BioMarin reported five-year follow-up data for Pombiliti and Opfolda in people with late-onset Pompe disease, showing maintained motor-function measures and relatively stable pulmonary function in an open-label extension study.
The analysis included 82 participants who received the combination continuously from the start of the Phase 3 PROPEL study. Sixty-two had previously received enzyme replacement therapy for an average of 7.6 years before entering PROPEL, while 20 had not previously received such treatment.
Among participants previously treated with enzyme replacement therapy, the mean change from baseline in percent-predicted six-minute walk distance was 0.7% at year five. Mean change in percent-predicted forced vital capacity, a measure of lung function, was negative 2.8%.
In the treatment-naïve group, followed through year 4.5, mean percent-predicted six-minute walk distance increased 10.5% from baseline. Mean percent-predicted forced vital capacity declined 4.4%.
No new safety signals were identified, BioMarin said. Treatment-related adverse events were reported in 42 participants, or 51.2% of the study population. The company said most of those events were manageable.
Five participants discontinued treatment for safety reasons. Two stopped treatment because of serious treatment-related adverse events: one case involving chills and hypoxia, and one anaphylactoid reaction.
Pombiliti, or cipaglucosidase alfa-atga, is used with Opfolda, or miglustat, in late-onset Pompe disease. Pompe disease is a rare genetic disorder that can cause progressive muscle weakness and respiratory impairment.
BioMarin presented the results from the PROPEL open-label extension at the World Muscle Society’s annual congress in Hiroshima, Japan. The company also presented updated Phase 1/2 data for nivudirsen, an investigational exon 51-skipping treatment for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy.
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