BioCryst Pharmaceuticals, Inc.

The FDA has signed off on three drugs in the last two months to treat the rare genetic condition hereditary angioedema. The newest to come online is Ionis Pharmaceuticals' Dawnzera. Since 2008, the FDA has approved 11 treatments for a rare genetic condition called hereditary angioedema (HAE), three of which have occurred in the last two months.The latest drug to join this cohort of medicines arrived Thursday with the FDA approval of Ionis Pharmaceuticals’ Dawnzera (donidalorsen). Despite entering a competitive, crowded market, it shouldn’t be difficult for Dawnzera to differentiate itself. As the first RNA-targeted treatment for HAE, it is a first-in-class therapy and further distinguishes itself with its dosing schedule and ability to be injected at home.The U.S. regulator has signed off on Dawnzera for prophylactic use by those age 12 years and older to reduce the likelihood of the sudden, unpredictable episodes of severe swelling in the limbs, face, abdomen and larynx that characterize HAE. The disorder, which affects 7,000 people in the U.S., can be fatal when it strikes the airways and inhibits breathing.Dawnzera was designed to target plasma prekallikrein, a protein that activates inflammatory mediators associated with acute attacks of HAE.Paving the way for approval was a phase 3 trial in which Dawnzera reduced the HAE attack rate by 81% compared to placebo over 24 weeks. The attack rate reduction increased to 87% when measured beyond the second dose of Dawnzera.Dawnzera is the third in a run of recent approvals that give patients new options to combat the disease. The products will take on Takeda’s blockbuster standard of care, an injected treatment called Takhzyro, and BioCryst Pharmaceuticals’ fast-rising oral challenger Orladeyo.“Orladeyo has less benefit than Takhzyro, with respect to reducing attack rates, and it has tolerability issues, particularly (gastrointestinal) because it is oral,” Ionis CEO Brett Monia, Ph.D., said in an interview with Fierce. “With those two drugs alone, we’re seeing that patients are switching from one drug to another trying to get a better treatment. My point is—they’re unsatisfied.”Last month, the FDA endorsed Ekterly, the first approved product from small Massachusetts biotech KalVista Pharmaceuticals, which has become the first oral on-demand medicine to address the symptoms of HAE, while others are delivered by intravenous or subcutaneous administration.Adding to the competition in the HAE prophylactic space is CSL, which scored a June FDA nod for Andembry, a self-administered prefilled pen. The treatment is dosed monthly and inhibits factor XIIa, a plasma protein that plays a key role in triggering the swelling episodes.“It’s a quite different mechanism of action, with respect to the targets its going after—we’re targeting RNA, they’re targeting a protein with an antibody,” Monia said of Andembry. “It looks like a good drug. We think donidalorsen has an overall profile that will be more attractive to many patients.” CSL will be a formidable rival as it has vast experience in the HAE space. Its drugs Berinert and Haegarda, which were approved in 2009 and 2017, respectively, were the premier HAE treatments before Takhzyro became a blockbuster in 2022.Ionis believes Dawnzera’s advantage is in its dosing regimen, which calls for patients to begin with one injection every four weeks, with an option to switch to dosing every eight weeks. In an extension study of Ionis’ phase 3 trial, eight-week dosing performed at the same level as four-week dosing, limiting swelling episodes by 94% versus placebo.Meanwhile, Takhzyro starts patients on a two-week dosing schedule, with the option to switch to every four weeks. Andembry follows a monthly dosing regimen.Ionis will charge $57,462 per dose, which works out to a $747,000 price tag for annual injections, assuming a patient stays on the four-week schedule. While the annual pricing exceeds that charged by Takeda and CSL for their prophylactic treatments, it will be significantly less for patients who shift to the eight-week dosing plan.“We’re right in line with where the market is today,” Kyle Jenne, Ionis’ chief global product strategy officer, said during a post-approval webcast Thursday. “We expect the majority of (physicians) to start patients at every four weeks and then if they’re performing well and are well controlled, they could potentially have the option of moving them to every eight weeks. We’ll have to see what that split looks like over time.” Ionis also pointed to a switch cohort in its phase 3 study that evaluated the performance of Dawnzera in patients previously treated with Takhzyro, Orladeyo or Takeda's C1 inhibitor Cinryze. In switching to Dawnzera, patients reduced their mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch.Additionally, a total of 84% of patients surveyed preferred Dawnzera over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions.The switch data are not included on Dawnzera’s label but will “remain important for Ionis’s messaging on market positioning and reimbursement,” according to William Blair analyst Myles Minter, Ph.D.“Overall, we see Dawnzera’s profile as superior to current prophylactic options, with less frequent dosing and at-home auto-injector dosing being further differentiators. We also view Dawnzera’s profile as comparable to CSL Behring’s Andembry,” Minter added in an Aug. 21 note.

