Switching to Pombiliti + Opfolda may help boost mobility in LOPD
Patients report other measures tended to be better after making the switch
Switching from Lumizyme (alglucosidase alfa) to Pombiliti + Opfolda (a combination of cipaglucosidase alfa and miglustat) may help patients with late-onset Pompe disease move around more easily, suggesting a shift toward better quality of life, according to an exploratory analysis of Phase 3 data.
The Phase 3 PROPEL trial (NCT03729362) tested the safety and efficacy of Pombiliti + Opfolda in adult patients who had never received enzyme replacement therapy (ERT) or who had been treated with Sanofi’s Lumizyme for at least two years.
The findings support Pombiliti + Opfolda as “an important new treatment option,” researchers wrote.
Their study, “Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease,” was published in the Journal of Patient-Reported Outcomes. It was funded by Amicus Therapeutics, the company that markets Pombiliti + Opfolda. Two study authors are employees at Amicus.
Pompe disease occurs when the body cannot break down glycogen, a complex sugar stored in muscles, because an enzyme called acid alpha-glucosidase (GAA) is absent or defective. As a result of muscle weakness, patients often experience limitations in activities of daily life. In the late-onset form of Pompe disease, symptoms typically begin to appear after 1 year of age through to adulthood. Typically, the earlier the disease is diagnosed in life, the more severe the symptoms.
Pombiliti + Opfolda contains man-made GAA enzyme plus stabilizing compound
ERT, an approach that supplies the body with the missing enzyme, is the standard treatment for Pompe. Lumizyme is approved to treat all types of Pompe, and is marketed as Myozyme outside the U.S. Pombiliti + Opfolda contains a man-made form of the GAA enzyme (cipaglucosidase alfa) and a pharmacological compound that stabilizes the active enzyme (miglustat). It is approved in the U.S. and elsewhere for adults with LOPD.
Data from the PROPEL trial showed Pombiliti + Opfolda outperformed Lumizyme at helping patients with LOPD walk farther, though the difference was only statistically significant in the subset of patients who had been on prior ERT. Benefits of Pombiliti + Opfolda also included a lesser decline in lung function.
In the new study, the researchers focused on patient-reported outcomes, which are tools used to measure how patients perceive their physical and mental health. These included the Subject’s Global Impression of Change, meaning the difference between the patient’s current and previous health state (in domains such as physical well-being, breathing, and ability to move around), and the physical function and fatigue forms of the Patient-Reported Outcome Measurement Information System.
After 52 weeks (one year), a significantly greater proportion of Pompe disease patients in the Pombiliti + Opfolda group than in the Lumizyme group reported they had improved in moving around (90% vs. 59%). Other measures, such as fatigue, self-care, physical function, and mental health, tended to be better in patients treated with Pombiliti + Opfolda, but the differences were not statistically different.
For the researchers, this study “highlights the importance of capturing the patient’s perspective in LOPD,” they wrote. “Continued research will elucidate the long-term and real-world impact of [Pombiliti + Opfolda] on patients’ quality of life.”
Study limitations, according to the investigators, included the exploratory nature of the analysis, a consequence of PROPEL not being powered to compare Lumizyme to Pombiliti + Opfolda for patient-reported outcomes. More studies are needed to confirm the long-term benefits of Pombiliti + Opfolda on such measures, they added.
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