Amicus Therapeutics and the FDA Have Met to Discuss the Path for a Potential Pompe Disease Therapy


Amicus Therapeutics has met with the United States Food and Drug Administration to discuss the investigational drug AT-GAA, which is being researched as a potential treatment for Pompe disease. The FDA advised Amicus on a clinical study that Amicus hopes could support approval of the drug, and on what evidence would be needed for Accelerated Approval. For more detailed information you can view the source press release here, at Globe Newswire.

About Pompe Disease

Pompe disease, also known as glycogen storage disease type II, is a genetic disorder caused by the build-up of the sugar glycogen in cells, impairing their functioning. The condition progresses at different rates in different people and can first begin to affect people at different ages. Typically, people who develop the condition at a younger age tend to experience more severe symptoms, and these can become fatal. One of the main features that span all of three of these types of Pompe disease is muscle weakness and wasting, which can cause people mobility problems and, in many cases, affect breathing.

There are several existing therapies to alleviate the symptoms of Pompe disease, but new and more effective treatments are urgently needed.

About AT-GAA

AT-GAA is an investigational therapy being developed as a potential treatment for Pompe disease. It is made up of an enzyme (rhGAA) and another compound. Pre-clinical studies of AT-GAA have shown that it is linked to increased tissue enzyme levels, lower levels of glycogen in muscle, and more muscle strength. The drug is currently being researched further in a Phase 1/2 study to establish its safety and effects.

Feedback from the FDA

Amicus, who are developing AT-GAA, held a meeting with the FDA to discuss what the path to approval could be for the therapy. The discussion included feedback from the FDA about the design of a study of AT-GAA that Amicus hope could be used to gain FDA approval, and whether the therapy would be eligible for Accelerated Approval.

The FDA emphasised the need to get “new treatments to Pompe patients as fast as possible”, but said that AT-GAA doesn’t currently have enough data to qualify for Accelerated Approval. In response, Amicus has said that it plans to produce more data about the therapy that can be provided to the FDA.

The pivotal study that was discussed with the FDA is planned to involve roughly one hundred patients with Pompe disease and will compare AT-GAA to the current standard of care.

There are also plans to carry out studies in other patient groups next year, including paediatric Pompe patients.

Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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