A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

开始日期2024-04-02

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT05234567

/ RecruitingN/A

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

开始日期2022-08-25

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT04189315

/ Withdrawn临床4期

A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia

The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).

开始日期2020-04-01

申办/合作机构

Alexion Pharmaceuticals, Inc.

100 项与阿司福酶α 相关的临床结果

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100 项与阿司福酶α 相关的专利（医药）

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144

项与阿司福酶α 相关的文献（医药）

2025-12-01·Current Osteoporosis Reports

Medical Management of Hypophosphatasia: Review of Data on Asfotase Alfa

Review

作者: Dunbar, Nancy S ; Dahir, Kathryn McCrystal

Abstract :

Purpose:

Hypophosphatasia (HPP) is a rare, dento-osseous disorder caused by impaired activity of tissue non-specific alkaline phosphatase (TNSALP), a key enzyme in tissue mineralization. This review provides a clinical perspective on the current medical treatment of both children and adults with HPP.

Recent Findings:

Dental problems, rickets in children, and osteomalacia in adults are common in HPP. However, disease manifestations in individual patients are exceptionally variable. Recent studies broadened our understanding of HPP symptoms. For example, data showed behavioral health challenges in HPP children, and a large, real-world data set from the Global HPP Registry demonstrated that HPP adults regardless of the time of disease onset exhibit significant disease burden and are broadly affected by non-skeletal impairments, such as pain and chronic fatigue. Treatment for HPP relies on the enzyme replacement asfotase alfa. Small, mostly pediatric trials initially established dosing, safety and efficacy of asfotase alfa, and latest data corroborated the long-term safety and efficacy in both children and pediatric-onset adults. Data from several recent observational studies, including the Global HPP Registry, underscored that asfotase alfa improves physical functions, non-skeletal symptoms such as pain, and quality-of-life (QoL) in adults irrespective of age-of-onset. Clinical use of asfotase alfa is based on prescribing information and evidence-based consensus guidelines. However, recommendations for initiation of therapy are just emerging. Alternatives to asfotase alfa remain limited, but a derivative, efzimfotase alfa, currently undergoes clinical testing.

Summary:

Studies in larger HPP patient populations suggest efficacy of enzyme replacement therapy independent of patient age and time of disease onset.

2025-04-03·Expert Opinion On Drug Safety

Disproportionality analysis of adverse events associated with asfotase alfa: a post-marketing study using the FDA Adverse Event Reporting System

Article

作者: Wen, Wang ; Jing, Han ; Haixia, Sun ; Hao, Lv ; Qianxiu, Chen ; Zongchao, Fu ; Zhe, Wang

BACKGROUND:

Asfotase alfa (AA) is an FDA-approved enzyme replacement therapy for hypophosphatasia (HPP). Limited real-world data on its adverse events (AEs) exist. This study evaluates AA-related AEs using the U.S. FDA's Adverse Event Reporting System (FAERS) database.

METHODS:

Reports for AA were extracted from FAERS and analyzed per FDA guidelines. AEs were categorized using MedDRA version 26.1. Disproportionality analysis was conducted using the reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and empirical Bayes geometric mean (EBGM) algorithms. Time-to-onset (TTO) was calculated, with a Weibull shape parameter test to assess risk over time.

RESULTS:

Out of 13,702,373 reports, 5,040 AA-related AEs were identified, with 198 significant preferred terms (PTs). Common AEs included injection site reactions and pain, with additional PTs for ear/labyrinth disorders and infections. The median onset for 234 AEs with reported times was 170 days (IQR 18-390 days). No significant differences in AEs were found across gender or age groups.

CONCLUSION:

Most AEs align with known data, but newly identified ear/labyrinth disorders and infections require further investigation to enhance AA's safety profile.

