PTC Announces Expansion of Translarna™ Label to Include Ambulatory Patients as Young as 2 Years Old in Brazil
PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced that Brazil's ANVISA has approved the expansion of Translarna™ (ataluren) to include children aged 2 to 5 with nonsense mutation Duchenne muscular dystrophy (Duchenne). This broadens the prior indication of 5 years and older, allowing earlier treatment that may preserve muscle function and slow disease progression. Translarna is the sole treatment targeting the underlying cause of Duchenne due to a nonsense mutation and is licensed in the EEA for ambulatory patients aged 2 and older.
- ANVISA approval for Translarna expansion to ages 2-5 increases potential patient base.
- Early treatment may significantly improve outcomes for young patients with Duchenne.
- Translarna remains an investigational drug in the U.S., limiting market potential.
SOUTH PLAINFIELD, N.J., Oct. 25, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian National Health Surveillance Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved the expansion of the indication of Translarna™ (ataluren) to include ambulatory children aged 2 to 5 years old with nonsense mutation Duchenne muscular dystrophy (Duchenne).1 This broadens the use beyond the previous age range of 5 years and older.
"Expanding access to Translarna to children with nonsense mutation Duchenne in Brazil is important to us," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "We know that with Duchenne, early diagnosis and treatment gives children the best chance of preserving muscle function and delaying the progression of the disease."
Duchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe.2,3 Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation. Clinical trials and real-world evidence have demonstrated Translarna's potential to slow disease progression and improve patient outcomes.1-3
About Translarna (ataluren)
Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, the tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States.
About Duchenne Muscular Dystrophy (Duchenne)
Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s.
More information regarding Duchenne is available through the Muscular Dystrophy Association and the Parent Project Muscular Dystrophy. Additionally, information and resources are available at www.duchenneandyou.com.
About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The Company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Twitter at @PTCBio, Instagram and LinkedIn.
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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 contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the commercialization of its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.
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: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area (EEA), including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC's ability to enroll, fund, complete and timely submit to the EMA the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, which is a specific obligation to continued marketing authorization in the EEA; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; 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 SEC. You are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna.
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 press release except as required by law.
2 Birnkrant DJ et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018; 17:251–67. | ||
3 Duchenne Muscular Dystrophy. Muscular Dystrophy Association. Available at: https://www.mda.org/disease/duchenne-muscular-dystrophy. Last Accessed October 2021. | ||
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SOURCE PTC Therapeutics, Inc.
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