FDA Delays Marketing Application For Experimental Pompe Disease Treatment

According to a story from Stat News, The US Food and Drug Administration (FDA) has recently sent a message to the pharmaceutical company Amicus Therapeutics in which they denies the company’s bid to file a marketing application for the company’s experimental treatment for Pompe disease. The FDA says that there is not enough clinical data available to support such an application.

About Pompe Disease

Pompe disease, which is also known as glycogen storage disease type II, is a metabolic disorder in which a deficiency of the enzyme responsible for the processing of glycogen allows the substance to build up in body tissue, causing damage to neurons and muscles. The disorder is caused by a genetic mutation. The disease can appear at birth, but there is a late onset form of Pompe disease as well. Symptoms include enlarged heart, poor muscle tone, heart disease, failure to thrive, difficulty breathing, muscle weakness, and trouble feeding. The late onset form typically has a better prognosis, and outcomes are dependent on the onset and severity of symptoms. Treatment mostly involves addressing symptoms and enzyme replacement therapy, which can prolong survival substantially. Early intervention is essential for the best results. To learn more about Pompe disease, click here.

More Data Needed

The investigational product that is under development by Amicus is called AT-GAA. The request for more data from the FDA means that the company will have to delay its application until some time next year. Unfortunately for Amicus, the company is facing a similar setback in the EU. There, the EU has ordered Amicus to conduct a larger scale clinical trial that will compare AT-GAA to the current treatment standard for Pompe disease. This trial is expected to involve 100 patients.

Accelerated Approval

It should be noticed that Amicus was not seeking to go through the normal channels to get AT-GAA and were hoping to get accelerated approval for the drug. This approval was only based on data from a Phase II clinical trial. Results from the trial were encouraging, and patients displayed better lung function and walking ability. This initial data was from ten patients, and the company plans to treat an additional ten in order to obtain the new data that the FDA requires.


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