Treatment options for Duchenne muscular dystrophy (DMD) have been limited and challenging to develop due to the progressive nature of the disease. Viltolarsen (Viltepso) is one of the latest exon-skipping therapies that has shown promising results in improving pulmonary function in boys and men with DMD, according to the results of the open-label, phase II Galactic53 trial presented at the American Academy of Neurology annual meeting.
The results from the study indicated a significant improvement in pulmonary function, as measured by percent predicted forced vital capacity (FVC%p) and peak cough flow (PCF), in participants receiving viltolarsen. Among ambulatory participants, 90% showed an increase or stabilization in FVC%p from baseline, with a statistically significant overall change at week 49. Additionally, nonambulatory patients also demonstrated an increase in FVC%p with viltolarsen treatment, in contrast to a decrease seen in the control group.
The findings of the study suggest an additional treatment benefit of viltolarsen for patients with DMD who are amenable to exon 53-skipping therapy. This is particularly significant as previous data on pulmonary function in nonambulatory patients were limited, making these results encouraging for the broader DMD population.
Viltolarsen was granted accelerated approval status by the FDA in 2020, highlighting the urgent need for effective treatments for DMD. A confirmatory phase III trial is currently underway to further assess the clinical benefit of the drug in DMD patients with a confirmed dystrophin gene mutation amenable to exon 53 skipping. This ongoing research will provide more insights into the long-term efficacy and safety of viltolarsen in a larger patient population.
The study also compared viltolarsen with other exon 53-skipping therapies that have received accelerated approval, such as golodirsen and eteplirsen. While these therapies have shown some benefits in improving muscle function, viltolarsen’s focus on pulmonary function marks a unique contribution to the treatment landscape for DMD.
The safety profile of viltolarsen appeared to be favorable, with most participants experiencing mild-to-moderate treatment-emergent adverse events that were generally well-tolerated. There were no serious adverse events, discontinuations due to adverse events, or deaths reported during the study, indicating a good overall safety profile for viltolarsen.
The results of the Galactic53 trial demonstrate the potential of viltolarsen in improving pulmonary function in boys and men with Duchenne muscular dystrophy. The data presented at the American Academy of Neurology meeting support the continued development and investigation of viltolarsen as a promising treatment option for patients with DMD amenable to exon 53-skipping therapy. Further research and clinical trials will be essential to confirm these findings and establish the long-term benefits of viltolarsen in managing this complex and devastating disease.
Leave a Reply