The Food and Drug Administration on Thursday approved a drug Ionis Pharmaceuticals developed for the rare genetic disease hereditary angioedema, making the therapy, known as donidalorsen, the third new medicine to reach market this year for the rare genetic condition.Donidalorsen, which Ionis will sell under the brand name Dawnzera, is approved to prevent the swelling attacks associated with hereditary angioedema in adults and children at least 12 years of age. Dawnzera has a list price of $57,462 per dose, company executives said in a conference call.The price is based on the efficacy, the data and the supporting evidence, Chief Global Product Strategy Officer Kyle Jenne told analysts on the call.The payers, we believe, will be very accepting of the price, since it's in line with the other products that are in the HAE space today.HAE is a rare, inherited condition estimated to affect about 1 in every 50,000 people worldwide. The disease is characterized by recurrent swelling attacks that can last for days if untreated, and most commonly affect the limbs, face, gastrointestinal tract and throat. These episodes can be life-threatening.Despite its rarity, HAE has become a crowded area of drug research. Prior to Dawnzerasapproval, several therapies were already available to treat or prevent these attacks. Two Kalvista Pharmaceuticals Ekterly and CSL Behrings Andembry made it to market this year. A gene editing medicine is in late-stage testing, too.That competition will likely limit the size of Dawnzeras opportunity, wrote BMO Capital Markets analyst Kostas Biliouris, in a Thursday note to investors. Biliouris projects about $520 million in peak yearly sales.Ionis, though, believes Dawnzera could become the preferred choice for patients looking to prevent swelling attacks. Current options, like the once-monthly injection Andembry and Biocryst Pharmaceuticals pill Orladeyo, reduce the rate of attacks, but dont ward them off completely. In a poll Ionis conducted this year, a majority of patients contacted hadnt yet found their best preventive option.Patients are looking for better efficacy, better convenience and better tolerability, said CEO Brett Monia, in an interview. At least one of those boxes are not checked with existing treatments, he added, noting as evidence how people with HAE often bounce around from one treatment to another over the course of a year.Dawnzera is different. Its the first RNA-targeting medicine for HAE, and works by reducing levels of prekallikrein, a protein implicated in the onset of swelling attacks. Phase 3 results published in The New England Journal of Medicine last year showed that, among patients who received injections every four weeks, monthly attack rates were on average 81% lower than among those who received placebo. An every-other-month dose was associated with a 55% reduction versus placebo.Monia also pointed to results from a study testing Dawnzera in patients who switched from another preventive therapy. That study found further reductions in swelling attack frequency among those who changed to Dawnzera. About 84% of trial participants also said they preferred Ionis treatment, citing convenience and better symptom management.We don't see any reason why [Dawnzera]would not be the preferred first-line treatment for those patients who are newly diagnosed, Monia