Pombiliti plus Opfolda selected as winner of Galien pharma award
Award recognizes excellence in scientific innovation to improve human health
Pombiliti plus Opfolda, a combination treatment developed by Amicus Therapeutics for adults with late-onset Pompe disease (LOPD), has been selected as the winner of the 2024 Prix Galien U.K. Award for Best Pharmaceutical Product.
This award is given by the Galien Foundation to recognize companies that have helped push the development of new medications that improve human health. The award “is regarded among the highest accolades for biopharmaceutical and medical technology research,” according to a press release from Amicus.
“It is a great honor for Pombiliti + Opfolda to be awarded the prestigious Prix Galien U.K. Award for Best Pharmaceutical Product,” said Bradley Campbell, president and CEO of Amicus. “We believe that this award embodies the dedication and continued success of our team at Amicus, together with patients, physicians and the broader Pompe community, to develop and deliver the first and only two-component therapy for eligible adults living with late-onset Pompe disease.”
Pompe disease is a disorder caused by mutations in the gene that provides instructions to make the enzyme acid alpha-glucosidase (GAA). This enzyme is normally needed to break down a sugar molecule called glycogen, which the body normally uses as a form of energy storage. In Pompe, lack of functional GAA leads to a toxic buildup of glycogen that damages cells, particularly muscle cells, ultimately leading to disease symptoms.
Therapy aims to enhance walking ability, respiratory function
Pombiliti contains a working version of the GAA enzyme (cipaglucosidase alfa), which is administered via infusion into the bloodstream every two weeks. Opfolda (miglustat) is given as oral capsules taken before Pombiliti infusions that can act as a molecular chaperone, stabilizing the enzyme so it maintains its activity. Working in concert, the two-part therapy is intended to enhance walking ability and improve or stabilize respiratory function.
After benefits shown in clinical trials, including the Phase 3 PROPEL (NCT03729362) study that compared Pombiliti plus Opfolda to the enzyme replacement therapy alglucosidase alfa (marketed as Lumizyme in the U.S.), the therapy was approved last year for certain adults with LOPD in the U.K., the U.S., and the European Union.
“This therapy is the realization of the hard work and efforts of so many individuals dedicated to the mission to make medicines that have a meaningful impact on the lives of people living with rare diseases like Pompe disease,” Campbell said. “We are grateful to the Galien Foundation for this very important recognition.”
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