PTC Therapeutics Announces that The Committee for Medicinal Products for Human Use (CHMP) Recommendation of Translarna ™ (ataluren) Label Update for Non-Ambulatory Patients with Duchenne Muscular Dystrophy
Original press release > SOUTH PLAINFIELD, N.J., June 29, 2020,/PRNewswire/ — PTC Therapeutics, Inc.
PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended by a majority of votes to remove the statement “efficacy has not been demonstrated in non-ambulatory patients” from the Summary of product characteristics (SmPC) for Translarna™ (ataluren). This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation. The change also should support reimbursement agencies granting continued access to Translarna for patients who become non-ambulatory during their treatment. The CHMP’s positive opinion is subject to final approval by the European Commission, which is typically granted in a two-month time frame.
Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics – “We are excited to see that the CHMP adopted the positive opinion for this label modification allowing patients who become non-ambulatory to continue to use Translarna. All nonsense mutation Duchenne patients should be able to benefit from continued Translarna use, ensuring they have the best chance of preserving muscle function for as long as possible.”
Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation and works by restoring dystrophin production. The EMA approves it for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged two years and older. Real-world evidence and clinical trials have demonstrated the potential of Translarna to slow disease progression and improve outcomes before and beyond the loss of ambulation:
- In a long-term, open-label extension study (Study 019), Translarna preserved lung function in non-ambulatory patients for an additional four years compared with patients treated with standard of care from a long-term natural history study (the Cooperative International Neuromuscular Research Group (CINRG) natural history database of Duchenne patients).
- Data from the STRIDE Registry, the first international drug registry for Duchenne patients receiving Translarna, demonstrated that boys treated with Translarna and standard of care (SoC) preserved the ability to walk for years longer than those on SoC alone, as well as experienced a slower decline in lung function.
- Children treated with Translarna in a real-world setting as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort in the CINRG natural history study, with a median age at loss of ambulation of 14.5 years and 11 years, respectively (72% relative risk reduction).
- There was a trend toward a delay in the age at the decline in pulmonary function in STRIDE patients compared with CINRG patients, as measured by predicted FVC < 50% and FVC < 1 L.4
- These data suggest that treatment with ataluren, in addition to SoC, may delay loss of ambulation, as well as pulmonary functional decline, in patients with nmDMD.
- View the original content here.
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 two years and older. Ataluren is an investigational new drug in the United States.
- Learn more about nonsense mutation here.
- La Force DMD blog / experimental therapies
Ataluren in Canada
At this moment, PTC Therapeutics has not applied for marketing approval with Health Canada.
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 their mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit them at www.ptcbio.com. Read more here > La Force DMD Blog / PTC Therapeutics.
