A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age

The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

开始日期2022-10-25

申办/合作机构

BioCryst Pharmaceuticals, Inc.

NCT04933721

/ Active, not recruiting临床3期

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

开始日期2021-07-23

申办/合作机构

BioCryst Pharmaceuticals, Inc.

NCT03873116

/ Completed临床3期

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema

This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan.

开始日期2018-12-27

申办/合作机构

BioCryst Pharmaceuticals, Inc.

100 项与 Berotralstat Hydrochloride 相关的临床结果

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100 项与 Berotralstat Hydrochloride 相关的转化医学

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100 项与 Berotralstat Hydrochloride 相关的专利（医药）

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项与 Berotralstat Hydrochloride 相关的文献（医药）

2024-12-01·PEDIATRIC ALLERGY AND IMMUNOLOGY

Hereditary angioedema in children: Review and practical perspective for clinical management

Review

作者: Blanc, Sibylle ; Boccon‐Gibod, Isabelle ; Bouillet, Laurence ; Carbasse, Aurélia ; Villedieu, Mona ; Nugues, Frédérique ; Bourgoin‐Heck, Mélisande ; Audouin‐Pajot, Christine ; Hoarau, Cyrille ; Dermesropian, Angelina ; Pagnier, Anne ; Voidey, Mélanie ; Jurquet, Anne‐Laure ; Reumaux, Héloïse ; Kevorkian‐Verguet, Charlotte

Abstract:

Background:

Hereditary angioedema (HAE) in children has specific features and requires multidisciplinary management.

Methods:

We performed a literature search and underwent in‐depth discussions to provide practical tools for physicians.

Results:

HAE is a rare, life‐threatening genetic disorder. Its epidemiology is poorly documented in children. Clinical manifestations usually appear during childhood or early adolescence. Classical signs, often preceded by prodromal symptoms, include transient, localized, non‐pitting, non‐pruritic swelling of deep dermal/subcutaneous or mucosal/submucosal tissues, leading to oedema of the extremities, face, lips, tongue, trunk and genitals, recurring gastrointestinal symptoms and laryngeal edema possibly causing asphyxiation and death. Diagnosis is often delayed due to low awareness in the medical community, and particularly challenging in case of isolated abdominal crises or atypical presentation and in neonates or infants. It relies on biological tests (measurement of serum/plasma levels of C1INH function, C1INH protein, and C4), genetic testing in selected cases, and imaging for differential diagnosis of acute abdominal crises. Main differential diagnosis for peripheral oedema is mast cell‐mediated oedema that accounts for 95% of angioedema in clinical practice. Quality of life can be significantly impaired. Disease management includes treatment of attacks, short‐term and long‐term prophylaxis, psychological support, avoidance of triggers, patients’ and parents’ education and coordination of all stakeholders, ideally within a specialized healthcare network. New plasma kallikrein inhibitors, namely lanadelumab (subcutaneous route) and berotralstat (oral route) have facilitated long‐term prophylaxis thanks to improved usability.

Conclusion:

Diagnostic and treatment of HAE are particularly challenging in children and require specific management by multiple stakeholders.

2024-09-01·Clinical and Translational Allergy

Advent of oral medications for the treatment of hereditary angioedema

Review

作者: Caballero, Teresa ; Frade, Joao P. ; Valerieva, Anna ; Audhya, Paul K. ; Magerl, Markus ; Craig, Timothy

Abstract:

Background:

Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, debilitating episodes of submucosal and/or subcutaneous tissue swelling, which may be life‐threatening depending on anatomic location. The two primary management strategies for HAE are ready access to effective on‐demand treatment in all patients and the prevention of attacks (short‐term prophylaxis [STP] and long‐term prophylaxis [LTP]) in appropriate patients. All approved on‐demand and most LTP medications require subcutaneous or intravenous administration. Injection‐related challenges include trypanophobia (fear of needles), difficulty with self‐administration, injection‐site reactions (e.g., pain, erythema, bleeding, bruising), and anxiety—all contributing to poor compliance and administration delays. Oral HAE treatments may improve outcomes by reducing treatment barriers.

Aim:

To review oral therapies, approved or in development, for on‐demand treatment and/or prevention of HAE attacks.

Materials and Methods:

To provide a comprehensive review, data was obtained from publicly available resources through a targeted PubMed literature review and supplemented by information provided on company websites (search cutoff of May 31, 2024).

