying full Prescribing Information, including Boxed Warning. About BioMarin BioMarin is a global biotechnology company dedicated to translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that offer new possibilities for people living with genetically defined conditions around the world. To learn more, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the Phase 3 PEGASUS trial evaluating PALYNZIQ, including plans to present results at an upcoming medical conference; the safety profile and potential benefit of PALYNZIQ for adolescents, including ability to lower blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone; the development of BioMarin's PALYNZIQ program generally, including plans to submit detailed results to global health authorities later this year to request a label expansion for PALYNZIQ to include adolescents with the goal of making PALYNZIQ available to younger people living with PKU; and the continued clinical development of PALYNZIQ. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of PALYNZIQ; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2024, as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BioMarin RareConnections® and PALYNZIQ® are registered trademarks of BioMarin Pharmaceutical Inc. SOURCE BioMarin Pharmaceutical Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

作者 | 颜色编辑 | 凯西覆盖全国26个省，大型联盟集采来袭。 0126省联盟集采官宣涉50个药3月28日，山西省药械集中招标采购中心发布《关于做好二十六省联盟药品集中带量采购品种数据填报工作的通知》（以下简称《通知》），大型联盟集采开始报量。此前，一份《关于填报二十六省联盟药品集中带量采购品种采购需求量的通知》在业内流传——新疆牵头26省组成药品集采联盟，启动新一轮药品集中带量采购，共涉及50个药品，覆盖妇科及激素类、造影剂等重点品种。《通知》指出，通过新疆医保服务平台选择药品报量登录模块完成填报工作，所有公立医疗机构（含军队医疗机构）均应参加，其他医疗机构自愿参加。此次联盟成员包括新疆、山西、内蒙古、辽宁、黑龙江、上海、江苏、浙江、安徽、福建、江西、河南、湖北、湖南、广东、广西、海南、重庆、四川、贵州、云南、西藏、陕西、甘肃、青海、宁夏26个省，覆盖全国大部分地区，规模庞大。50个药中，妇科及激素类药品占比约60%，涉及避孕药、阴道抗感染用药、子宫内膜异位症治疗药等，包括奥硝唑阴道栓、醋酸甲地孕酮分散片、醋酸甲地孕酮胶囊、醋酸甲地孕酮片、醋酸甲地孕酮软胶囊、醋酸甲羟孕酮分散片等。此外，不少造影剂也被纳入，如碘比醇注射液、碘美普尔注射液等，主要用于医学影像检查。近些年来，造影剂在中国公立医疗机构终端有增长趋势，2024年上半年同比增长1.65%。妇科用药市场需求持续增长，米内网数据显示，2022年中国公立医疗机构妇科抗感染药销售额超20亿元。随着女性健康意识的提高，这类药物的需求增加，销售规模有望进一步扩容。由于部分妇科用药长期游离于医保支付边缘，患者负担较重，集采有望降低价格。不过，妇科用药集采并非一帆风顺。例如，在第八批国采中，常用的口服避孕药物左炔诺孕酮口服常释剂型遗憾流标。此次26省联盟集采最终能有多少妇科品种成功中选，仍需观察。接下来，信息填报工作即将开始。《通知》要求，医疗机构在2025年3月28日—2025年4月7日进行填报。填报时间结束后，不再接受数据补报。2024年4月10日前医保部门完成审核。山西要求，医疗机构依据品种目录，参考省级医保部门导入的采购平台历史采购数据等，准确填报未来一年每个产品的采购需求量，对填报的采购需求量与历史采购量差异较大的产品，需要作出说明。医疗机构填报口服剂型药品需求的，制剂年预采购量/包装数量须≥10（即至少10盒）；填报注射剂型药品需求的，制剂年预采购量须≥10（即至少10支）。二十六省联盟集采药品名单可在赛柏蓝公众号后台回复“50”获取~02地方开展全国联盟集采扩围集采品种总量增加今年年初，国家医保局表示，2025年，医保部门将持续深入推进药品、医用耗材集中带量采购，将在地方层面开展具备专业特色的全国联盟采购预计达到20个左右，包括中成药、中药饮片以及高值耗材等，预计2025年国家和联盟组织开展的药品集采品种将达到700个。更多地方联盟集采有望浮出水面。截至目前，新疆牵头的26省联盟集采是今年开展的较大规模的联盟集采之一。地方联盟集采正呈现快速发展趋势。一是覆盖范围持续扩大：从最初的化学药逐步拓展至中成药、生物药等领域，未来还将向专科用药和高值医用耗材延伸，实现全品类覆盖；二是联盟规模不断扩大：参与省份数量持续增加，通过扩大采购规模显著提升换价能力；三是规则体系日趋完善：采购规则不断优化细化，多地联盟集采已与国家集采、省级集采形成价格联动机制，推动全国价格协同；四是支付衔接更加紧密：医保支付标准与集采价格有效衔接，带动非集采品种同步降价。未来，地方联盟集采有望与国采形成互补，覆盖国采外更多品种。目前来看，今年已经开展的地方联盟集采包括——3月初，四川与重庆、贵州、云南、西藏、青海组成六省省际采购联盟，对环磷腺苷葡胺、尼可地尔、尼莫地平等66个化学药品启动带量联动采购，采购周期原则上为两年。此外，四川省医保局还发布了对氯化钠等大容量注射液展开带量联动采购的公告，四川、贵州、云南、西藏、青海五省组成联盟，对0.9%氯化钠注射液、葡萄糖氯化钠注射液、5%/10%/20%葡萄糖注射液3个品种集采，按包材、容量区分后共84个品规。部分地方联盟集采在近日宣布中选结果和接续采购。广东省对广东联盟双氯芬酸等药品集中带量采购和广东联盟常见病慢性病药品集中带量采购协议期满的药品展开接续集采，涉及246个品种；4月1日起，由广东牵头组织联盟省份开展的第三、四、五批国采协议期满的阿哌沙班片等48个药品的中选结果开始执行；全国中成药联盟集采将在4月落地...... 随着地方联盟集采的推进，未来可能覆盖更多未过评化药、中成药、中药饮片等。附：26省联盟集采药品名单END内容沟通：郑瑶（13810174402）左下角「关注账号」，右下角「在看」，防止失联