2025-02-04·Cureus Journal of Medical Science

Long-Term Outcomes of Early Enzyme Replacement Therapy With Asfotase Alfa in Perinatal Benign Hypophosphatasia: Amelioration of Bone Deformities in a Young Child

Article

作者: Kobayashi, Masahisa ; Terayama, Shuntaro ; Oishi, Kimihiko ; Suemitsu, Tokumasa ; Samura, Osamu

Hypophosphatasia (HPP) is a congenital skeletal dysplasia. Enzyme replacement therapy (ERT) improves survival and bone mineralization of patients with perinatal severe HPP. However, there are few reports of ERT in patients with perinatal benign HPP, and its long-term efficacy remains unclear. Herein, we report the case of a boy with perinatal benign HPP who was initiated on early ERT with asfotase alpha. The patient was suspected of having HPP because of shortened limbs and deformed long bones on fetal 3D-CT. He was diagnosed with HPP based on clinical manifestations, low serum alkaline phosphatase levels, and ALPL gene variants. At the age of two years and three months, he had muscle weakness and motor developmental delay requiring support to walk, and ERT was initiated. His muscle strength improved immediately, and he could walk independently two months after starting ERT. Shortening and bowed limbs improved, and his body height increased from -3.48 SD (at the age of two years and three months) to -1.71 SD (at the age of nine years and eight months) after starting ERT. Hence, early ERT effectively improves motor development, bone deformity, and short stature in patients with perinatal benign HPP.

