Pompe Disease Patients on Amicus Drug Continue to Improve in Study

Xconomy New York — 

Patients treated with an experimental Amicus Therapeutics drug for Pompe disease continued to show improvement in their ability to walk, move, and breathe after as long as one year on the therapy, according to the latest clinical trial results from the rare disease drug developer.

The open-label study is small, testing the drug in just 20 patients. But the latest results build on interim data that the company released last October assessing patients at the six- and nine-month marks. Cranbury, NJ-based Amicus (NASDAQ: FOLD) is scheduled to present the latest results Thursday in San Diego at the WORLDSymposium, an annual conference on lysosomal storage disorders.

Pompe disease is an inherited disorder in which patients lack the enzyme needed to break down glycogen, a type of sugar stored in the muscle. This enzyme deficiency leads to a progressive weakening of muscle, affecting the ability for patients to move and for their hearts and lungs to function properly, eventually leading to death. Pompe affects between 5,000 and 10,000 people worldwide, according to the United Pompe Foundation.

Amicus’ drug, ABT200, was developed to deliver an engineered version of the deficient enzyme into the muscles of Pompe patients. With the latest results in hand, Amicus says that it is continuing to discuss with regulators the “best and fastest pathway” to bring its drug to Pompe patients. Here’s more on Amicus’ Pompe drug and the clinical trial.