Results:

Berotralstat, an oral plasma kallikrein (PKa) inhibitor, is approved for LTP. Sebetralstat, another PKa inhibitor, is the investigational first oral on‐demand HAE treatment to complete a phase 3 trial. Deucrictibant, an oral bradykinin B2 receptor antagonist, has completed phase 2 trials for on‐demand therapy and LTP. Several other oral PKa inhibitors (ATN249, VE‐4666, and VE‐4062) are in early development for LTP.

Conclusion:

Substantial advances have been made in the development of oral treatments for HAE. These treatments have the potential to improve and optimize clinical outcomes, satisfaction, and quality of life among patients with HAE.

2024-06-01·CELL BIOCHEMISTRY AND BIOPHYSICS

Unveiling therapeutic frontiers: DON/DRP-104 as innovative Plasma kallikrein inhibitors against carcinoma-associated hereditary angioedema shocks - a comprehensive molecular dynamics exploration

Article

作者: Oduro-Kwateng, Ernest ; Soliman, Mahmoud E S

Abstract:

Human plasma kallikrein (PKa) is a member of the serine protease family and serves as a key mediator of the kallikrein-kinin system (KKS), which is known for its regulatory roles in inflammation, vasodilation, blood pressure, and coagulation. Genetic dysregulation of KKS leads to Hereditary Angioedema (HAE), which is characterized by spontaneous, painful swelling in various body regions. Importantly, HAE frequently coexists with various cancers. Despite substantial efforts towards the development of PKa inhibitors for HAE, there remains a need for bifunctional agents addressing both anti-cancer and anti-HAE aspects, especially against carcinoma-associated comorbid HAE conditions. Consequently, we investigated the therapeutic potential of the anti-glutamine prodrug, isopropyl(S)-2-((S)-2-acetamido-3-(1H-indol-3-yl)-propanamido)-6-diazo-5-oxo-hexanoate (DRP-104), and its active form, 6-Diazo-5-oxo-l-norleucine (DON), recognized for their anti-cancer properties, as novel PKa inhibitors. Utilizing structure-based in silico methods, we conducted a comparative analysis with berotralstat, a clinically approved HAE prophylactic, and sebetralstat, an investigational HAE therapeutic agent, in Phase 3 clinical trials. Inhibiting PKa with DON resulted in relatively heightened structural stability, rigidity, restricted protein folding, and solvent-accessible loop exposure, contributing to increased intra-atomic hydrogen bond formation. Conversely, PKa inhibition with DRP-104 induced restricted residue flexibility and significantly disrupted the critical SER195-HIS57 arrangement in the catalytic triad. Both DON and DRP-104, along with the reference drugs, induced strong cooperative intra-residue motion and bidirectional displacement in the PKa architecture. The results revealed favorable binding kinetics of DON/DRP-104, showing thermodynamic profiles that were either superior or comparable to those of the reference drugs. These findings support their consideration for clinical investigations into the management of carcinoma-associated HAE.

