更新于：2024-11-01
Cardiomyopathy, Hypertrophic, Familial
家族性肥厚性心肌病
更新于：2024-11-01
基本信息
别名 Asymmetric Septal Hypertrophy, Familial、CARDIOMYOPATHY, FAMILIAL HYPERTROPHIC、CMH + [30] |
简介 An autosomal dominant inherited form of HYPERTROPHIC CARDIOMYOPATHY. It results from any of more than 50 mutations involving genes encoding contractile proteins such as VENTRICULAR MYOSINS; cardiac TROPONIN T; ALPHA-TROPOMYOSIN. |
关联
3
项与 家族性肥厚性心肌病 相关的药物作用机制 CPT1A抑制剂 [+1] |
原研机构- |
最高研发阶段批准上市 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
靶点- |
作用机制- |
在研适应症 |
非在研适应症- |
最高研发阶段早期临床1期 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
作用机制 心肌肌球蛋白复合体抑制剂 |
在研机构 |
原研机构 |
在研适应症 |
非在研适应症- |
最高研发阶段临床前 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
13
项与 家族性肥厚性心肌病 相关的临床试验NHLBI SESAME (SEptal Scoring Along Midline Endocardium) Early Feasibility Study
Background:
Some people have a condition in which the wall (septum) that separates the two main pumping chambers of the heart is too thick. This thick septum causes a condition called "left ventricular outflow tract obstruction" (LVOTO), which reduces blood flow out of the heart. LVOTO can cause serious heart disease; symptoms may include shortness of breath, chest pain, heart failure, or death. Researchers want to find better ways to treat LVOTO.
Objective:
To test a new procedure where excess tissue is sliced away from the septum in people with LVOTO. This procedure is called "septal scoring along midline endocardium" (SESAME).
Eligibility:
Adults aged 21 years with LVOTO.
Design:
Participants will have baseline tests. They will have imaging scans and tests of their heart structure and function. They will take a walking test and answer questions about how their heart condition affects their life.
Participants will stay in the hospital 2 to 6 days for the SESAME procedure.
They will be completely or partially asleep for the procedure. A tube will be inserted into the mouth and down the throat to take pictures of the heart. Pictures may also be taken with a tube inserted inside the heart.
Next, tubes will be inserted into the groin and guided through the blood vessels up to the heart. Guidewires will be inserted into the heart. Doctors will watch the path the wires take with x-rays and ultrasound. When the wire is in the correct place, it will be electrified to slice excess tissue away from the septum.
Participants will have 3 follow-up visits within 1 year.
Some people have a condition in which the wall (septum) that separates the two main pumping chambers of the heart is too thick. This thick septum causes a condition called "left ventricular outflow tract obstruction" (LVOTO), which reduces blood flow out of the heart. LVOTO can cause serious heart disease; symptoms may include shortness of breath, chest pain, heart failure, or death. Researchers want to find better ways to treat LVOTO.
Objective:
To test a new procedure where excess tissue is sliced away from the septum in people with LVOTO. This procedure is called "septal scoring along midline endocardium" (SESAME).
Eligibility:
Adults aged 21 years with LVOTO.
Design:
Participants will have baseline tests. They will have imaging scans and tests of their heart structure and function. They will take a walking test and answer questions about how their heart condition affects their life.
Participants will stay in the hospital 2 to 6 days for the SESAME procedure.
They will be completely or partially asleep for the procedure. A tube will be inserted into the mouth and down the throat to take pictures of the heart. Pictures may also be taken with a tube inserted inside the heart.
Next, tubes will be inserted into the groin and guided through the blood vessels up to the heart. Guidewires will be inserted into the heart. Doctors will watch the path the wires take with x-rays and ultrasound. When the wire is in the correct place, it will be electrified to slice excess tissue away from the septum.
Participants will have 3 follow-up visits within 1 year.
开始日期2024-11-04 |
Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life.
Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.
Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.
开始日期2022-09-01 |
申办/合作机构 |
Hearts in Rhythm Organization Hypertrophic Cardiomyopathy Registry, Biobank and Imaging Data Repository (HiRO-HCM)
The Hearts in Rhythm Organization (HiRO) is a national network of Canadian researchers/clinicians, working towards a better understanding of the rare genetic causes of sudden cardiac death (SCD). The HiRO Hypertrophic Cardiomyopathy registry, biobank and imaging data repository (HiRO-HCM) is a multicenter study that will prospectively enroll patients with HCM as well as those carrying sarcomeric gene variants predisposing to HCM.
