The experimental drug discussed in this article, ACE-1334, is for systemic sclerosis which is a lung disease. Unfortunately, it's not for multiple sclerosis.
We’re excited that ACE-1334 has received Orphan Drug designation, as it aligns with our vision to develop novel therapies for pulmonary diseases of high unmet medical need,” said Habib Dable, President and Chief Executive Officer of Acceleron.
Systemic sclerosis-associated interstitial lung disease (SSc-ILD; also known as systemic scleroderma-associated interstitial lung disease) is a rare, progressive, autoimmune connective tissue disorder characterized by immune dysregulation. It is reported to be the most common cause of death among patients with systemic scleroderma. Patients with SSc-ILD ultimately experience severely compromised pulmonary function, with the disease initially causing cough, shortness of breath, and fatigue. SSc-ILD, which affects an estimated 50,000 patients in the United States and Europe, is thought to arise from a combination of genetic susceptibility, autoimmunity, inflammation, fibrotic activity, and vascular insult. The disease has an estimated 10-year survival rate of approximately 56%. Current treatment for SSc-ILD includes a range of potentially high-risk approaches, including immunosuppression, hematopoietic stem cell transplantation, and lung transplantation.