203

项与 Berotralstat Hydrochloride 相关的新闻（医药）

2024-12-21

·漫游药化

FDA批准上市的38个小分子药物结构

FDA今年很忙，但是再忙也未敢忘批药，截至2021年1月1日，FDA批准了53款新药，其中有38个小分子药物，作为化学人一定会很好奇这些化合物有啥特点，结构是啥，小编今天就带大家先睹为快。 1，Avapritinib (BLU-285) 阿伐普利尼 Avapritinib (BLU-285)是一种小分子激酶抑制剂，可有效抑制PDGFRα D842V突变体的活性、在细胞背景下抑制PDGFRα D842V自我磷酸化（IC50=30 nM）；同时也是KIT突变（KIT D816V）的抑制剂（IC50=0.5 nM）。 2，Tazemetostat (EPZ-6438) Tazverik(tazemetostat) (EPZ-6438) 是组蛋白赖氨酸甲基转移酶（Histone-Lysine N-methyltransferase）EZH2的抑制剂。 3，Pizensy(乳糖醇) Pizensy是一种口服的乳果糖类似物。它是一种渗透性泻药（osmotic laxitaive），可促进水分流入肠道，进而在结肠内达到通便作用。 4，Nexletol (bempedoic acid) bempedoic acid由美国Esperion Therapeutic 公司研发，是一种治疗血脂异常及降低其他心血管疾病风险的新型小分子化合物。合成路线如下： 5，Barhemsys(氨磺必利) 氨磺必利是一个多巴胺D2和D3受体阻断剂，上世纪80年代开始应用于精神分裂症治疗。 6，Nurtec ODT（rimegepant） Biohaven公司的口服CGRP抑制剂rimegepant，用于成人偏头痛的急性治疗。 7， Isturisa(osilodrostat) 3月6日，FDA批准库欣综合征（Cushing syndrome，CS）一线用药Osilodrostat上市，商品名：Isturisa，患者无论是否接受受过垂体切除手术治疗，都可应用Osilodrostat作为治疗药物。Osilodrostat是FDA批准的首个11-β羟化酶抑制，可直接阻断肾上腺皮质醇合成。 8，Zeposia(ozanimod) ozanimod（商品名ZEPOSIA），用于成人复发性MS（RRMS）患者的治疗。 9，Selumetinib（司美替尼） Selumetinib （司美替尼）（ARRY-142886 ; AZD6244）是一种MEK 1/2抑制剂和潜在的新药，由AlstraZeneca于2003年获得Array BioPharma Inc.的许可。阿斯利康和MSD于2017年签订了selumetinib的共同开发和联合商业化协议。 10，Tukysa(Tucatinib) Tukysa（Tucatinib）妥卡替尼 /图卡替尼 (ONT-380)是一种口服TKI(酪氨酸激酶)抑制剂，对HER2具有高度特异性，但对同属人表皮生长因子受体家族的EGFR没有明显抑制作用。 11，Pemigatinib （帕米加替尼） Pemigatinib（INCB054828）是一种口服有效的，选择性FGFR抑制剂，它对FGFR1，FGFR2，FGFR3，FGFR4的IC50值分别为0.4 nM，0.5 nM，1.2 nM和30 nM。 12，Ongentys(opicapone) 13，INCB28060 (Capmatinib) 卡马替尼；苯扎米特 INCB28060是一种新型， ATP竞争性的c-MET抑制剂，IC50为0.13 nM，抑制RONβ， EGFR和HER-3活性。 14，Selpercatinib (LOXO-292) 塞尔帕替尼 Selpercatinib（LOXO-292）是一种有效的，特异性RET抑制剂。 15，Ripretinib 瑞普替尼 Ripretinib（DCC-2618）是一种具有口服活性的，选择性的KIT和PDGFR-alpha抑制剂。 16， Cerianna (fluoroestradiol F18) 17，Artesunate（青蒿琥酯）青蒿琥酯是一类控制疟疾症状的抗疟药。 18，Tauvid（flortaucipir F18） Tauvid（flortaucipir F18）是第一种获得FDA批准，帮助对大脑中的tau病理进行成像的药物。Tauvid是一种放射性诊断试剂，用于需要接受阿尔茨海默病评估的认知障碍成人患者。 19，Lurbinectedin（卢比克替定） lurbinectedin是PharmaMar研发的海鞘素衍生物，为肿瘤创新药。目前，绿叶制药与PharmaMar在华合作共同开发该药物。 20，Dojolvi（triheptanoin）三庚酸甘油酯 Dojolvi（triheptanoin）三庚酸甘油酯，是一种高纯度、人工合成的7碳脂肪酸甘油三酯，在甘油骨架上通过多个化学合成步骤添加了三个7碳脂肪酸形成。