FDA Duchenne Muscular Dystrophy and Related Dystrophinopathies Developing Drugs for Treatment
标准简介
Duchenne Muscular Dystrophy and Related Dystrophinopathies Developing Drugs for Treatment[附网盘链接]是Food And Drug Administration发布的FDA标准，适用于美国。标准截图
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标准文档类型为Duchenne Muscular Dystrophy and Related Dystrophinopathies Developing Drugs for Treatment[附网盘链接]高清PDF版本（文字版），标准文档内可进行搜索，可以复制原文，可粘贴。标准部分原文
Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment 1
Guidance for Industry This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA office responsible for this guidance as listed on the title page. I. INTRODUCTION This guidance addresses FDA’s current thinking regarding clinical development programs and trial designs for drugs to support an indication for the treatment of one or more dystrophinopathies: Duchenne muscular dystrophy (DMD) and related dystrophinopathies including Becker muscular dystrophy (BMD), DMD-associated dilated cardiomyopathy (DCM), 2, 3
and symptomatic carrier states in females. The most prominent pathology in dystrophinopathies is degeneration of skeletal and cardiac muscle leading to progressive loss of muscle function, respiratory and cardiac failure, and premature death. This guidance does not address the development of drugs to treat secondary complications of muscle degeneration in dystrophinopathies (e.g., drugs specifically for heart failure or pulmonary infections). This guidance does not contain discussion of the general issues of statistical analysis or clinical trial design, as these topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical 4
Trials, respectively. 1
This guidance has been prepared by the Division of Neurology Products in the Center for Drug Evaluation and Research in cooperation with the Center for Biologics Evaluation and Research (CBER), at the Food and Drug Administration. 2
For the purposes of this guidance, all references to drugs include both human drugs and therapeutic biological products unless otherwise specified. 3
In addition to consulting guidances, sponsors are encouraged to contact the division to discuss specific issues that arise during the development of drugs for the treatment of dystrophinopathies. 4
We update guidances periodically. To make sure you have the most recent version of a guidance, check the FDA Drugs or Biologics guidance web pages at https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm.
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