项与阿司福酶α 相关的新闻（医药）

2025-02-06

·Business Wire

AstraZeneca’s Full Year and Q4 2024 results

CAMBRIDGE, England--( BUSINESS WIRE )--AstraZeneca: Revenue and EPS summary FY 2024 % Change Q4 2024 % Change $m Actual CER i $m Actual CER - Product Sales 50,938 16 19 13,362 18 19 - Alliance Revenue 2,212 55 55 714 68 69 - Collaboration Revenue 923 56 54 815 >2x >2x Total Revenue 54,073 18 21 14,891 24 25 Reported EPS $4.54 18 29 $0.97 56 71 Core ii EPS $8.21 13 19 $2.09 44 49 Financial performance for FY 2024 (Growth numbers at constant exchange rates) Total Revenue up 21% to $54,073m, driven by a 19% increase in Product Sales, continued growth of partnered medicines (Alliance Revenue) and the achievement of sales-based milestones (Collaboration Revenue) Total Revenue growth from Oncology was 24%, CVRM 20%, R&I 25%, V&I 8% and Rare Disease 16% Core EPS increased 19% to $8.21 Second interim dividend declared of $2.10 per share, making a total annual dividend declared for FY 2024 of $3.10 per share, an increase of 7%. Dividend to be further increased in FY 2025 Guidance for FY 2025: Total Revenue is expected to increase by a high single-digit percentage and Core EPS is expected to increase by a low double-digit percentage, both at CER Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “ Our company delivered a very strong performance in 2024 with Total Revenue and Core EPS up 21% and 19% respectively. We also delivered nine positive high value Phase III studies in the year, which coupled with increasing demand for our medicines in all key regions, will help sustain our growth momentum into 2025. This year marks the beginning of an unprecedented, catalyst-rich period for our company, an important step on our Ambition 2030 journey to deliver $80 billion Total Revenue by the end of the decade. In 2025 alone, we anticipate the first Phase III data for seven new medicines, along with several important new indication opportunities for our existing medicines. We are also investing in and making significant progress with transformative technologies that have the potential to drive our growth well beyond 2030, many of which have now entered pivotal trials.” Key milestones achieved since the prior results announcement Positive read-outs for Truqap in combination with abiraterone and androgen deprivation therapy in PTEN -deficient de novo metastatic hormone-sensitive prostate cancer (CAPItello-281) and Tagrisso with or without chemotherapy in resectable early-stage EGFR m NSCLC (NeoADAURA) US approvals for Imfinzi in limited-stage small cell lung cancer (ADRIATIC), Calquence in combination with bendamustine and rituximab in mantle cell lymphoma (ECHO), Datroway (datopotamab deruxtecan) in HR+ HER2- metastatic breast cancer (TROPION-Breast01) and Enhertu in chemotherapy-naïve HER2-low and -ultralow metastatic breast cancer (DESTINY-Breast06). EU approvals for Tagrisso in unresectable EGFR m NSCLC (LAURA) and Kavigale for prevention of COVID-19 (SUPERNOVA). Japan approvals for Imfinzi in endometrial cancer (DUO-E), Lynparza plus Imfinzi in pMMR endometrial cancer (DUO-E), Calquence tablet formulation in chronic / small lymphocytic leukaemia, Datroway in HR+ HER2- metastatic breast cancer, Fasenra in EGPA (MANDARA) and Kavigale for prevention of COVID-19. China approvals for Lynparza in gBRCAm HER2- early breast cancer (OlympiA), Orpathys in locally advanced or metastatic MET Exon 14 NSCLC (NCT04923945) Guidance The Company issues its Total Revenue and Core EPS guidance for FY 2025 at CER, based on the average foreign exchange rates through 2024. Total Revenue is expected to increase by a high single-digit percentage Core EPS is expected to increase by a low double-digit percentage The Core Tax rate is expected to be between 18-22% The Company is unable to provide guidance on a Reported basis because it cannot reliably forecast material elements of the Reported results, including any fair value adjustments arising on acquisition-related liabilities, intangible asset impairment charges and legal settlement provisions. Please refer to the cautionary statements section regarding forward-looking statements at the end of this announcement. Currency impact If foreign exchange rates for February 2025 to December 2025 were to remain at the average rates seen in January 2025, it is anticipated that Total Revenue in FY 2025 would incur a low single-digit percentage adverse impact compared to the performance at CER, and Core EPS would incur a mid-single-digit percentage adverse impact. The Company’s foreign exchange rate sensitivity analysis is provided in Table 17. Capital allocation In FY 2025, the Company intends to increase the annual dividend declared to $3.20 per share. The Company also expects to increase capital expenditure iii by approximately 50%, driven by manufacturing expansion projects and investment in IT systems, to support portfolio growth and build capacity for transformative technologies. China In relation to the illegal drug importation allegations, in January 2025, AstraZeneca received a Notice of Transfer to the Prosecutor and an Appraisal Opinion from the Shenzhen City Customs Office regarding suspected unpaid importation taxes amounting to $0.9 million. To the best of AstraZeneca’s knowledge, the importation taxes referred to in the Appraisal Opinion relate to Imfinzi and Imjudo . A fine of between one and five times the amount of unpaid importation taxes may also be levied if AstraZeneca is found liable. AstraZeneca continues to fully cooperate with the Chinese authorities. In December 2024 AstraZeneca announced the appointment of Iskra Reic as Executive Vice President, International, which encompasses China, Asian and Eurasian markets, Middle East & Africa, Latin America, Australia & New Zealand. Iskra succeeds Leon Wang who is on extended leave from the Company while under investigation in China. Table 1: Key elements of Total Revenue performance in Q4 2024 % Change Revenue type $m Actual % CER % Product Sales 13,362 18 19 Alliance Revenue 714 68 69 $392m Enhertu (Q4 2023: $281m) $133m