ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma

CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been approved for multiple conditions, all related to the immune system. These designations will help CSL Behring with marketing and will give financial incentives to continue development.

About Systemic Scleroderma

Systemic scleroderma is an autoimmune disease that is also known as systemic sclerosis. It is causes an accumulation of scar tissue in the skin and organs, resulting in the thickening of the skin. It is estimated to affect between 40,000 and 165,000 in the United States and typically affects females more than males.

Characteristic symptoms include fibrosis, Raynaud’s phenomenon, swelling in the hands and fingers, open sores on the fingers, heartburn, calcium deposits under the skin, difficulty swallowing, high blood pressure, joint pain, gastrointestinal malfunction, diarrhea, shortness of breath, and kidney problems. In severe cases, fibrosis leads to impairment and failure of the organs.

These symptoms are the result of an immune system malfunction. The majority of cases are sporadic, and doctors are unsure of the exact reason that this disease occurs. They do know that collagen builds up and leads to fibrosis.

A diagnosis may occur after a number of different tests, such as X-rays, biopsies, blood tests, physical exams, CT scans of the lungs, breathing tests, and echocardiograms. After a diagnosis is obtained, treatment consists of symptom management. Doctors may recommend medications to treat the fibrosis, occupational therapy, surgery, pain relievers, physical therapy, antibiotics, medications that suppress the immune system, blood pressure drugs, and medications to combat stomach acid.

About Privigen

Privigen is an IV immunoglobulin (IVIG) therapy. It is aimed at the immune system, as it delivers antibodies to help with immune responses. It has been approved by the FDA for the treatment of multiple immune system disorders, such as primary immunodeficiency. Evidence has shown that it will be beneficial in the treatment of systemic scleroderma, as it is believed to have immunomodulatory effects and helps to reduce fibrosis.

Phase 1 of a clinical trial studied the safety and effect of Privigen on 14 participants over the course of six months. Participants were required to have a history of scleroderma and have received ineffective treatment for it. It will continue to be evaluated in Phase 2 of the trial, which will be placebo controlled. 144 people are already enrolled, and researchers are still accepting participants. Researchers are hopeful that Privigen will have similar effects on systemic scleroderma as it has had on other immune system disorders.

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