Co and NHS England negotiate Translarna managed access agreement for patients with absurd muscular dystrophy mutation Duchenne SOUTH PLAINFIELD, N.J., 15 April 2016 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT), announced today that the National Institute for Health and Care Excellence (NICE) has recommended translarna ∇ (Ataluren) to outpatients aged five years and older with the derbnonsense duchenne muscular dystrophy (nmDMD) mutation associated with an Access to Management Agreement (MAA) with England. The provision of patient access depends on the completion of the draft NICE guidelines that the Agency expects in May 2016. July 7: The Fight Continues: Duchenne action during the parliamentary session to discuss existing barriers to access to drugs A: A Managed Access Agreement is an agreement developed between NHS England, NICE and a drug manufacturer that allows patients to obtain new treatments while collecting long-term data and before NICE makes a final decision on whether or not a drug should be recommended for NHSE funding. For Translarna, the MAA will last 5 years, while the data will be collected, and it will be reviewed in 2021. Translarna was approved by the European Commission in August 2014 for the treatment of NmDMD and is currently available to patients in 23 countries, either through extensive access programmes or through commercial sales. “We are very pleased with NICE`s recommendation, which Translarna recognizes as an innovative drug that has the potential to change the course of this devastating disease,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “This is a milestone for boys and young men in England with a senseless DMD mutation.
We are grateful to the patients, families, interest associations and physicians who supported PTC Therapeutics through this important access process, and look forward to working with NHS England to conclude the Access to Management 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: 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; expectations for regulatory approvals, including the ability of TPCs to submit or advance regulatory submissions in a timely (or generally- timely) manner, the period during which the results of the