{"id":8173,"date":"2019-04-15T16:28:51","date_gmt":"2019-04-15T16:28:51","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=8173"},"modified":"2022-05-03T18:11:35","modified_gmt":"2022-05-03T18:11:35","slug":"fibrogen-orphan-drug-designation-pamrevlumab","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/fibrogen-orphan-drug-designation-pamrevlumab\/","title":{"rendered":"FibroGen receives orphan drug designation for Pamrevlumab"},"content":{"rendered":"

FibroGen Receives Orphan Drug Designation from the U.S. FDA For Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy<\/strong><\/h2>\n

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FibroGen, Inc. a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company\u2019s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD).<\/h4>\n

DMD is caused by the absence of the dystrophin protein, resulting in abnormal muscle structure and function and buildup of fibrosis in muscle<\/strong>, which diminishes mobility, pulmonary function, and cardiac function. Constant myofiber<\/strong> breakdown results in persistent activation of myofibroblasts<\/strong> and aberrant production of extracellular matrix (ECM) proteins, including collagens and fibronectin, leading to extensive fibrosis in skeletal muscles.<\/p>\n

Pamrevlumab<\/strong> is a fully human monoclonal antibody that inhibits the activity of connective tissue growth factor<\/strong>, or CTGF<\/strong>, a critical mediator in the progression of fibrosis and related serious diseases.<\/p>\n

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Elias Kouchakji, M.D., Senior Vice President, Clinical Development and Drug Safety<\/strong> – \u201cWe are pleased to have received Orphan Drug Designation from the FDA for pamrevlumab in the treatment of DMD. There is a high unmet medical need for patients suffering from this debilitating disease needing a new treatment option. All 21 non-ambulatory DMD patients in our ongoing phase 2 study with pamrevlumab have completed the first 52 weeks of treatment. We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis. We look forward to the continued development of this investigational therapeutic.\u201d News release here.<\/span><\/a><\/p><\/blockquote>\n

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About Pamrevlumab<\/h2>\n

Pamrevlumab<\/strong> is a first-in-class antibody developed by FibroGen<\/strong> to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure.<\/strong> Pamrevlumab is advancing towards Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and pancreatic cancer. Pamrevlumab has been granted Orphan Drug Designation in IPF, pancreatic cancer, and Duchenne muscular dystrophy (DMD)<\/strong>. Pamrevlumab has also received Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with IPF and patients with locally advanced unresectable pancreatic cancer and is currently in a Phase 2 trial for DMD<\/strong>. Across all trials, pamrevlumab has consistently demonstrated excellent safety and tolerability profile to date. For information about pamrevlumab studies currently recruiting patients, please visit\u00a0www.clinicaltrials.gov.<\/a><\/span><\/p>\n

Pamrevlumab is being evaluated in ongoing Phase 2 clinical studies for the treatment of idiopathic pulmonary fibrosis, pancreatic cancer, and Duchenne muscular dystrophy.<\/h4>\n