Piotr Swat | Light Rocket | Getty Images
On Friday, BioMarin announced five-year data from a Phase 3 trial of its drugs Ponbility and Opfolda to treat late-onset Pompe disease (LOPD).
Data show that the combination therapy helped adults with the disease maintain walking distance over five years of continuous treatment and keep respiratory function relatively stable in the long term. No new safety signals were identified in the data.
In December, BioMarin acquired Amicus Therapeutics, which sells combination therapies. This move expands BioMarin’s presence in the rare metabolic disease space.
The analysis followed 82 patients, including both those who were new to enzyme replacement therapy and those who had been treated with enzyme replacement therapy before joining the study.
LOPD is a rare, inherited lysosomal disease that can be severe and associated with progressive muscle weakness throughout the body, which worsens over time. In many patients, the muscles that control breathing are affected.
The results were presented at the 31st World Muscle Society Annual Meeting in Japan.
CNBC Cures is underwritten by AstraZeneca Rare Diseases Alexion.