The objectives of HiRO-HCM are:
to better understand the natural history of the disease and identify clinical markers and biomarkers for adverse outcomes;
to derive and validate risk prediction models for disease expression, complications and response to therapy;
to better define the genetic architecture of sarcomeric and non-sarcomeric HCM.
The objectives of HiRO-HCM are:
to better understand the natural history of the disease and identify clinical markers and biomarkers for adverse outcomes;
to derive and validate risk prediction models for disease expression, complications and response to therapy;
to better define the genetic architecture of sarcomeric and non-sarcomeric HCM.
开始日期2021-02-23 |
申办/合作机构 |
100 项与 家族性肥厚性心肌病 相关的临床结果
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100 项与 家族性肥厚性心肌病 相关的转化医学
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0 项与 家族性肥厚性心肌病 相关的专利（医药）
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2,841
项与 家族性肥厚性心肌病 相关的文献（医药）2024-12-01·Science of The Total Environment
Airborne polystyrene nanoplastics exposure leads to heart failure via ECM-receptor interaction and PI3K/AKT/BCL-2 pathways
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2024-11-01·Heart
Comatose patient with asymmetric septal hypertrophy
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30
项与 家族性肥厚性心肌病 相关的新闻（医药）2023-10-25
TN-201 Being Developed for the Potential Treatment of MYBPC3-associated HCM
Dosing in Phase 1 Clinical Trial Expected to Commence in Q3 2023
SOUTH SAN FRANCISCO, Calif., May 02, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM).
TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-based investigational gene therapy for the treatment of HCM caused by mutations in the MYBPC3 gene, the most common genetic cause of HCM. TN-201 is designed to deliver a fully functional MYBPC3 gene to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies of MYBPC3 knock-out models, TN-201 has been shown to halt disease progression and demonstrated significant and durable disease reversal and survival benefit after a single dose.
“Receipt of Fast Track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations,” said Whit Tingley, M.D., Ph.D., Chief Medical Officer of Tenaya. “As we prepare to begin dosing patients later this year, we look forward to continued close collaboration with the FDA under this designation in support of TN-201’s development.”
The FDA Fast Track program is designed to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical data demonstrates the potential to address unmet medical need. Companies with therapies that receive the Fast Track designation from the FDA are eligible for increased communication with the agency and may qualify for accelerated approval and priority review if relevant criteria are met. The goal of the program is to deliver approved treatments to patients with a serious or life-threatening condition as quickly as possible. This designation is not an assurance that regulatory approval will be received. TN-201 also has received Orphan Drug Designation from the FDA and Orphan Medicinal Product designation from the European Commission for the treatment of HCM due to mutations in the MYBPC3 gene.
In January 2023, Tenaya announced that the FDA cleared its Investigational New Drug application (IND) for TN-201. Tenaya is initiating the MyPeak-1 Phase 1b clinical trial to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-201. The multi-center, open-label study will enroll adults diagnosed with MYBPC3-associated nonobstructive HCM. Tenaya anticipates the first patient will be dosed in this clinical trial during the third quarter of 2023. Tenaya is also conducting two non-interventional studies to support the development of TN-201: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a natural history study of pediatric patients with MYBPC3-associated HCM.
About MYBPC3-Associated Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disorder and variants in the Myosin Binding Protein C3 (MYBPC3) gene are the most common genetic cause of HCM. MYBPC3-associated HCM is estimated to account for approximately 20 percent of the overall HCM population and to affect approximately 115,000 patients in the United States alone. MYBPC3-associated HCM is a chronic, progressive condition characterized by left ventricular thickening, hypercontractility, fibrosis, abnormal heart rhythms, cardiac dysfunction and impaired diastolic relaxation. This in turn leads to serious complications including debilitating symptoms such as shortness of breath, fainting and palpitations; heart failure; significant impairment in overall quality of life; and sudden cardiac death in some adults and children. There are currently no approved therapeutics addressing the underlying genetic cause of HCM. TN-201 is Tenaya’s first-in-class adeno-associated virus (AAV)-based gene therapy candidate being developed to treat hypertrophic cardiomyopathy (HCM) due to disease-causing variants in the MYBPC3 gene.
