Translarna Managed Access Agreement

The actual results, performance or performance of the TPC may differ materially from the results expressed or implied in its forward-looking statements due to a large number of risks and uncertainties, including those relating to: whether NICE`s final guidelines recommend translarna for the treatment of nmDMD; NHS England`s effective repayment decisions and the acceptability of the MAA`s final terms; TPC`s ability to maintain the marketing authorization for translarna for the marketing of nmDMD in the European Economic Area (EEA), which is subject to current conditions and is also reviewed and renewed annually by the EMA following its reassessment of the risk-benefit ratio of the authorisation; TPC`s ability to commercialize and market Translarna in general, particularly as a treatment for nmDMD, including its ability to successfully negotiate favourable pricing and reimbursement procedures in countries where it can obtain administrative approval; the timing and outcome of future interactions between the TPC and the FDA with respect to translarna for the treatment of NmDMD, including whether the TPC should conduct additional clinical and non-clinical trials at a significant cost and whether these studies can, if successful, allow the FDA trial of an NDA submission; if the FDA, EMA or other regulatory authorities agree to interpret the results of ACT DMD and other data on the safety and efficacy of Translarna for the treatment of NmDMD or other clinical trials of TPC; the outcome of price and refund negotiations in areas where TPC has the right to sell Translarna; whether patients and health professionals can access Translarna in an alternative way when price and reimbursement negotiations do not have positive results in their territory, including whether Translarna is accessible through a reimbursement route provided for by German law and whether these problems of access to German patients are minimized while maintaining a sustainable price; regulatory approval expectations, including the ability of TPCs to transmit or advance regulatory submissions in a timely (or general manner), the period during which regulatory review results become available, negative decisions made by regulators, other delays or delays in the regulatory process, and TPC`s ability to meet existing or future regulatory standards for Translarna; The scope of Translarna`s administrative authorizations or authorizations (if any), including marking and other issues that may affect Translarna`s commercial availability or potential; the ability of TPCs to meet additional obligations, including additional studies or studies on cost-effectiveness, licensing or compliance with professional and business practice requirements in areas where they can obtain authorisation from the authorities; The introduction, implementation and availability of data from clinical trials and studies; The TPC`s scientific approach and the overall progress of development; The base of eligible patients and the commercial potential of other translarna and TPC candidates; and the factors examined in the “Risk Factors” section of the TPC`s latest annual report on Form 10-K, as well as any updates to these risk factors, which are introduced from time to time in other TPC applications to the SEC. They are encouraged to carefully consider all of these factors. As with any pharmaceutical drug under development, there are significant risks to the development, approval and marketing of new products. There is no guarantee that Translarna will obtain full administrative approval in each jurisdiction or maintain the current marketing authorization in the EEA or that it will generally be commercially successful, particularly with regard to the treatment of nmDMD. A: An Access to Management Agreement is an agreement between NHS England, NICE and a drug manufacturer that allows patients to obtain new treatments while collecting long-term data