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Duchenne muscular dystrophy is characterized by progressive muscle degeneration. In DMD patients, the pulmonary function becomes progressively impaired as the dystrophic process affects respiratory muscles, including the diaphragm. Strategies to arrest this severe gradual deterioration are needed to extend lives and improve quality of life.
Results of three clinical trials using eteplirsen show promising results
Sarepta, along with Harvard Medical School, The Children’s Hospital of Philadelphia, Nationwide Children’s Hospital, the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center and Ohio State University published results from three trials in the Journal of Neuromuscular Diseases.
This study conducted in three clinical trials supports that Sarepta Therapeutics’ Exondys 51 (eteplirsen) slows respiratory decline in Duchenne muscular dystrophy (DMD).
The respiratory decline in patients treated with eteplirsen was significantly lower, and this was true across all stages of the disease evaluated.
As the disease progresses, patients require increasing levels of clinical treatment. Patients are at increased risk of death once this respiratory decline reaches a critical threshold.
Eteplirsen may slow the rate of respiratory decline and therefore may delay time to milestones of decrease. This may have notable positive implications on quality of life. Longer-term follow-up is needed.
Pulmonary function
The pulmonary function can be measured by assessing different parameters of lung function. As an example, the total amount of air that can be moved through the lungs after a maximal inspiration and then exhalation (forced vital capacity [FVC]). The FVC measures output of inspiratory and expiratory muscles. This is an excellent measure of respiratory function reserve and is widely used in DMD to assess respiratory function.
About Exondys 51
- Exondys 51 was approved in the US on September 2016.
- Exondys 51 is approved for a specific subset of DMD patients that are amenable to exon 51 skipping therapies. That accounts for about 13% of DMD patients.
- In September 2018, the European Medicines Agency (EMA) rejected Sarepta’s application for Exondys 51.
- NEGATIVE OPINION FOR EXONDYS® IN EUROPE
- The therapy costs about $300,000 US dollar per patient per year.
- The company is awaiting an FDA decision on its exon 53-skipping therapy, Golodirsen, this summer. It would be appropriate for about 8% of DMD patients.
- NEWS ABOUT GOLODIRSEN, SKIPPING EXON 53
- This drug is not currently available in Canada, as Health Canada must approve its use in the Canadian market.