About Tenaya Therapeutics
Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging its integrated and interrelated Gene Therapy, Cellular Regeneration and Precision Medicine platforms and proprietary core capabilities, the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit .
Forward Looking Statements
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “potential,” “look forward,” “eligible,” “may,” “anticipates,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, statements regarding the therapeutic potential of TN-201 for MYBPC3-associated HCM patients; the timing for dosing of patients in the Phase 1b clinical trial evaluating TN-201; eligibility for increased communication with the FDA and potential qualification for accelerated approval and priority review for TN-201; and statements by Tenaya’s Chief Medical Officer. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the timing, scope and likelihood of regulatory filings and approvals; Tenaya’s ability to initiate and complete clinical trials for its product candidates; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; the level of costs associated with Tenaya’s research, development, manufacturing and other activities and the company’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya’s ability to successfully operate a manufacturing facility for clinical or commercial supply; Tenaya’s commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s reliance on third parties; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Contacts
Investors
Michelle Corral
Vice President, Investor Relations and Corporate Communications
Tenaya Therapeutics
IR@tenayathera.com
Media
Wendy Ryan
Ten Bridge Communications
wendy@tenbridgecommunications.com
快速通道临床1期孤儿药基因疗法
2023-05-03
Akosua Mireku
@AkosuaMireku1
Close-up photo of a stressed man who is suffering from a chest pain and touching his heart area. Hypertrophic cardiomyopathy is a condition affecting the heart. Credit: damircudic via Getty Images
The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM.
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In January 2023, Tenaya announced FDA clearance for its Investigational New Drug (IND) application for TN-201. With this recent news, San Francisco-based Tenaya is initiating a multi-centre, open-label Phase Ib clinical trial for the drug. The company predicts that the first patient will be dosed in the MyPeak-1 trial in the third quarter of 2023. Tenaya is also running two supplementary non-interventional studies for the drug. One study is looking at the seroprevalence of AAV9 antibodies among adults with MYBPC3-associated HCM, and the other, MyClimb, is a natural history study of pediatric patients with MYBPC3-associated HCM.
TN-201 is an intravenously delivered
adeno-associated virus (AAV)-
based
investigational gene therapy
that works by correcting mutations in the MYBPC3 gene, the most common cause of HCM. Mutations in this gene cause a decrease in the maximal force of cardiac contractions. Tenaya’s treatment delivers a functional MYPC3 gene, restoring myosin-binding protein levels to normal. TN-201 is one of four AAV-based gene therapies in Tenaya’s pipeline.
In April 2022,
Bristol Myers Squibb’s
Camzyos (mavacamten) became the
first FDA-approved cardiac myosin inhibitor
to treat obstructive HCM. Results from a Phase III trial where
Cytokinetics
is studying its investigational cardiac myosin inhibitor aficamten are expected in December.
HCM is the most common inherited cardiac disorder. In the US, MYBPC3-associated HCM accounts for approximately 20% of all HCM cases, affecting approximately 115,000 patients. According to the Mayo Clinic, HCM refers to “a disease in which the heart muscle becomes thickened (hypertrophied)”. Many people with HCM are asymptomatic. HCM can lead to other complications such as atrial fibrillation, blood clots, and strokes.
In a May 2 press release
, Tenaya’s CMO Dr. Whit Tingley said, “Receipt of fast track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations”.
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How is the Biopharmaceutical industry evolving?
2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape.
GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects.
Access the report to:
Benchmark the impact of major themes on the Biopharmaceutical industry.
Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents.
Evaluate the effects of COVID-19 on the sector.
Download the full report to understand what to expect and how to align your strategies for success.