专门为LC-FAOD患者提供中链、奇数碳脂肪酸作为能源和代谢产物替代品。 21，Fostemsavir(BMS-663068) Fostemsavir (BMS-663068) 是 BMS-626529 的原药，能结合gp120，抑制 HIV-1 与细胞 CD4 受体的结合。 22，Byfavo（Remimazolam）瑞米唑仑/雷米马唑仑 Byfavo（Remimazolam）瑞米唑仑/雷米马唑仑是一种起效和失效迅速的静脉注射苯二氮卓（benzodiazepine）镇静剂。可用于在时长不超过30分钟的有创医疗程序中，例如结肠镜检查和支气管镜检查。 23，Inqovi(cedazuridine+地西他滨) Inqovi（cedazuridine/decitabine[地西他滨]，口服C-DEC，ASTX727），该药用于骨髓增生异常综合症（MDS）和慢性骨髓单核细胞白血病（CMML）成人患者的治疗。 24，Xeglyze(Abametapir) Xeglyze（Abametapir）0.74％洗护剂是一种金属蛋白酶抑制剂，金属蛋白酶是对虫卵发育和若虫和成虫虱子存活，至关重要的生理过程所必需的酶。 25，Nifurtimox (Synonyms: 硝呋替莫) Nifurtimox 是一种用于锥虫病 (Trypanosoma cruzi) 的抗虫剂。Nifurtimox 有潜力用于神经母细胞瘤细胞的研究。Nifurtimox 影响乳酸脱氢酶 (LDH) 活性。 26，Olinvyk（oliceridine） Olinvyk的活性药物成分为oliceridine，这是一种首创的（first-in-class）静脉镇痛药。oliceridine是首个G蛋白选择性激动剂，与静脉注射吗啡相比，镇痛效果更好。oliceridine以μ阿片受体为靶点，通过一种优化作用机制（MOA），优先参与负责疗效的信号通路，减少引起不良反应的信号通路的激活。 27， Evrysdi(risdiplam) risdiplam是治疗所有3种类型SMA的首个口服药物。 28，Winlevi(clascoterone) Clascoterone（又称CB-03-01）是一种局部雄激素受体（AR）抑制剂，该新的化学实体正在开发治疗痤疮（暂定品牌名为Winlevi）和男女性脱发（暂定品牌名Breezula）。根据美国FDA审查其1%浓度治疗痤疮以及后期开发其高浓度溶液治疗男性雄激素雄性脱发的结果，其被认为是一种“first-in-class”药物。值得一提的是，这是近40年来针对痤疮治疗的首个新机制药物。 29，Detectnet（Cu 64 DOTATATE）放射性诊断试剂铜64盐酸盐 Detectnet是首款将Cu 64放射性同位素与生长抑素类似物DOTATATE偶联的诊断试剂。 30，Pralsetinib 普雷西替尼 Pralsetinib (Blu-667) 是新一代的，高效的，选择性RET抑制剂，对于WT RET，RET突变体V804L，V804M，M918T和CCDC6-RET的IC50为0.3-0.4 nM。 31，瑞德西韦（Remdesivir） 2020年10月22日，美国食品药品管理局（FDA）批准了吉利德科学的抗病毒药物瑞德西韦用于治疗新冠住院患者，成为美国首个正式获批的新冠治疗药物。 32，Lonafarnib (Synonyms: Sch66336) Lonafarnib 是一种口服有效的法尼基蛋白转移酶 (FPTase) 抑制剂，作用于 H-ras，K-ras 和 N-ras，IC50 分别为 1.9 nM，5.2 nM 和 2.8 nM。Lonafarnib 具有抗肝炎三角洲病毒 (HDV) 的活性。 33， Imcivree(Setmelanotide) setmelanotide是一款首创、寡肽类MC4R激动剂，开发用于治疗罕见肥胖遗传性疾病。 34，Gallium 68 PSMA-11 35，Orladeyo（Berotralstat）贝罗司他 rladeyo（Berotralstat）贝罗司他是一种新型、口服、每日1次、强效、选择性人血浆激肽释放酶抑制剂，通过抑制激肽释放酶来阻止缓激肽的生成从而预防水肿发作。Orladeyo （berotralstat）用于预防12岁及以上儿童和成人遗传性血管性水肿（HAE）发作，Orladeyo是首个预防HAE发作的口服非甾体药物。 36，Tirbanibulin (Synonyms: KX2-391; KX-01) 37，Relugolix 瑞卢戈利 Relugolix，也称为TAK-385，是一种口服活性的黄体激素释放激素（LH-RH）受体拮抗剂，IC50为0.12 nM。 38,Gemtesa（vibegron） vibegron可治疗膀胱过度活动症导致的急迫性尿失禁，尿急和尿频等症状。

