Amicus Therapeutics is developing a treatment for Pompe disease through its AT-GAA program. They have recently announced that they have met with regulators from the European Union and Germany who have given Amicus clinical and manufacturing advice for how to proceed with the AT-GAA program. You can read the full article here, at Globe Newswire.
About Pompe Disease
Pompe disease, also known as glycogen storage disease type II, is a condition caused by a build-up of glycogen in cells. This prevents tissues, such as muscles and organs, from functioning normally. There are three main forms of the condition: classic infantile-onset, non-classic infantile onset, and late onset. The typical symptoms and severity differ between the three types, but common effects of the condition include muscle weakness, heart problems (although this is less common for late-onset), and breathing difficulties. However, individual experiences differ significantly. Pompe is thought to affect approximately five to ten thousand people worldwide.
The Regulators’ Advice
Amicus met with two regulatory authorities: the Scientific Advice Working Party (SAWP) from the European Union, and German authorities (BfArM).
The SAWP advised Amicus about how to best carry out a planned pivotal study. A pivotal study is designed to provide support for drug marketing approval. The pivotal study is planned to take place later this year (pending FDA feedback) and enrol eighty patients who will be treated for up to one year. It would be open to patients who are currently taking part in Amicus’s on-going Stride Study 003. The SAWP also supported including more groups of Pompe patients, including pediatric and ERT-treatment naïve patients in research, including studies planned to begin in 2019.
However, the SAWP also decided that there is not currently enough information about the experimental treatment AT-GAA for a Conditional Marketing Authorization Application to be submitted, and more information needs to be gathered. The European Medicines Agency says that Conditional Marketing Authorizations are given to experimental treatments where “the benefit of immediate availability outweighs the risk of less comprehensive data than [is] normally required.” However, the SAWP did describe current data on AT-GAA as “promising.”
The meeting between Amicus and BfArM focused on the manufacturing process for ATB200, and a general strategy was agreed upon.
About AT-GAA (ATB200/AT2221)
ATB200/AT2221 is an investigational drug combination that is currently being developed as a possible treatment for Pompe. It is made up of two components; ATB200, which contains an enzyme, and AT2221, which is a ‘pharmacological chaperone’, meaning that it is hoped to help proteins fold into the correct shape to function. Early preclinical studies support the effectiveness of the experimental treatment, and current clinical trials are further investigating this.
