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In BioMarin’s report on adults with late-onset Pompe disease (LOPD) who continued POMBILITI (cipaglucosidase alfa-atga) plus OPFOLDA (miglustat), the ERT-experienced group’s average percent-predicted six-minute walk distance was nearly unchanged through year five: up 0.7 percentage points from the PROPEL baseline. Its average percent-predicted forced vital capacity fell 2.8 points. The ERT-naïve group’s reported results run only through year 4.5, not five years.
These are group averages from an open-label extension, not a controlled five-year comparison or a guarantee that each participant maintained function.
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What the reported walking and lung-function results show
BioMarin announced the findings on October 2, 2026, following their presentation at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan. The results concern participants who continued the two-drug combination from the start of the Phase 3 PROPEL study.
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| Prior enzyme replacement therapy (ERT) status | Baseline participants | Reported follow-up | Mean change in pp6MWD | Mean change in ppFVC |
|---|---|---|---|---|
| ERT-experienced | 62 | Through year 5 | +0.7 percentage points (SD 7.76) | −2.8 percentage points (SD 8.55) |
| ERT-naïve | 20 | Through year 4.5 | +10.5 percentage points (SD 11.44) | −4.4 percentage points (SD 6.89) |
Here, pp6MWD means percent-predicted six-minute walk distance, a measure of walking capacity; ppFVC means percent-predicted forced vital capacity, a measure of lung function. The reported figures are mean changes from PROPEL baseline, expressed in percentage points. The ERT-experienced participants had received ERT for a mean of 7.6 years at baseline.
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The figures come from BioMarin’s October 2, 2026 announcement. The higher pp6MWD average in the ERT-naïve group should not be read as evidence that prior ERT caused a different outcome: the subgroups were not presented as a randomized comparison, and their follow-up durations differ.
How to interpret “durable motor function”
The ERT-experienced group’s mean walking-distance change of +0.7 percentage points through year five is consistent with an average that remained close to its PROPEL baseline. It does not show that every participant stayed stable. The standard deviation indicates variation around the mean, but the announcement does not provide individual trajectories or year-specific participant counts.
The data are from ATB200-07 (NCT04138277), a multicenter international open-label extension for adults who completed the Phase 3 PROPEL study (ATB200-03; NCT03729362). PROPEL’s primary endpoint was six-minute walk distance. The extension also tracks FVC, creatine kinase, Hex4, patient-reported outcomes, and safety.
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Because this is an extension report without a concurrent control comparison for these long-term changes, it cannot by itself establish the treatment’s causal effect over five years or quantify how attrition may have affected the averages. BioMarin calls the findings durable benefits; that is the company’s interpretation of the reported results, not an independent clinical assessment. The announcement names a late-breaking presentation, “Long-Term (Five-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline,” e-poster #3.081LBeP.
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Safety findings in the extension
BioMarin reported no new safety signals. It said 42 participants (51.2%) experienced treatment-related adverse events, most of which were manageable, and five discontinued treatment for safety reasons. Two discontinuations were attributed to serious treatment-related events: chills and hypoxia in one participant, and an anaphylactoid reaction in another.
POMBILITI is an enzyme replacement product; OPFOLDA is an oral enzyme stabilizer. BioMarin’s announcement describes the U.S. indication as adults with LOPD weighing at least 40 kg who are not improving on their current ERT. The company also summarizes risks including serious hypersensitivity, infusion-associated reactions, cardiorespiratory failure, and pregnancy and embryo-fetal risks. For current warnings and treatment decisions, consult the full prescribing information and a clinician.
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What the announcement does not establish
- It does not show that every participant maintained or improved walking capacity or lung function.
- It does not give year-specific denominators or individual patient trajectories for the reported long-term changes.
- It does not provide a concurrent control comparison that would isolate the combination’s causal effect over five years.
- The ERT-naïve results shown here extend to year 4.5; they are not five-year outcomes.
The release says the extension treated 118 people in total, including 82 who continued combination therapy from PROPEL and 37 who switched from its comparator arm. Those subgroup counts add to 119; BioMarin’s release does not explain the one-participant discrepancy. The detailed conference poster and its granular methods are not included in that announcement.
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