First approved duo therapy for late-onset Pompe disease wins award
In clinical trials, Pombiliti + Opfolda helped adults walk longer distances
Amicus Therapeutics has been honored with the WORLDSymposium 2024 New Treatment Award for Pombiliti + Opfolda (cipaglucosidase alfa/miglustat), the first medication duo to be approved for adults with late-onset Pompe disease.
The award recognizes new treatments that received regulatory approval for lysosomal diseases like Pompe disease caused by a buildup of toxic materials in cells. Lysosomal diseases are rare genetic disorders that result from a lack of enzymes.
“We are honored that WORLDSymposium has recognized Pombiliti and Opfolda with the 2024 New Treatment Award,” Bradley Campbell, president and CEO of Amicus, said in a company press release.
Campbell called the award “a testament to more than a decade of dedication and commitment from Team Amicus along with dozens of families, physicians, researchers, and advocates around the world who worked tirelessly to see this medicine approved.”
“Their partnership has been pivotal in continuing our promise to lead the fight in rare diseases, and we share this award with the entire global Pompe disease community,” Campbell said.
Pombiliti + Opfolda for Pompe disease
Pompe disease occurs when there isn’t enough activity of the acid alpha-glucosidase (GAA) enzyme to break down glycogen, a complex sugar. As a result, it builds up to toxic levels and damages cells, especially muscle cells that store it as a fuel reserve.
Pombiliti + Opfolda contains cipaglucosidase alfa, a recombinant (lab-made) version of the GAA enzyme that gets taken up by muscle cells, along with miglustat, a small molecule that keeps the active GAA enzyme stable and functional over time. The treatment is expected to ease late-onset symptoms.
In clinical trials, Pombiliti + Opfolda helped adults with late-onset Pompe disease walk longer distances. It also helped them maintain lung function for up to four years, including in those who’d been on enzyme replacement therapy (ERT).
The two ingredients in Pombiliti + Opfolda were approved by the U.S. Food and Drug Administration in 2023 for adults with late-onset Pompe disease who weighed at least 40 kg (about 88 pounds) and who weren’t improving on their current ERT.
Both Pombiliti and Opfolda were approved that same year in the European Union (EU) and the U.K. for adults with late-onset Pompe disease. It had been available in the U.K. through an early access program before being approved.
Two other treatments were also recognized with a New Treatment Award. Elfabrio (pegunigalsidase alfa) is approved in the U.S. and EU for adults with Fabry disease, while Lamzede (velmanase alfa) is the first FDA-approved ERT for alpha-mannosidosis.
Amicus also won a New Treatment Award in 2017 for Galafold (migalastat), the first and only oral chaperone therapy approved for Fabry disease.
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