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High materiality7/10

BioMarin's five-year PROPEL data reinforce POMBILITI + OPFOLDA durability in LOPD

Oct 2, 2026, 11:14 AM EDT1 sourcesAI-analyzed

Why it may matterVerify against the original reporting

Durable five-year data for LOPD support long-term value of POMBILITI + OPFOLDA, appealing to biotech investors and potentially lifting sentiment and multiple on BioMarin's rare-disease portfolio; limited near-term catalysts but strengthens pipeline credibility.

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BioMarin reported five-year PROPEL open-label extension results for POMBILITI + OPFOLDA in late-onset Pompe disease, showing durable motor function and stabilized pulmonary decline with manageable safety. The gains were larger in previously untreated patients, while ERT-experienced users demonstrated smaller but positive improvements; data were presented at the World Muscle Society Congress in Hiroshima. These long-term findings support the therapy's value proposition and could bolster investor sentiment around BioMarin's rare-disease portfolio, particularly if ongoing follow-ups continue to confirm durability.

Five-year PROPEL data show durable motor function in late-onset Pompe disease.
ERT-experienced: 6MWD +0.7%; naïve: +10.5% at Year 5.
FVC declines modestly; 51.2% experienced treatment-related AEs; 5 discontinuations.
No new safety signals identified; long-term data bolster POMBILITI + OPFOLDA.
BMN 351 (nivudirsen) shows positive dystrophin expression in Duchenne muscular dystrophy.

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