BUSINESS OVERVIEW 6 The deficiencies identified may be minor, for example, requiring has such designation, the product is entitled to orphan product labeling changes, or major, for example, requiring additional exclusivity, which means that the FDA may not approve any other clinical trials. Additional ly, the complete response letter may applications to market the same drug or biological product for the include recommended actions that the applicant might take to same indication for seven years, except in limited circumstances, place the application in a condition for approval. If a complete such as a showing of clinical superiority to the product with response letter is issued, the applicant may either resubmit the orphan exclusivity. Competitors, however, may receive approval BLA, addressing all of the deficiencies identified in the letter, or of different products for the indication for which the orphan withdraw the application. product has exclusivity or obtain approval for the same product but for a different indication for which the orphan product has If a product receives regulatory approval, the approval may exclusivity. Orphan product exclusivity also could block the be significantly limited to specific diseases and dosages or the approval of one of our products for seven years if a competitor indications for use may otherwise be limited, which could restrict obtains approval of the same biological product as defined by the the commercial value of the product. Further, the FDA may FDA or if our product candidate is determined to be contained require that certain contraindications, warnings or precautions be within the competitor’s product for the same indication. If included in the product labeling. The FDA may impose restrictions a drug or biological product designated as an orphan product and conditions on product distribution, prescribing, or dispensing receives marketing approval for an indication broader than what in the form of a risk management plan, or otherwise limit the is designated, it may not be entitled to orphan product exclusivity. scope of any approval. In addition, the FDA may require post- Orphan drug status in the European Union has similar, but not marketing clinical trials, sometimes referred to as Phase IV clinical identical, benefits. trials, designed to further assess a biological product’s safety and effectiveness, and testing and surveillance programs to monitor Expedited Development and Review Programs the safety of approved products that have been commercialized. The FDA is authorized to expedite the review of BLAs in several One of the performance goals agreed to by the FDA under the ways. For example, the Fast Track program is intended to expedite PDUFA is to review 90% of standard BLAs in 12 months of receipt or facilitate the process for reviewing new drugs and biological and 90% of priority BLAs in eight months of receipt, whereupon a products that meet certain criteria. Specifically, new drugs and review decision is to be made. The FDA does not always meet its biological products are eligible for Fast Track designation if they PDUFA goal dates for standard and priority BLAs, and its review are intended to treat a serious or life-threatening condition and goals are subject to change from time to time. The review process demonstrate the potential to address unmet medical needs for and the PDUFA goal date may be extended by three months the condition. Fast Track designation applies to the product and if the FDA requests or the BLA sponsor otherwise provides the specific indication for which it is being studied. The sponsor of additional information or clarification regarding information a new drug or biologic may request the FDA to designate the drug already provided in the submission within the last three months or biologic as a Fast Track product at any time during the clinical before the PDUFA goal date. development of the product. Unique to a Fast Track product, the FDA may consider for review sections of the marketing application Orphan Drug Designation on a rolling basis before the complete application is submitted, Under the Orphan Drug Act, the FDA may grant orphan if the sponsor provides a schedule for the submission of the designation to a drug or biological product intended to treat a rare sections of the application, the FDA agrees to accept sections of disease or condition, which is generally a disease or condition that the application and determines that the schedule is acceptable, affects fewer than 200,000 individuals in the United States, or and the sponsor pays any required user fees upon submission of more than 200,000 individuals in the United States and for which the first section of the application. there is no reasonable expectation that the cost of developing and Any product submitted to the FDA for marketing, including making a drug or biological product available in the United States under a Fast Track program, may be eligible for other types of for this type of disease or condition will be recovered from sales FDA programs intended to expedite development and review, of the product. Orphan product designation must be requested such as breakthrough therapy designation, priority review and before submitting a BLA. After the FDA grants orphan product accelerated approval. designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the FDA. Orphan product Priority review. Any product is eligible for priority review if it designation does not shorten the duration of the regulatory has the potential to provide safe and effective therapy where review and approval process. no satisfactory alternative therapy exists or a significant improvement in the treatment, diagnosis or prevention of a If a product that has orphan designation subsequently receives disease compared to marketed products. The FDA will attempt to the first FDA approval for the disease or condition for which it direct additional resources to the evaluation of an application for 92 – GENSIGHT BIOLOGICS – 2017 Registration Document