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Country Code
UK (+44)
USA (+1)
Algeria (+213)
Andorra (+376)
Angola (+244)
Anguilla (+1264)
Antigua & Barbuda (+1268)
Argentina (+54)
Armenia (+374)
Aruba (+297)
Australia (+61)
Austria (+43)
Azerbaijan (+994)
Bahamas (+1242)
Bahrain (+973)
Bangladesh (+880)
Barbados (+1246)
Belarus (+375)
Belgium (+32)
Belize (+501)
Benin (+229)
Bermuda (+1441)
Bhutan (+975)
Bolivia (+591)
Bosnia Herzegovina (+387)
Botswana (+267)
Brazil (+55)
Brunei (+673)
Bulgaria (+359)
Burkina Faso (+226)
Burundi (+257)
Cambodia (+855)
Cameroon (+237)
Canada (+1)
Cape Verde Islands (+238)
Cayman Islands (+1345)
Central African Republic (+236)
Chile (+56)
China (+86)
Colombia (+57)
Comoros (+269)
Congo (+242)
Cook Islands (+682)
Costa Rica (+506)
Croatia (+385)
Cuba (+53)
Cyprus North (+90392)
Cyprus South (+357)
Czech Republic (+42)
Denmark (+45)
Djibouti (+253)
Dominica (+1809)
Dominican Republic (+1809)
Ecuador (+593)
Egypt (+20)
El Salvador (+503)
Equatorial Guinea (+240)
Eritrea (+291)
Estonia (+372)
Ethiopia (+251)
Falkland Islands (+500)
Faroe Islands (+298)
Fiji (+679)
Finland (+358)
France (+33)
French Guiana (+594)
French Polynesia (+689)
Gabon (+241)
Gambia (+220)
Georgia (+7880)
Germany (+49)
Ghana (+233)
Gibraltar (+350)
Greece (+30)
Greenland (+299)
Grenada (+1473)
Guadeloupe (+590)
Guam (+671)
Guatemala (+502)
Guinea (+224)
Guinea - Bissau (+245)
Guyana (+592)
Haiti (+509)
Honduras (+504)
Hong Kong (+852)
Hungary (+36)
Iceland (+354)
India (+91)
Indonesia (+62)
Iran (+98)
Iraq (+964)
Ireland (+353)
Israel (+972)
Italy (+39)
Jamaica (+1876)
Japan (+81)
Jordan (+962)
Kazakhstan (+7)
Kenya (+254)
Kiribati (+686)
Korea North (+850)
Korea South (+82)
Kuwait (+965)
Kyrgyzstan (+996)
Laos (+856)
Latvia (+371)
Lebanon (+961)
Lesotho (+266)
Liberia (+231)
Libya (+218)
Liechtenstein (+417)
Lithuania (+370)
Luxembourg (+352)
Macao (+853)
Macedonia (+389)
Madagascar (+261)
Malawi (+265)
Malaysia (+60)
Maldives (+960)
Mali (+223)
Malta (+356)
Marshall Islands (+692)
Martinique (+596)
Mauritania (+222)
Mayotte (+269)
Mexico (+52)
Micronesia (+691)
Moldova (+373)
Monaco (+377)
Mongolia (+976)
Montserrat (+1664)
Morocco (+212)
Mozambique (+258)
Myanmar (+95)
