Amicus Therapeutics recently announced FDA approval for their combination treatment for Pompe disease, a rare enzyme deficiency condition. This approval furnishes the pharmaceutical company with the opportunity to support patients as yet inadequately served by the current standard of care, specifically treatments by Sanofi. Globenewswire has reported that the granted approval extends to the treatment of late-onset Pompe-adults who are not experiencing improvement from present enzyme replacement therapies.
Pompe disease, as explored by the Cleveland Clinic, results from patients’ inability to produce an enzyme called acid alpha-glucosidase (GAA), which is responsible for breakdown of a sugar known as glycogen. The ensuing accumulation of GAA inflicts damage on muscles inducing progressively deteriorating respiratory issues and walking difficulties.
The Philadelphia-based company uses a two-pronged strategy in its combat against Pompe. They use a re-engineered variant of the deficient enzyme, administered intravenously, in tandem with miglustat, a small molecule that mediates the stability of Pombiliti within the bloodstream, as such, enabling muscle cells to optimally absorb the engineered enzyme.
While the clinical trial results of the Amicus combination therapy were not significantly better than those for Sanofi’s Lumizyme, the therapy did demonstrate a statistically significant improvement in forced vital capacity, a relevant measure of lung function, and a key secondary objective of the study. Data from an open-label extension study further showed sustained improvements in muscle function, walking, and breathing among treated patients over two years. Notably, there was also evidence of reduced biological indicators of muscle damage.
Despite disruptions due to Covid-19 travel restrictions delaying reviews and inspections of the manufacturing facility, Amicus project availability of the Pompe treatment within two weeks, as noted in an investor presentation. Furthermore, the company estimates that the U.S. Pompe market presents a $500 million opportunity, extending to $1.2 billion globally. Amicus anticipates that the combination therapy could reach peak sales of approximately $1 billion.
This latest FDA approval represents the second commercialized therapy by Amicus. Their first product, Galafold, treats Fabry disease, another rare enzyme deficiency. In a bid to maintain broad access to the treatment, the CEO, Brad Campbell, committed to ensure that price increases, for the two products combined, will not exceed the consumer price index.