上市批准临床结果

2024-12-04

·GlobeNewswire-Health Care

BioCryst Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

RESEARCH TRIANGLE PARK, N.C., Dec. 04, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the compensation committee of BioCryst’s board of directors granted five newly-hired employees stock options to purchase an aggregate of 17,400 shares, and restricted stock units (RSUs) covering an aggregate of 12,150 shares, of BioCryst common stock. The options and RSUs were granted as of November 29, 2024, as inducements material to each employee entering into employment with BioCryst. The options and RSUs were granted in accordance with Nasdaq Listing Rule 5635(c)(4). The options have an exercise price of $7.50 per share, which is equal to the closing price of BioCryst common stock on the grant date. The options and RSUs vest in four equal annual installments beginning on the one-year anniversary of the grant date, in each case subject to the new employee’s continued service with the company. Each stock option has a 10-year term. The options and RSUs are subject to the terms and conditions of BioCryst’s Inducement Equity Incentive Plan and a stock option agreement or restricted stock unit agreement, as applicable, covering the grant. About BioCryst PharmaceuticalsBioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com

蛋白降解靶向嵌合体

2024-11-18

·GlobeNewswire-Health Care

BioCryst Launches ORLADEYO® (berotralstat) in Ireland

RESEARCH TRIANGLE PARK, N.C., Nov. 18, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Health Services Executive (HSE) in Ireland has recommended ORLADEYO® (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older. With this recommendation, HAE patients in Ireland will have access to the first oral, once-daily therapy for the reduction of recurrent HAE attacks. “The positive HSE recommendation of ORLADEYO broadens access to modern prophylaxis, providing greater choice for prescribing physicians and potentially a better quality of life for HAE patients in Ireland,” said Charlie Gayer, chief commercial officer of BioCryst. The HSE decision in Ireland follows the European Commission marketing authorization of ORLADEYO in April 2021. To date, ORLADEYO is licensed in 44 countries. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATIONAn increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch/ Please see full Prescribing Information. The Irish Summary of Product Characteristics (SPC) and Patient Information Leaflet (PIL) for ORLADEYO (berotralstat) can be found through medicines.ie. Anyone can report suspected side effects to the HPRA. You can submit a report by visiting the HPRA’s reporting webpage to complete an online form hpra.ie/report. Adverse events should also be reported to BioCryst Pharmaceuticals at medinfoeurope@biocryst.com or +353 1 699 4405. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding BioCryst’s plans and expectations for ORLADEYO. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance and demand; the FDA, HSE, or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; and risks related to the international expansion of BioCryst’s business. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact: John Bluth+1 919 859 7910jbluth@biocryst.com Niamh Lyons+353 87 639 7083nlyons@biocryst.com

上市批准

100 项与 Berotralstat Hydrochloride 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11673 D11674	-	B06AC	DB15982

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
遗传性血管性水肿	美国	BioCryst Pharmaceuticals, Inc.	2020-12-03

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
I型和II型遗传性血管性水肿	临床3期	美国	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	澳大利亚	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	丹麦	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	德国	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	以色列	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	意大利	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	西班牙	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	瑞士	BioCryst Pharmaceuticals, Inc.	2018-02-16
I型和II型遗传性血管性水肿	临床3期	英国	BioCryst Pharmaceuticals, Inc.	2018-02-16

