Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access

According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP) for their investigational therapy pegunigalsidase alfa. This program will be for the proposed use of the drug as a treatment for Fabry disease, a rare disease. Meanwhile, the US Food and Drug Administration (FDA) is currently evaluating the Biologics License Application (BLA) for the medication.

About Fabry Disease

Fabry disease is a rare genetic disorder that primarily affects the heart, skin, and kidneys. As a lysosomal storage disease, it is characterized by a deficiency in the enzyme responsible for processing sphingolipids, which accumulate in the body as a result. The disorder is caused by mutations of the GLA gene. Symptoms include pain (which can affect the extremities, the entire body, or the digestive tract), kidney dysfunction, abnormalities of the heart valve and heart rhythms, fatigue, inability to sweat, and angiokeratomas (small red dots that appear on the skin). Treatments include enzyme replacement therapy, treatments to address organ specific problems, and Galafold. Galafold is effective in roughly 50 percent of patients, and only works for patients with certain types of mutations. Enzyme replacement therapy can help partially halt or reverse disease progression. To learn more about Fabry disease, click here.

About The Program

As of now, pegunigalsidase alfa is only available to patients that are involved in the ongoing phase 3 clinical trial. The EAP will allow the treatment to be used on other patients under certain circumstances, such as when the patient’s doctor determines that the patient will not receive satisfactory results using currently available therapies. The drug has received FDA Priority Review. Participants in the program will receive a dose level of 1 mg/kg body weight and data on adverse effects and other factors will be collected.

There is a significant need for more therapeutic options for Fabry disease, as some patients do not experience major benefits from the the current treatments. This problem is even further compounded by the fact that the number of newly diagnosed Fabry disease is increasing and even surpassing scientific predictions.

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