Namibia (+264)
Nauru (+674)
Nepal (+977)
Netherlands (+31)
New Caledonia (+687)
New Zealand (+64)
Nicaragua (+505)
Niger (+227)
Nigeria (+234)
Niue (+683)
Norfolk Islands (+672)
Northern Marianas (+670)
Norway (+47)
Oman (+968)
Palau (+680)
Panama (+507)
Papua New Guinea (+675)
Paraguay (+595)
Peru (+51)
Philippines (+63)
Poland (+48)
Portugal (+351)
Puerto Rico (+1787)
Qatar (+974)
Reunion (+262)
Romania (+40)
Russia (+7)
Rwanda (+250)
San Marino (+378)
Sao Tome & Principe (+239)
Saudi Arabia (+966)
Senegal (+221)
Serbia (+381)
Seychelles (+248)
Sierra Leone (+232)
Singapore (+65)
Slovak Republic (+421)
Slovenia (+386)
Solomon Islands (+677)
Somalia (+252)
South Africa (+27)
Spain (+34)
Sri Lanka (+94)
St. Helena (+290)
St. Kitts (+1869)
St. Lucia (+1758)
Sudan (+249)
Suriname (+597)
Swaziland (+268)
Sweden (+46)
Switzerland (+41)
Syria (+963)
Taiwan (+886)
Tajikstan (+7)
Thailand (+66)
Togo (+228)
Tonga (+676)
Trinidad & Tobago (+1868)
Tunisia (+216)
Turkey (+90)
Turkmenistan (+7)
Turkmenistan (+993)
Turks & Caicos Islands (+1649)
Tuvalu (+688)
Uganda (+256)
Ukraine (+380)
United Arab Emirates (+971)
Uruguay (+598)
Uzbekistan (+7)
Vanuatu (+678)
Vatican City (+379)
Venezuela (+58)
Vietnam (+84)
Virgin Islands - British (+1284)
Virgin Islands - US (+1340)
Wallis & Futuna (+681)
Yemen (North)(+969)
Yemen (South)(+967)
Zambia (+260)
Zimbabwe (+263)
Country
UK
USA
Afghanistan
Åland Islands
Albania
Algeria
American Samoa
Andorra
Angola
Anguilla
Antarctica
Antigua and Barbuda
Argentina
Armenia
Aruba
Australia
Austria
Azerbaijan
Bahamas
Bahrain
Bangladesh
Barbados
Belarus
Belgium
Belize
Benin
Bermuda
Bhutan
Bolivia
Bonaire, Sint Eustatius and Saba
Bosnia and Herzegovina
Botswana
Bouvet Island
Brazil
British Indian Ocean Territory
Brunei Darussalam
Bulgaria
Burkina Faso
Burundi
Cambodia
Cameroon
Canada
Cape Verde
Cayman Islands
Central African Republic
Chad
Chile
China
Christmas Island
Cocos Islands
Colombia
Comoros
Congo
Democratic Republic of
the Congo
Cook Islands
Costa Rica
Côte d"Ivoire
Croatia
Cuba
Curaçao
Cyprus
Czech Republic
Denmark
Djibouti
Dominica
Dominican Republic
Ecuador
Egypt
El Salvador
Equatorial Guinea
Eritrea
Estonia
Ethiopia
Falkland Islands
Faroe Islands
Fiji
Finland
