CAEL-101 from Fortress Biotech (FBIO) Receives Orphan Drug Designations for Amyloidosis
Release Date: 07-Apr-2017
U.S. Department of Health and Human Services has confirmed the transfer of two U.S. Food and Drug Administration (FDA) Orphan Drug Designations for CAEL-101 (also known as 11-1F4) from Columbia University (“Columbia”) to Fortress’ subsidiary, Caelum Biosciences, Inc. (“Caelum”). The two Orphan Drug Designations include the use of CAEL-101 as a therapeutic agent for patients with AL amyloidosis, and the use of CAEL-101 as a radio-imaging agent in amyloidosis.
Fortress Biotech, Inc. is a bio-pharmaceutical company focused to attain, develop and commercialize novel pharmaceutical and biotechnology products. CAEL‐101 is a fibril‐reactive monoclonal antibody (mAb) currently being evaluated in a Phase 1b study for the treatment of AL amyloidosis, a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow.
FDA Orphan Drug Designation is granted to investigational therapies that address rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.
“Global Orphan Drug Clinical Pipeline Insight” reports by kuick research gives comprehensive insight on various clinical and non-clinical aspects associated with ongoing clinical trials of 808 orphan designated drugs across the globe. The in-depth clinical insight presented in the report helps the reader to analyze and identify the various stakeholders involved in the clinical development and commercialization of orphan designated drugs in the global market. Currently there are more than 300 orphan designated drugs commercially available in the global market and around 800 drugs in clinical development phase.