Tezspire (Q4 2023: $80m) $161m Beyfortus (Q4 2023: $41m) Collaboration Revenue 815 >2x >2x $600m Lynparza (Q4 2023: $245m) $111m Beyfortus (Q4 2023: $27m) $100m Koselugo (Q4 2023: nil) Total Revenue 14,891 24 25 Therapy areas $m Actual % CER % Oncology 6,344 27 29 Tagrisso up 20% (21% at CER), Calquence up 20%, Enhertu up 48% (54% at CER) CVRM 3,138 16 17 Farxiga up 21% (22% at CER) , Lokelma up 35% R&I 2,127 27 28 Breztri up 29%. Saphnelo up 65%, Tezspire up 86% (85% at CER), Symbicort up 31% (33% CER) V&I 651 58 55 Beyfortus Total Revenue up >3x Rare Disease 2,377 21 22 Ultomiris up 32% (33% at CER), partially offset by decline in Soliris of 24% (22% at CER), Strensiq up 38% (37% at CER) and Koselugo up >3x Other Medicines 254 (7) (6) Total Revenue 14,891 24 25 Regions $m Actual % CER % US 6,532 28 28 Product Sales up 25% Emerging Markets 3,134 13 19 - China 1,364 (1) (3) Decline primarily due to low rates of seasonal respiratory viral infections, and impact from year-end hospital budget dynamics - Ex-China Emerging Markets 1,770 26 42 Europe 3,948 37 35 Product Sales up 20% (18% at CER) Established RoW 1,277 1 2 Total Revenue 14,891 24 25 Key alliance medicines Combined sales of Enhertu , recorded by Daiichi Sankyo Company Limited (Daiichi Sankyo) and AstraZeneca, amounted to $3,754m in FY 2024 (FY 2023: $2,566m) Combined sales of Tezspire , recorded by Amgen and AstraZeneca, amounted to $1,219m in FY 2024 (FY 2023: $653m) Table 2: Key elements of financial performance in Q4 2024 Metric Reported Reported change Core Core change Comments iv Total Revenue $14,891m 24% Actual 25% CER $14,891m 24% Actual 25% CER See Table 1 and the Total Revenue section of this document for further details Product Sales Gross Margin 80% Stable Actual +1pp CER 79% -1pp Actual Stable CER Variations in Product Sales Gross Margin can be expected between periods, due to product seasonality, foreign exchange fluctuations and other effects R&D expense $4,677m 52% Actual 52% CER $3,573m 23% Actual 22% CER Increased investment in the pipeline Core R&D-to-Total Revenue ratio of 24% (Q4 2023: 24%) Reported R&D includes $753m impairment recorded against the vemircopan (ALXN2050) intangible asset SG&A expense $5,410m 1% Actual 1% CER $4,275m 6% Actual 7% CER Market development for recent launches and pre-launch activities Core SG&A-to-Total Revenue ratio of 29% (Q4 2023: 34%) Other operating income and expense v $100m -7% Actual -6% CER $101m -7% Actual -6% CER Operating Margin 14% +3pp Actual +4pp CER 28% +5pp Actual +6pp CER See commentary above on Gross Margin, R&D, SG&A and Other operating income and expense Net finance expense $365m 9% Actual 8% CER $310m 20% Actual 20% CER Recent debt issued at higher interest rates Decrease in interest income Higher level of Net debt Tax rate 10% +17pp Actual +15pp CER 16% +7pp Actual +7pp CER Variations in the tax rate can be expected between periods EPS $0.97 56% Actual 71% CER $2.09 44% Actual 49% CER Further details of differences between Reported and Core are shown in Table 12 Table 3: Pipeline highlights since prior results announcement Event Medicine Indication / Trial Event Regulatory approvals and other regulatory actions Tagrisso EGFR m NSCLC (Stage III unresectable) (LAURA) Regulatory approval (EU, CN) Imfinzi Limited-stage SCLC (ADRIATIC) Regulatory approval (EU) Imfinzi Advanced endometrial cancer Regulatory approval (JP) Calquence Tablets for chronic lymphocytic leukaemia Regulatory approval (JP) Calquence Mantle cell lymphoma (1st-line) (ECHO) Regulatory approval (US) Lynparza + Imfinzi Advanced endometrial cancer with mismatch repair proficiency (DUO-E) Regulatory approval (JP) Lynparza gBRCAm HER2- eBC (OlympiA) Regulatory approval (CN) Enhertu HR+ HER2-low and -ultralow mBC (DESTINY-Breast06) Regulatory approval (US) Datroway HR+ HER2- mBC (TROPION-Breast01) Regulatory approval (JP, US) Orpathys MET exon 14 skipping altered NSCLC (NCT04923945) Regulatory approval (CN) Fasenra EGPA (MANDARA) Regulatory approval (JP) Kavigale Prevention of COVID-19 (SUPERNOVA) Regulatory approval (EU, JP) Regulatory submissions or acceptances* Imfinzi Muscle-invasive bladder cancer (NIAGARA) Regulatory submission (US, JP) Imfinzi + Imjudo NSCLC (1st-line) (POSEIDON) Regulatory submission (CN) Calquence Chronic lymphocytic leukaemia (1st-line) (AMPLIFY) Regulatory submission (EU) Datroway EGFR m NSCLC (later line) (TROPION-Lung05) Regulatory submission (US) Tezspire Severe uncontrolled asthma (NAVIGATOR/ DIRECTION) Regulatory submission (CN) Koselugo Neurofibromatosis type 1 adult (KOMET) Regulatory submission (EU, JP) Phase III / registrational data readouts and other developments Tagrisso Resectable early-stage EGFR m NSCLC (NeoADAURA) Primary endpoint met Truqap PTEN -deficient de novo metastatic hormone-sensitive prostate cancer (CAPItello-281) Primary endpoint met *US, EU and China regulatory submission denotes filing acceptance Other pipeline updates In January 2025, the vemircopan (ALXN2050) Phase II development programme was terminated. The decision was based on safety and efficacy data from Phase II trials. Upcoming pipeline catalysts For recent trial starts and anticipated timings of key trial readouts, please refer to the Clinical Trials Appendix, available on www.astrazeneca.com/investor-relations.html . Sustainability highlights The Company convened an event on health equity for investors and analysts in November that detailed AstraZeneca’s health equity strategy, which is embedded from the Company’s science through to healthcare delivery and community engagement. At the end of 2024, the Company’s cumulative reduction in Scope 1 and 2 greenhouse gas (GHG) emissions was 77.5% from the 2015 baseline. Conference call A conference call and webcast for investors and analysts will begin today, 6 February 2025, at 11:00 UK time. Details can be accessed via astrazeneca.com . Reporting calendar The Company intends to publish its Q1 2025 results on 29 April 2025. To read AstraZeneca's Full Year and Q4 2024 Financial Results press release in full, click here .

