"This is an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy," said
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with DMD lose the ability to walk from as early as 10 years of age and experience life-threatening lung and heart complications in their late teens and early twenties.
"We are extremely pleased by the NICE recommendation, which recognizes Translarna as an innovative medicine with the potential to change the course of this devastating disease," said
PTC and NHS England are in the process of finalizing an MAA outlining financial and clinical details surrounding the use of Translarna, including a confidential financial arrangement. The MAA is expected to allow PTC to collect further data on the efficacy of Translarna for the treatment of nmDMD over a five-year period with NICE guidance to be reviewed again at the end of that period.
Translarna was approved by the
About Translarna™ (ataluren)
Translarna, discovered and developed by
Further information about Translarna, including the European Public Assessment Report, Summary of Product Characteristics and Patient Information Leaflet, is available on the
This medicinal product is subject to additional monitoring. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system or to PTC at firstname.lastname@example.org.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA is copied from DNA through the transcription process. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information on the company, please visit our website www.ptcbio.com.
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Forward Looking Statements:
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements, other than those of historical fact, contained in this release are forward-looking statements, including statements about our expectations regarding the potential timing and outcome of final guidance from NICE with respect to Translarna for the treatment of nmDMD; the ability of NHS England and
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: whether final guidance from NICE recommends Translarna for the treatment of nmDMD; actual reimbursement decisions by NHS England and the acceptability of final terms of the MAA; PTC's ability to maintain the marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area, or EEA, which is subject to ongoing conditions and is also subject to annual review and renewal by the EMA following its reassessment of the risk benefit balance of the authorization; PTC's ability to commercialize and commercially manufacture Translarna in general and specifically as a treatment for nmDMD, including its ability to successfully negotiate favorable pricing and reimbursement processes on a timely basis in the countries in which it may obtain regulatory approval; the timing and outcome of future interactions PTC has with the FDA with respect to Translarna for the treatment of nmDMD, including whether PTC is required to perform additional clinical and non-clinical trials at significant cost and whether such trials, if successful, may enable FDA review of a NDA submission; whether the FDA, the EMA or other regulators agree with PTC's interpretation of the results of ACT DMD and other data with respect to the safety and efficacy of Translarna for the treatment of nmDMD or PTC's other clinical trials; the outcome of pricing and reimbursement negotiations in those territories in which PTC is authorized to sell Translarna; whether patients and healthcare professionals may be able to access Translarna through alternative means if pricing and reimbursement negotiations in the applicable territory do not have a positive outcome, including whether Translarna may be accessed through a reimbursed importation pathway provided under German law and whether such pathway will minimize any access issues for German patients while maintaining a sustainable price; expectations for regulatory approvals, including PTC's ability to make or advance regulatory submissions in a timely manner (or at all), the period during which the outcome of regulatory reviews will become available, adverse decisions by regulatory authorities, other delay or deceleration of the regulatory process, and PTC's ability to meet existing or future regulatory standards with respect to Translarna; the scope of regulatory approvals or authorizations for Translarna (if any), including labeling and other matters that could affect the availability or commercial potential of Translarna; PTC's ability to fulfill any additional obligations, including with respect to further trials or studies relating to cost-effectiveness, obtaining licenses or satisfying requirements for labor and business practices, in the territories in which it may obtain regulatory approval; the initiation, conduct and availability of data from clinical trials and studies; PTC's scientific approach and general development progress; the eligible patient base and commercial potential of Translarna and PTC's other product candidates; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K as well as any updates to these risk factors filed from time to time in PTC's other filings with the
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this release except as required by law.
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