BUSINESS OVERVIEW 6 If granted, data exclusivity prevents regulatory authorities in • the holder of the marketing authorization for the original orphan the European Union from referencing the innovator’s data to medicinal product is unable to supply sufficient quantities of assess a generic application. During the additional two-year the medicinal product, or period of market exclusivity, a generic marketing authorization • the second applicant can establish in the application that the can be submitted, and the innovator’s data may be referenced, second medicinal product, although similar to the orphan but no generic product can be marketed until the expiration of the medicinal product already authorized, is safer, more effective market exclusivity. or otherwise clinically superior. However, there is no guarantee that a product will be considered Pursuant to Regulation (EC) No. 847/2000 of April 27, 2000 by the European Union’s regulatory authorities to be a new laying down the provisions for implementation of the criteria for chemical entity, and products may not qualify for data exclusivity. designation of a medicinal product as an orphan medicinal product Orphan Medicinal Products and definitions of the concepts “similar medicinal product” and “clinical superiority,” a sponsor applying for designation of a The European Union provides opportunities for market exclusivity. medicinal product as an orphan medicinal product shal l apply Pursuant to abovementioned Regulation (EC) No. 141/2000, for designation at any stage of the development of the medicinal products receiving orphan designation in the European Union product. can obtain market exclusivity for a certain number of years in the European Union following the marketing approval. The abovementioned Regulation (EC) No. 141/2000 provides for other incentives regarding orphan medicinal products. It Where a marketing authorization in respect of an orphan notably provides for a protocol assistance. The sponsor of an medicinal product is granted pursuant to Regulation (EC) orphan medicinal product may indeed, prior to the submission of No. 726/2004 or where al l the Member States have granted an application for marketing authorization, request advice from marketing authorizations for this product, in accordance with EMA on the conduct of the various tests and trials necessary the procedures for mutual recognition, the Community and the to demonstrate the quality, safety and efficacy of the medicinal Member States shall not, for a period of 10 years, accept another product. Besides, EMA shal l draw up a procedure on the application for a marketing authorization, or grant a marketing development of orphan medicinal products, covering regulatory authorization or accept an application to extend an existing assistance for the definition of the content of the application for marketing authorization, for the same therapeutic indication, in authorization. respect of a similar medicinal product. Regulation (EC) No. 141/2000 also provides that medicinal This period may however be reduced to six years if, at the end of products designated as orphan medicinal products under the the fifth year, it is established, in respect of the medicinal product provisions of this Regulation shall be eligible for incentives made concerned, that the above-mentioned criteria no longer met, inter available by the Community and by the Member States to support alia, where it is shown on the basis of available evidence that the research into, and the development and availability of, orphan product is sufficiently profitable not to justify maintenance of medicinal products and in particular aid for research for small- and market exclusivity. medium-sized undertakings provided for in framework programs Pursuant to Regulation No. 1901/2006, for orphan medicinal for research and technological development. products, instead of an extension of the supplementary protection Protection of Personal Data certificate, the 10 year period of orphan market exclusivity should be extended to 12 years if the requirement for data on The processing of personal data col lected during clinical trials use in the pediatric population is fully met (i.e. when the request should comply with the simplified standards adopted in this contains the results of all studies carried out under the approved respect by the Commission Nationale de l’Informatique et des Pediatric Investigation Plan (“PIP”) Plan and when the declaration Libertés, or CNIL (i.e. the “Reference Methodologies”), and attesting the conformity of the request to this PIP is included in the sponsor of the trial shal l file with the CNIL a compliance the marketing authorization). undertaking with these Reference Methodologies, as applicable, through a simplified notification procedure. Patients then always However, by way of derogation, a marketing authorization may be shall have a right to access and correct their personal data, and to granted, for the same therapeutic indication, to a similar medicinal object to their processing/withdraw their consent, pursuant to product if: Act No. 78-17 of January 6, 1978, as amended. • the holder of the marketing authorization for the original As from the entry into force of the General Data Protection orphan medicinal product has given his consent to the second Regulation (GDPR) on May 25, 2018, the current regime should applicant, or remain pretty much the same, as the draft bil l “CNIL 2” stil l GENSIGHT BIOLOGICS – 2017 Registration Document– 87