临床2期财报高管变更上市批准临床3期

2024-12-02

·蒲公英Ouryao

最新粤港澳大湾区内地九市临床急需进口港澳药品医疗器械目录

转自：广东省药监局编辑：水晶 12月2日，广东省药监局网省消息，广东省药监局和广东省卫健委发布粤港澳大湾区内地九市临床急需进口港澳药品医疗器械目录（2024年）的通告。《通告》指出，本批目录收录34个上市药品及37个医疗器械。对已列入目录的药械产品，将于2024年12月1日起按照《条例》第九条进行管理。 34个药品：卡那奴单抗、维莫德吉胶囊、盐酸维那卡兰注射液、阿仑珠单抗、罗氟司特片、氨己烯酸薄膜衣片、卡博替尼薄膜衣片、阿培利司薄膜衣片、厄达替尼片、注射用羟钴胺素、布西珠单抗、伊匹木单抗注射液、巴氯芬注射液、艾沙妥昔单抗注射用浓缩液、肾上腺素注射液（预充笔）、莱博雷生片、瑞玛奈珠单抗、硫酸瑞美吉泮口崩片、注射用坦昔妥单抗、二十碳五烯酸乙酯软胶囊、阿扎胞苷片、曲美木单抗、注射用芦比替定、阿司福酶α注射液、阿伏利尤单抗、特泽利尤单抗、磷酸芦可替尼乳膏、罗莫佐单抗、重组人促卵泡素α/促黄体素α注射液、注射用重组人促卵泡素α/促黄体素α、艾普奈珠单抗、伊曲莫德片、依洛硫酸酯酶α注射液、维替索妥尤单抗。根据《广东省粤港澳大湾区内地九市进口港澳药品医疗器械管理条例》（以下简称《条例》）中对急需港澳药械实施目录管理的要求，广东省药品监督管理局、广东省卫生健康委员会经研究决定发布广东省粤港澳大湾区内地九市临床急需港澳药品医疗器械目录（2024年）。本批目录将按《国家药品监督管理局国家卫生健康委员会关于临床急需境外新药审评审批相关事宜的公告》（2018年第79号）发布并符合《条例》要求的港澳已上市药品及《条例》实施前已批准的共71个药品及医疗器械收录。对已列入目录的药械产品，将于2024年12月1日起按照《条例》第九条进行管理。特此通告。附件：1.广东省粤港澳大湾区内地九市临床急需进口港澳药品目录（2024年） 2.广东省粤港澳大湾区内地九市临床急需进口港澳医疗器械目录（2024年）广东省药品监督管理局广东省卫生健康委员会 2024年11月29日