France
French Guiana
French Polynesia
French Southern Territories
Gabon
Gambia
Georgia
Germany
Ghana
Gibraltar
Greece
Greenland
Grenada
Guadeloupe
Guam
Guatemala
Guernsey
Guinea
Guinea-Bissau
Guyana
Haiti
Heard Island and McDonald Islands
Holy See
Honduras
Hong Kong
Hungary
Iceland
India
Indonesia
Iran
Iraq
Ireland
Isle of Man
Israel
Italy
Jamaica
Japan
Jersey
Jordan
Kazakhstan
Kenya
Kiribati
North Korea
South Korea
Kuwait
Kyrgyzstan
Lao
Latvia
Lebanon
Lesotho
Liberia
Libyan Arab Jamahiriya
Liechtenstein
Lithuania
Luxembourg
Macao
Macedonia, The Former
Yugoslav Republic of
Madagascar
Malawi
Malaysia
Maldives
Mali
Malta
Marshall Islands
Martinique
Mauritania
Mauritius
Mayotte
Mexico
Micronesia
Moldova
Monaco
Mongolia
Montenegro
Montserrat
Morocco
Mozambique
Myanmar
Namibia
Nauru
Nepal
Netherlands
New Caledonia
New Zealand
Nicaragua
Niger
Nigeria
Niue
Norfolk Island
Northern Mariana Islands
Norway
Oman
Pakistan
Palau
Palestinian Territory
Panama
Papua New Guinea
Paraguay
Peru
Philippines
Pitcairn
Poland
Portugal
Puerto Rico
Qatar
Réunion
Romania
Russian Federation
Rwanda
Saint Helena, Ascension and Tristan da Cunha
Saint Kitts and Nevis
Saint Lucia
Saint Pierre and Miquelon
Saint Vincent and The Grenadines
Samoa
San Marino
Sao Tome and Principe
Saudi Arabia
Senegal
Serbia
Seychelles
Sierra Leone
Singapore
Slovakia
Slovenia
Solomon Islands
Somalia
South Africa
South Georgia and The South
Sandwich Islands
Spain
Sri Lanka
Sudan
Suriname
Svalbard and Jan Mayen
Swaziland
Sweden
Switzerland
Syrian Arab Republic
Taiwan
Tajikistan
Tanzania
Thailand
Timor-Leste
Togo
Tokelau
Tonga
Trinidad and Tobago
Tunisia
Turkey
Turkmenistan
Turks and Caicos Islands
Tuvalu
Uganda
Ukraine
United Arab Emirates
US Minor Outlying Islands
Uruguay
Uzbekistan
Vanuatu
Venezuela
Vietnam
British Virgin Islands
US Virgin Islands
Wallis and Futuna
Western Sahara
Yemen
Zambia
Zimbabwe
Kosovo
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临床3期快速通道基因疗法上市批准临床2期
2022-10-06
