HILA approves access to Translarna™ the first treatment approved for the underlying cause of childhood muscle wasting disease
- Decision by the Finnish Pharmaceuticals Pricing Board (HILA) means access for ambulatory patients with nonsense mutation Duchenne muscular dystrophy from two years of age and older -
Gothenburg, Sweden [October 18, 2021]– PTC Therapeutics announced the decision of the Finnish Pharmaceuticals Pricing Board [Lääkkeiden hintalautakunta] to allow access to Translarna™ (ataluren) when prescribed for the treatment of nonsense mutation DMD patients (nmDMD) to ambulatory patients aged two years and older.1 The decision will take effect from 1st December 2021.
“This is a momentous occasion for the families of children in Finland and everyone at PTC,” said Joakim Andersson, PTC Therapeutics’ Regional Head Nordics, Baltics and BeNeLux. “Over many years we have been tireless in our efforts to collaborate with HILA to enable patient access to Translarna treatment and we are thankful to the unwavering support of the entire Duchenne community.”
Duchenne muscular dystrophy is a severe progressive disease linked to the X-chromosome, occurring in approximately 1 in every 3,500–5,000 live male births.2,3,4 The disease is present from birth and symptoms usually appear in the first few years of life. It leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe.5
Translarna is indicated in Finland for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged two years and older.6 Real world evidence and clinical trials have demonstrated its potential to slow disease progression and improve patient outcomes.7,8,9
“This decision is long-awaited and very significant for families whose children may now benefit from a treatment that can slow down disease progression”, said Ritva Pirttimaa from the Finnish Neuromuscular Disorders Association. “Patient advocacy work has been done since 2015 to help secure access to Translarna. We are grateful for work done by parents, clinicians and the wider DMD community in Finland“, she said.
PTC Therapeutics is committed to provide Translarna to all patients in need of therapy. This decision means that there are now just a very few countries in the EU where patients do not have access to Translarna through some form of national reimbursement system.
For more information, please contact info.nordic@ptcbio.com.
About Translarna™ (ataluren)
Translarna, 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, tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties 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 DMD can lose the ability to walk as early as age ten, followed by loss of the use of their arms.
DMD patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information on the signs and symptoms of DMD can be found at: www.lihastautiliitto.fi
About PTC Therapeutics
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. For more information on PTC, please visit www.ptcbio-en.se.
For More Information: info.nordic@ptcbio.com
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1 https://www.hila.fi/luettelot/kokouspaatokset/
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 Parent Project Muscular Dystrophy. Available at https://www.parentprojectmd.org/about- ppmd/history/. Last Accesses October 2021
4 Ellis JA et al. 195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14-16th December, 2012, Naarden, The Netherlands. Neuromuscul Disord. 2013;23:682–9. 5 Duchenne Muscular Dystrophy. Muscular Dystrophy Association. Available at: https://www.mda.org/disease/duchenne-muscular-dystrophy. Last Accessed October 2021.
6 Summary of Product Characteristics. Translarna. European Medicines Agency. Accessed October 2021. https://www.ema.europa.eu/en/documents/product-information/translarna-epar-product- information_en.pdf
7 Finkel RS, et al. Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy. PLoS One. 2013;8(12):e81302.
8 McDonald CM, et al. Long-term pulmonary function outcomes in non-ambulatory patients with nonsense muatation Duchenne muscular dystrophy treated with ataluren: 240-week data from an open- label extension study. Vienna, Austria: s.n., July 6-10 2018. Poster presented at the International Congress on Neuromuscular Diseases.
9 Mercuri E, et al. Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. J Comp Eff Res. 2020;9(5):341-360.