2024-09-12

·蒲公英Ouryao

【广东】第7批大湾区临床急需进口港澳药品目录发布

来源：广东药监局编辑：wangxinglai2004 2024年09月11日，广东药监发布了《第七批粤港澳大湾区内地临床急需进口港澳药品医疗器械目录通告》，涉及药品10种。根据《粤港澳大湾区药品医疗器械监管创新发展工作方案》（国市监药〔2020〕159号）和《广东省粤港澳大湾区内地临床急需进口港澳药品医疗器械管理暂行规定》（粤药监规许〔2021〕4号）要求，结合工作实际，广东省药品监督管理局、广东省卫生健康委员会决定发布第七批粤港澳大湾区内地临床急需进口港澳药品医疗器械目录（以下简称目录）。涉及药品名称：阿扎胞苷片、曲美木单抗、注射用芦比替定、罗培干扰素α-2b注射液、泊那替尼片、阿司福酶α注射液、阿伏利尤单抗、索米妥昔单抗、磷酸芦可替尼乳膏、特泽利尤单抗；一、药品目录二、医疗器械目录

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外链

KEGG	Wiki	ATC	Drug Bank
D10595	阿司福酶α	A16AB13	DB09105

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适应症	国家/地区	公司	日期
婴儿低磷酸酯脢症	美国	Alexion Pharmaceuticals, Inc.	2015-10-23
低碱性磷酸酶血症	日本	Alexion Pharmaceuticals, Inc.	2015-07-03

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适应症	最高研发状态	国家/地区	公司	日期
儿童低磷酸酯脢症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2016-06-06
儿童低磷酸酯脢症	临床2期	德国	Alexion Pharmaceuticals, Inc.	2016-06-06
遗传性热变性异形红细胞增多症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	加拿大	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	阿拉伯联合酋长国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	英国	Alexion Pharmaceuticals, Inc.	2008-09-01

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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