作为三大医疗终端之一，药店端发挥着越来越重要的作用。此前，因疫情等多方因素，药店发展沉寂一时，而今医保监管趋严、互联网医疗布局抢占市场等挑战也层出不穷。随着集中度、连锁率双提升，未来中国药店的“故事”该如何讲好呢？将如何发展？零售药店是不可或缺的医疗终端，近年来零售药店端的销售额在整体医疗终端中的占比不断提升。从一组数据中可见零售药店终端的增长动力与韧性。根据米内网数据显示，2021年三大药品销售终端的销售额为17747亿元，虽然公立医院以63.5%占据主要地位，但从近几年销售额占比的变化情况来看，零售药店和基层医疗机构的占比正在逐渐增大。尤其是在2020年受新冠疫情影响后，医院端销售额进一步减少，而药店端重要性得到凸显，2021年药店销售额占比提升至26.9%。三大药品终端市场销售额占比情况三大药品终端销售额增速比较资料来源：米内网、华安证券研究所对比三大药品终端的销售额增速，2020年疫情影响下三大终端增速都出现下滑的情况下，零售端仍旧保持正增长状态，而2021年零售端也仍以10.3%的增速领先医院和基层医疗终端。无论是从政策上还是数据上来看，零售药店都日渐成为新焦点。尤其是在近年来药品“零加成”、带量采购、双通道政策、处方流转平台等重磅政策的推行下，零售端的价值越来越被处方药厂家所重视。01“互补”还是“取代”？医药电商“来势汹汹”受2020年疫情高基数、短期财务等因素的影响，药店行业增速在2021年下半年持续下降。但短期的利空因素都在2021年消化后，2022年上半年收入、利润增速开始恢复，迎来拐点。具体来看，从2021年下半年开始，增速出现下滑，2021年12月零售药店市场销售额同比增长率为-3.0%，达到低点。相比之下，2022年增速出现明显拐点，2022年2月以来增速转正，6月份同比增长达到6.5%，整体而言2022上半年中国零售药店总销售额同比增长3.4%。行业逐渐恢复，驶向发展车道。不过，零售药店还面临一个不可忽视的“竞争对手”——医药电商。医药电商作为医药零售的新生力量发展迅猛，虽然起步较晚，但也正分食医药零售市场。米内网数据显示，按照终端平均零售价计算，2021年中国实体药店和网上药店（含药品和非药品）销售规模达7950亿元。其中，实体药店占比超70%，网上药店首次突破2000亿元，达到2234亿元，同比增长40.2%。不过，网上药店增速较快，但相较于整个医药零售市场，占比仍然相对较小。实际上，零售药店线下优势仍然不可替代。一方面，线下药店与O2O品类上相互补充。O2O品类具有“急懒夜专私”的特性，在感冒、胃肠道，咽喉、计生、ED品类方面占比明显大于零售门店总体品类占比。而在心脑血管疾病、高血压等指导性更强的疾病中，线下药店占比明显高于O2O。可以看出线上线下更多的互补的关系，而不是简简单单的取代关系。另一方面，O2O业务的开展依托于线下药店，覆盖受限于城市发展水平。全国医药O2O市场规模逐渐提升，O2O在零售药店占比在2021年3月升至2.8%。医药O2O渠道主要分为四大类，分别是：外卖平台（以美团/饿了么为主）、传统电商平台（如京东健康药急送）、健康服务平台（如平安好医生）以及连锁自建的O2O平台（如叮当快药）。但无论如何这些O2O业务的开展都是依托于线下药店。目前O2O集中在一二线城市，TOP10城市占比达到54.5％。主要原因是O2O需要强大的城市人力和运力基础，并且人口越密集效率越高，而目前一线城市才能满足这样的条件。整体而言，医药电商特点是增速虽快但占比仍小，并且用户的线上购药心智未完全成熟，所以目前线下仍是主要渠道。而且政策上看，互联网药品新规的落地带来监管趋严，线下药店的重要性进一步凸显。所以未来零售药店行业线下与线上自建、品种相互补充是发展的大方向。在经历了初创期、快速成长期、跨区域连锁发展期、集中度提高期、多元化发展期这5个阶段的快速发展后，中国药店数量从2006年的近32万家增长为2021年的58.7万家。其中，连锁药店数量也得到长足发展，从2006年的12.2万家增长至2021年的33.53万家，年复合增长率为7%，连锁率也从38%升至57.17%。零售药店连锁化是药品零售行业的大势所趋。不过相较于美日零售药店连锁化率基本达到60%以上的态势，中国药店连锁率仍有很大的提升空间，并且在集中度上，中国十强连锁的市占率仅为25.2%，远不及日本CR10市占率的70.7%和美国CR4的79.6%。华安证券认为，参考美日的连锁率水平，假设到2025年中国药店连锁率达到60%水平，期间每年以5%的增速增长，到2025年中国的连锁药店数将突破40万家，仍有不小增长空间。02“集中化、专业化、多元化”之路开启，头部药店充分受益华安证券认为，在未来，中国零售药店行业将朝着集中化、专业化及多元化的方向持续发展，头部药店充分受益。第一，集中度提升是大趋势，扩张是头部连锁药店的必选之路。全国空白市场仍旧空间巨大，对于头部连锁药店而言，无论是自建、并购还是加盟的方式，依靠资本和品牌加速拓展大有可为。以六大上市连锁药店为例，各药店门店总数都在稳步增长，门店布局上继续保持扩张态势。截至2022年上半年，益丰药房拥有9200家门店，大参林8896家，老百姓10009家，一心堂8990家，健之佳3283家，漱玉平民4009家。而实现药店集中化路径有三条，分别为自建、加盟、并购。一方面，各大连锁药店都在逐年推进自有门店的数量，截止2022年上半年，一心堂、老百姓、益丰药房、大参林、健之佳、漱玉平民直营门店数分别为8990、7254、7684、7520、3283、2970家，上半年净增门店数分别为430、1657、1391、703、239、668家。另一方面，在当前医药零售市场的大环境下，发展加盟也已经成为一个大的趋势。加盟作为一种有效的下沉方式可以使公司触及通过职业经理人难以达到的县域乡镇市场，快速拓展下沉市场。