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ACAAI2024 人工标引	N/A	遗传性血管性水肿	257	Berotralstat	選醖醖築製廠獵遞襯鬱(壓觸鹽餘鏇願衊範觸構) = 艱獵網繭鹽獵膚觸膚築廠顧窪窪網網鹽鬱選鑰 (憲憲觸糧範構構顧鹹鑰 ) 更多	积极	2024-10-24
				Lanadelumab	選醖醖築製廠獵遞襯鬱(壓觸鹽餘鏇願衊範觸構) = 壓簾憲餘顧餘齋積選襯廠顧窪窪網網鹽鬱選鑰 (憲憲觸糧範構構顧鹹鑰 ) 更多
ACAAI2024 人工标引	N/A	遗传性血管性水肿	466	Berotralstat (patients with C1-inhibitor deficiency)	憲繭選鑰製壓顧憲顧構(糧餘齋蓋獵襯製膚築襯) = 遞廠鹽蓋窪鑰簾積製築顧繭壓願鹹艱壓範糧網 (鏇醖獵襯範衊願鹽襯築 ) 更多	积极	2024-10-24
ACAAI2024 人工标引	N/A	遗传性血管性水肿	353	Berotralstat (HAE-nl-C1-INH)	襯選鹹鬱鹹獵築衊鑰鏇(選構簾衊選齋願網觸襯) = 遞醖築築鏇鏇衊範範夢遞憲鹹襯夢鹹衊顧憲餘 (觸簾願鏇艱製積顧鹽淵 ) 更多	积极	2024-10-24
ACAAI2024	N/A	遗传性血管性水肿 C1-inhibitor deficiency-	466	Berotralstat	襯衊鏇積夢醖襯觸鑰糧(壓襯範糧觸齋壓蓋範艱) = 顧襯簾膚艱積鏇築齋觸壓糧構壓壓蓋窪蓋憲遞 (選繭鏇衊網獵窪顧憲鹹, -1.92 ~ -1.18)	积极	2024-10-24
Optime Care Specialty Pharmacy (ACAAI2024)	N/A	遗传性血管性水肿 C1-inhibitor levels/function within normal laboratory reference range(s)-	353	Berotralstat	壓艱廠窪簾艱襯選願壓(廠齋鏇鹹糧淵艱構膚遞) = 觸淵積簾鑰憲構醖艱蓋淵衊選築廠選壓壓壓觸 (鹽窪衊鬱衊膚壓積選獵, -3.10 ~ -1.85)	积极	2024-10-24
ACAAI2024	N/A	遗传性血管性水肿	357	Berotralstat	艱顧糧壓觸夢鏇艱衊構(醖鬱積壓淵餘構獵淵築) = 遞網網襯鹽範蓋夢願製衊範淵簾鹽膚衊餘鹽齋 (遞襯夢鹹餘繭糧願鑰鏇 )	积极	2024-10-24
				Lanadelumab	艱顧糧壓觸夢鏇艱衊構(醖鬱積壓淵餘構獵淵築) = 淵鏇獵繭廠遞獵繭膚蓋衊範淵簾鹽膚衊餘鹽齋 (遞襯夢鹹餘繭糧願鑰鏇 )
GlobeNewswire 人工标引	N/A	遗传性血管性水肿	260	ORLADEYO® (berotralstat)	窪選鏇願壓膚簾鬱簾窪(鏇憲艱願衊鹽壓襯製繭) = 艱窪觸蓋鹹觸糧鹽顧鹽夢鏇觸簾廠鬱衊顧憲積 (鬱餘膚獵淵遞選鑰顧積 ) 更多	积极	2024-05-09
				ORLADEYO® (berotralstat) (long-term prophylaxis)	窪選鏇願壓膚簾鬱簾窪(鏇憲艱願衊鹽壓襯製繭) = 鬱獵鏇鏇壓鹹蓋廠簾鹹夢鏇觸簾廠鬱衊顧憲積 (鬱餘膚獵淵遞選鑰顧積 ) 更多
NCT03873116 (APeX-J, Pubmed \| World Allergy Organ J)	临床3期	遗传性血管性水肿	17	Berotralstat 150 mg	製淵壓觸襯襯顧膚廠構(鹹繭鑰構遞構鏇壓憲構) = 淵衊淵壓憲選膚鹹觸簾遞淵壓醖簾製鹹鑰鹹遞 (壓鏇願襯窪襯窪窪餘糧, 0.09) 更多	积极	2024-03-01
AAAAI2024	N/A	遗传性血管性水肿Ⅲ型一线 C1-inhibitor deficiency	-	Berotralstat (HAE-C1INH)	觸選窪網築構醖鏇觸鹽(艱糧觸範築淵範夢襯齋) = 餘憲廠範網築鹽選襯積憲齋壓鹹顧衊簾構鏇廠 (鏇餘襯餘窪鬱窪壓範憲, 0.33 ~ 3.33) 更多	积极	2024-02-23
				Berotralstat (HAE-nl-C1INH)	觸選窪網築構醖鏇觸鹽(艱糧觸範築淵範夢襯齋) = 願醖餘廠願窪遞選憲遞憲齋壓鹹顧衊簾構鏇廠 (鏇餘襯餘窪鬱窪壓範憲, 1.33 ~ >3.33) 更多
AAAAI2024	N/A	HAE Type I/II	335	Berotralstat 110 mg	餘簾膚夢襯遞憲齋範選(觸衊壓齋積衊憲蓋衊鏇) = 蓋簾鑰壓積遞築鬱憲醖簾艱廠鑰淵獵鬱網積鬱 (衊襯積積襯醖餘蓋製夢 )	积极	2024-02-23
				Berotralstat 150 mg	餘簾膚夢襯遞憲齋範選(觸衊壓齋積衊憲蓋衊鏇) = 襯糧製鏇獵鬱觸齋製鹹簾艱廠鑰淵獵鬱網積鬱 (衊襯積積襯醖餘蓋製夢 )