ESPE2023	N/A	低碱性磷酸酶血症 alkaline phosphatase (ALP) \| PEA \| B6 vitamin	-	Asfotase alfa	鹹憲壓鑰膚簾遞膚醖蓋(醖壓衊壓鹽窪選醖廠網) = 憲觸簾壓糧壓廠簾鹽網夢網範製構壓鹽鑰顧網 (鬱鏇積構觸鹽鑰膚鹽製 ) 更多	-	2023-09-21
UK Managed Access Agreement (WCO_IOF_ESCEO2023)	N/A	低碱性磷酸酶血症	25	Asfotase alfa	鏇積簾窪願觸餘網齋鏇(襯鏇範繭網遞範鑰窪繭) = 衊網衊鬱鹽鹽構廠遞糧襯鏇膚廠膚糧衊艱壓繭 (築鑰願膚糧夢構襯蓋積 )	积极	2023-05-04
WCO_IOF_ESCEO2023	N/A	儿童低磷酸酯脢症 myelinotoxic activity (MTA)	110	ASFOTASE ALFA (Group 1)	醖鹹繭艱簾衊遞鏇窪築(鹹選簾構憲鑰艱廠築窪) = 襯襯膚窪製壓淵襯膚憲鹹憲鹹網廠獵憲鑰壓範 (鏇淵窪顧窪顧鏇膚鬱構, 0.7)	积极	2023-05-04
WCO_IOF_ESCEO2023	N/A	儿童低磷酸酯脢症 myelinotoxic activity (MTA)	110	ASFOTASE ALFA (Group 2)	醖鹹繭艱簾衊遞鏇窪築(鹹選簾構憲鑰艱廠築窪) = 鑰獵簾範壓構鬱獵範選鹹憲鹹網廠獵憲鑰壓範 (鏇淵窪顧窪顧鏇膚鬱構, 0.81)	积极	2023-05-04
PROSPECTIVE STUDY (WCO_IOF_ESCEO2023)	N/A	-	50	Asfotase alfa (AA)	襯觸齋顧製衊構築築艱(願憲鹹窪遞夢範網構壓) = 憲衊構觸糧鏇衊繭製製鬱網獵觸範淵夢遞夢觸 (繭鹹憲膚襯網獵醖繭膚 ) 更多	积极	2023-05-04
UK Managed Access Agreement (WCO_IOF_ESCEO2023)	N/A	低碱性磷酸酶血症	25	Asfotase alfa	窪憲蓋鏇構餘壓廠鹽構(鏇窪鹹簾艱築艱夢遞範) = 夢築繭衊淵顧範繭糧襯範鏇範構壓觸鹹遞夢鑰 (廠鏇簾窪構觸壓廠積齋 )	积极	2023-05-04
PROSPECTIVE STUDY (WCO_IOF_ESCEO2023)	N/A	儿童低磷酸酯脢症	50	Asfotase alfa	襯範簾襯齋範夢鹽積鹹(願積襯積選繭夢鹽糧網) = 艱襯觸蓋繭獵衊網鬱鹽鏇襯顧蓋膚淵獵衊餘顧 (蓋艱顧鏇窪窪窪夢餘廠 ) 更多	积极	2023-05-04
NCT01176266 (Pubmed \| J Clin Endocrinol Metab) 人工标引	临床2期	低碱性磷酸酶血症	69	Asfotase Alfa	蓋鬱鹹積憲簾獵鹹蓋襯(襯鬱齋繭遞鑰蓋膚艱鏇) = 構築壓鹹壓醖積齋襯蓋網鑰遞餘鏇製願鹽構網 (獵艱範糧淵鹽網衊遞鬱 ) 更多	积极	2019-07-01
NCT01205152 \| EUCTR2009-009369-32-GB (Pubmed) 人工标引	临床2期	低碱性磷酸酶血症	11	Asfotase alfa	窪壓壓鹽鹹網鑰鹹壓築(鑰構築製選觸築齋衊鹹) = 鬱積積醖構淵壓壓遞簾艱築選顧積網鹹衊願構 (膚餘鬱衊憲鑰積構鑰鏇 ) 更多	积极	2019-02-01
ESPE2018	N/A	低碱性磷酸酶血症 ALPL	-	Asfotase Alfa	蓋憲醖構鬱糧繭壓淵鹽(淵鏇鹹築窪選繭廠醖膚) = The patient needed a wheelchair even for in-home movement, suffered from continuous skeletal pain, had undetectable ALP serum levels and extremely high plasma PLP (997 mcg/l) 製簾夢鹽艱鬱艱壓糧鏇 (積憲製衊鬱廠壓鏇遞窪 )	-	2018-09-27
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 0.5 mg/kg Dose)	夢廠築醖製衊積齋鏇襯(衊齋淵廠淵鏇餘鑰繭遞) = 構憲鏇醖願獵衊蓋獵艱築積餘鏇蓋鏇鑰憲選餘 (製糧鏇願鹽糧鹹壓繭選, 0.2399) 更多	-	2018-07-10
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 2.0 mg/kg Dose)	夢廠築醖製衊積齋鏇襯(衊齋淵廠淵鏇餘鑰繭遞) = 遞衊壓積遞選願願顧選築積餘鏇蓋鏇鑰憲選餘 (製糧鏇願鹽糧鹹壓繭選, 0.1949) 更多	-	2018-07-10