从披露的数据来看，益丰药房、大参林、老百姓和漱玉平民2022年上半年分别新增加盟店584、532、441和290家。此外，并购已经成为连锁药店规模发展的重要途径。老百姓、大参林、益丰药房等6家上市连锁药店整体并购力度逐年加大，自2019年至2021年，全年共计分别新发起33单、49单、85单并购，6家连锁药房带来的新增门店总数分别为1243家、1312家以及2191家，合计4746家。第二，中国零售药店行业将朝着专业化的发展路径持续生长。因药店是直面患者的医疗最前线机构之一，专业化能力将逐步成为发展的必要要素。而专业化体现在人员、物流、品种管理、医保资质等多个方面。一方面，专业人员在药店数量持续增长。我国注册执业药师数量增长迅速，从2014年的16.7万人增加到2022年7月的67.75万人，每万人口执业药师人数为4.8人，仍远低于世界药学联合会公布的每万人口累计拥有执业药师6.2人的标准。从执业单位来看，药品零售企业注册人数占绝对主力。截止2022年7月统计的注册于药品零售企业的执业药师62万人，占注册总数的91.2%。另一方面，我国药店的物流模式主要有三种：自建物流中心进行配送、依赖供应商配送和委托第三方配送。对于大型的并在扩张中的连锁药店而言，自建物流中心可以说是必经之路，自建物流问题，不简简单单是个物流整合的成本效率问题，更是配合扩张战略与扩张模式的关键一步。此外，面对繁多的药品、器械等品类，如何管理品种的比例和数量成为精细化管理的重要议题，这直接关系到药店的毛利率。毛利率的高低主要受高毛利商品销售占比的权重影响。即高毛利商品的销售占比越高，往往门店毛利率也越高。从中康数据显示的2021Q1零售药店品类结构分布来看，化学药和中成药销售合计占到74.3%，为主要营收来源，保健品、医疗器械以及中药饮片这些其他品类各占比5.5%、6.8%和13.3%。未来，医保资质对药店而言也非常重要。2021年职工医保个人账户在药店支出费用2060.92亿元，我国医保定点药店约占整体药店数量的7成。从CMH的“消费者对零售药店不满意的地方”调研显示，最令患者不满意的就是不能够医保支付。医保是患者选择购药渠道的重要考量因素。第三，药店还要向多元化发展，布局多场景，发展新增量。处方药外流虽是必然趋势，但真正实现处方的顺利流程却还存在一定难度，处方药从医院端走向药店终端也面临着各方面问题。所以在处方外流的大背景下，各种渠道都成为药店未来发展的布局点。但处方外流渠道有待梳理，多种医疗渠道形式下，处方外流可以流向药店、DTP药房、医药电商或者互联网医疗。因此处方外流的流向渠道争夺也必将成为药店的未来发展重点。随着激烈的竞争，大型连锁药房在向多元化发展的破局之路上，相比单一药房更有综合竞争力及渠道优势。目前，各大药房都在积极布局自己的DTP药店。益丰药房2021年在全国已建成DTP专业药房237家，其中已开通双通道医保门店130家。大参林2021年拥有DTP专业药房133家，进一步推动药品特别是DTP品种在门店的销售的同时，拓展业务板块获得新的增长点。老百姓2021年DTP药房数量为145家，销售占比约10%，药房专业属性高，承接处方外流能力强。此外，药店发展互联网+的未来趋势不可忽视。通过互联网扩大服务半径，提升综合竞争力。互联网带来的新零售概念与数字化模式促使线下药店进行改革升级，线下药店通过开展互联网医疗、B2C、O2O等新业务模式来扩大服务半径，为公司提供新兴成长动力。六大上市连锁药店也都在积极布局自身的线上业务，提升线上渠道市场占有率。2022上半年，一心堂、老百姓、益丰、健之佳、漱玉平民线上总营收分别为2.62亿元、5.6亿元、8.16亿元、6.25亿元、3.6亿元，5家增速超过60%。六大民营上市连锁2022半年线上渠道经营对比资料来源：第一药店财智、公司公告、华安证券研究所目前，以实体药店保障社区健康服务，以O2O模式提升门店服务半径与患者黏性，以新零售电商模式开拓空白市场赢取增量空间，已经成为行业的一大发展趋势。本文编自华安证券《不畏浮云遮望眼，长中短期逻辑看好药店行业持续发展——零售药店行业深度报告》。登记邮箱信息订阅E药经理人信息服务扫描二维码精彩推荐集采 | 国谈 | 医保动态 | 药审 | 人才 | 薪资 | 榜单 | CAR-T | PD-1 | mRNA | 单抗 | 商业化 | 国际化启思会 | 声音·责任 | 创百汇 | E药经理人理事会 | 微解药直播 | 大国新药 | 营销硬观点 | 投资人去哪儿 | 分析师看赛道 | 药事每周谈 | 医药界·E药经理人 | 中国医药手册创新100强榜单 | 恒瑞 | 中生 | 百济 | 石药 | 信达 | 君实 | 翰森 | 和黄医药 | 齐鲁 | 上海医药 | 复宏汉霖 | 康方生物 | 贝达 | 荣昌 | 科兴生物 | 泽璟制药 | 复星医药 | 海思科 | 再鼎 | 基石跨国药企50强榜单 | 辉瑞 | 艾伯维 | 诺华 | 强生 | 罗氏 | BMS | 默克 | 赛诺菲 | AZ | GSK | 武田 | 吉利德科学 | 礼来 | 安进 | 诺和诺德 | 拜耳 | 莫德纳 | BI | 晖致 | 再生元
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