BUSINESS OVERVIEW 6 • Decree No. 2016-1538 of November 16, 2016 on the unique studies. After submission of the complete file, the ANSM may agreement for the implementation of commercial clinical trials inform the sponsor that it objects to the implementation of the involving human beings in health care institutions; research. The sponsor can then modify the contents of its research • Decree No. 2016-1537 of November 16, 2016 on research project and submit an amended or supplemented request to the involving human beings; ANSM; this procedure may not, however, be applied more than • Order No. 2016-800 of June 16, 2016 on research involving once. If the sponsor does not alter the content of its request, the human beings; request is considered rejected. • Act No. 2016-41 of January 26, 2016 for the modernization of Under French law, the time limit for the examination of a request our health system; for authorization of a clinical trial cannot exceed 60 days from the date of receipt of the complete file by ANSM (Article R.1123-38 • Decision of December 29, 2015 establishing the rules for good PHC) and 45 days for the CPP (Article R.1123-23 PHC). clinical practice; • Act No. 2012-300 of March 5, 2012 (or “Loi Jardé”) related to In the event of a risk to public health or if the ANSM considers biomedical research involving human beings; that the conditions in which the research is implemented no longer correspond to the conditions indicated in the request • Act No. 2011-2012 of December 29, 2011 aiming to strengthen for authorization or does not comply with the provisions of the health safety of medicinal and health products; PHC, the ANSM may at any time request changes to procedures • Decree No. 2007-454 of March 25, 2007 on agreements for the realization of research, and suspend or ban this research and relationships between companies and members of some (Article L.1123-11 PHC). healthcare professions, amending the PHC; Under Article L.1123-7 PHC, the CPP shall deliver its opinion • Decision of December 11, 2006 establishing the rules of good on the conditions of validity of the research, particularly with manufacturing practice; respect to the protection of participants, their information and • Decision of November 24, 2006 establishing the rules for good how they collect informed consent, as well as the project’s general clinical practice for research involving human subjects; relevance, the satisfactory nature of the assessment of benefits • Decree No. 2006-477 of April 26, 2006 amending Chapter I and risks and the adequacy between the objectives pursued and of Title I I of Book I of the first part of the PHC on biomedical the means implemented. research; The decision of November 24, 2006 sets forth the rules for good • Decision of January 5, 2006 concerning the approval of a clinical practice (“GCP”), for biomedical research on medicines for methodology for the reference to the processing of personal human use provided for in Article L.1121-3 PHC. The purpose of data carried out within the context of biomedical research; the rules for GCP is to ensure both the reliability of data arising • Act No. 2004-806 of August 9, 2004 on public health policy; from clinical trials and the protection of persons participating in • Act No. 2004-801 of August 6, 2004 on data protection (and these clinical trials. GCPs shall apply to all clinical trials, including its implementing decrees); pharmacokinetics, bioavailability and bioequivalence studies in healthy volunteers and Phase I I to IV clinical trials. • Act No. 2002-3003 of March 4, 2002 on rights of patients and on the quality of the healthcare system and its implementing Under French law, a specific authorization issued by the Director decrees; and General of ANSM is required before commencing clinical trials involving some advanced therapy medicinal products, including • Act No. 78-17 of January 6, 1978 on information, technology in particular medicinal products for gene therapy and somatic cell and civil liberties, as notably amended by Act No. 2004-801 of therapy including xenogenic (see Article L.4211-9-1 PHC). August 6, 2004 on data protection and its implementing decrees. Market Exclusivity in the European Union Under French law, a favorable opinion of a competent research To obtain regulatory approval of an investigational biological and ethics committee (Comité de Protection des Personnes or “CPP”) product under European Union regulatory systems, we must of the jurisdiction in which the investigator exercises its activity is submit an MA. The application used to file the BLA in the United always required for the conduct of clinical trials. Besides, a prior States is similar to that required in the European Union, with the authorization issued by the ANSM is required for some types of exception of, among other things, country-specific document clinical trials (interventional research with risk above minimal risk). requirements. The European Union also provides opportunities In practice, the applicant must submit to the ANSM a request for market exclusivity. For example, in the European Union, for authorization of a clinical trial along with a file, which shall, in upon receiving marketing authorization, new chemical entities particular, contain information on the clinical protocol and specific generally receive eight years of data exclusivity and an additional product data and its quality control, as well as results of preclinical two years of market exclusivity. 86 – GENSIGHT BIOLOGICS – 2017 Registration Document