said. However, given the majority of patients are already taking preventive therapies, the company will focus its marketing efforts there, hoping the results its accrued will convince people to switch medications.The most common side effects associated with treatment were injection site reactions, urinary and upper respiratory tract infections and abdominal discomfort. The drugs prescribing information includes a warning for serious hypersensitivity reactions, including anaphylaxis, which came after the occurrence of two such instances in an open-label extension study. Both cases resolved quickly, though, and Ionis doesnt think the safety warning will have a meaningful impact on usage, said Kenneth Newman, senior vice president of clinical development.Dawnzera is the second approved medicine the company will sell on its own. A pioneer in developing RNA medicines called antisense oligonucleotides, the company has long been known for partnering its products instead of commercializing them. But under Monia, who became CEO in 2020, the company has changed course.Since shifting its strategy, the company has launched one drug on its own, Tryngolza, for a rare disorder called familial chylomicronemia syndrome. Multiple others are in late-stage testing.I felt it was going to be like turning around an aircraft carrier, Monia said, but the employees, the scientists, everybody at Ionis was ready for this.Editors note: This story has been updated with pricing information and other details from a conference call with Ionis executives. '

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。 8月12日，CDE官网显示，哈尔滨三联药业股份有限公司按3类化药注册申报的帕拉米韦注射液上市申请已获受理。据摩熵医药数据库显示，该药物近两年院内市场销售额超10亿元。 截图来源：CDE官网 帕拉米韦是新一代神经氨酸酶抑制剂，适用于全年龄段甲型或乙型流行性感冒患者的治疗。该药物由日本盐野义从美国BioCryst公司引进，2010年在日本获批上市，商品名为“Rapiacta”。 截图来源：摩熵医药全球药物研发数据库 2014年12月，帕拉米韦注射液在美国获批上市，是FDA唯一批准的抗流感注射剂，也是继扎那米韦和奥司他韦之后的又一新型流感病毒NA抑制剂。 据摩熵医药数据库显示，2022年帕拉米韦于院内市场斩获5.24亿元销售额，同比增长25.55%；2023年突破10亿大关，实现翻倍式增长。2024年销售额攀升至11.46亿元，处于当前最高水平。 截图来源：摩熵医药全国医院（全终端）销售数据库 在国内市场，帕拉米韦上市剂型涵盖帕拉米韦注射液与帕拉米韦氯化钠注射液。其中，帕拉米韦注射液的生产批文涉及35家企业，包括齐鲁制药、扬子江药业、中国大冢制药、石药集团等；而帕拉米韦氯化钠注射液的生产企业仅有2家，即广州南新制药和山东齐都药业。 截图来源：摩熵医药中国药品批文数据库 2013年，南新制药的帕拉米韦氯化钠注射液在国内获批上市。因原研产品未进入中国市场，该领域长期由南新制药主导。当前，该企业占据帕拉米韦94.49%的市场份额。 截图来源：摩熵医药全国医院（全终端）销售数据库 2023年3月，中润药业的帕拉米韦注射液获批上市，成为国内首仿。同年6月，扬子江药业集团四川海蓉药业获批，视同过评，成为国内第二家获得该品种仿制批文的企业。 截图来源：摩熵医药一致性评价数据库 当前还有浙江康吉尔药业、广州一品红制药、山东益康药业等10家企业的帕拉米韦注射液在审评审批中。 截图来源：摩熵医药中国药品审评数据库 摩熵医药数据显示，帕拉米韦原料药目前已有12家企业完成备案流程。其中有10家的产品处于 “A” 状态，即已通过审评，或与制剂成功实现关联。 截图来源：摩熵医药CDE原料药、药用辅料和药包材登记信息数据库 2025年4月7日上海阳光医药采购网公示，取消山西阳和医药帕拉米韦注射液中选资格，将该企业及生产企业山西国润制药列入违规名单，暂停这两家企业2025年4月8日至2026年10月7日参与国家药品集采的申报资格。 截图来源：上海阳光医药采购网 该企业取消中选资格后，山西、黑龙江、重庆备供厂家分别为广州绿十字、吉林四环、陕西博森生物，中选价分别为14.70元（60ml:0.3g1袋）、5.55元（20ml:0.2g1支）、5.99元（15ml:0.15g*1支）。 截图来源：摩熵医药药品集中采购数据库 作为年销售额超10亿元的抗流感病毒药物，帕拉米韦的市场竞争已趋激烈。从其市场格局的动态演变中不难发现，抗流感药物领域既充满机遇，也面临规范与竞争的双重考验。随着更多企业的深入布局，期待这一领域能在创新与合规的平衡中持续发展，为临床提供更优质、可及的治疗选择。 END 本文为原创文章，转载请留言获取授权 近期热门资源获取 中国临床试验趋势与国际多中心临床展望-202505 2024年医药企业综合实力排行榜-202505 中国带状疱疹疫苗行业分析报告-202505 2023H2-2024H1中国药品分析报告-202504 数据透视：中药创新药、经典验方、改良型新药、同名同方的申报、获批、销售情况-202503 2024年中国1类新药靶点白皮书-202503 中国AI医疗健康企业创新发展百强榜单-202502 解码护肤抗衰：消费偏好洞察与市场格局分析-202502 2024年FDA批准上市的新药分析报告-202501 小分子化药白皮书（上）-202501 2024年中国医疗健康投融资全景洞察报告-202501 2024年医保谈判及市场分析报告-202501 近期更多摩熵咨询热门报告，识别下方二维码领取 联系我们，体验摩熵医药更多专业服务 会议 合作 园区 服务 数据库 咨询 定制 服务 媒体 合作 点击上方图片，即可开启摩熵化学数据查询 点击阅读原文，申请摩熵医药企业版免费试用！