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- A BioMarin combination therapy helped adults with late-onset Pompe disease maintain walking distance over five years of continuous therapy.
- Respiratory function remained relatively stable, with no new safety signals identified.
- Late-onset Pompe disease is a rare disease that causes progressive muscle weakness, included in muscles used for breathing.
Piotr Swat | Lightrocket | Getty Images
On Friday, BioMarin announced five-year data from a Phase 3 trial for the drugs Pombiliti and Opfolda to treat late-onset Pompe disease, or LOPD.
The data shows that the combination therapy helped adults with the disease maintain their walking distance over five years of continuous therapy 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 marketed the combination therapy. The move expanded BioMarin’s presence in rare metabolic disease space.
The analysis followed 82 patients, including both people new to enzyme replacement therapy and those who had been treated with an enzyme replacement therapy prior to joining this study.
LOPD is a rare, inherited lysosomal disorder that can be severe and involves progressive muscle weakness throughout the body that worsens over time. In many patients, the muscles that control breathing are impacted.
The results were presented at the 31st annual Congress of the World Muscle Society in Japan.
CNBC Cures is underwritten by Alexion, AstraZeneca Rare Disease.
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