News TG Therapeutics’ Potential Neuromyelitis Optica Treatment Named Orphan Drug by FDA TG Therapeutics’ Potential Neuromyelitis Optica Treatment Named Orphan Drug by FDA by Patricia Silva, PhD | August 30, 2016 Share this article: Share article via email Copy article link TheĀ U.S. Food and Drug AdministrationĀ (FDA) has designated TG-1101Ā (ublituximab), aĀ glycoengineered anti-CD20 monoclonal antibody byĀ TG Therapeutics, anĀ orphan drugĀ to advance its development. The drug is a potentialĀ treatment for neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD), two relapsing-remitting autoimmune diseases with similarities to multiple sclerosis. Currently, there are no FDA-approvedĀ treatments for theseĀ disorders. “We are pleased to announce our first orphan drug designation for TG-1101 in a non-oncology indication, providing additional proprietary protection for TG-1101,”Ā Michael S. Weiss, executive chairman and interim CEO of TG Therapeutics, said in a press release. “NMO is closely related to Multiple Sclerosis, an area of significant interest to us. We look forward to presenting early data from our current Phase 1b study of TG-1101 in NMO at the ECTRIMS (European Committee for the TreatmentĀ and Research in Multiple Sclerosis) conference this September, which we believe will provide an early peek into the effects of TG-1101 in patients with autoimmune diseases,” Weiss added. An orphan drug designationĀ offers special status to a drug or biological product that is intended for the effective and safe prevention, diagnosis, or treatment of a rare disease or condition that affects less than 200,000 people in the United States. ItĀ qualifies the treatment’s sponsor for various development incentives, including tax credits for qualified clinical testing. NMOSD is a recently proposed unifying term for neuromyelitis optica ā also known as Devic’s disease ā and its related syndromes. It is a relapsing inflammatory demyelinating disease that most commonly affects optic nerves and the spinal cord, leading to sudden vision loss or weakness in one or both eyes, and a loss of sensation and bladder function. During a relapse, new damage to the optic nerves and/or spinal cord can lead to accumulating disability, as in MS. Unlike MS, however, there is no progressive phase inĀ this disorder, so that preventing attacks is a critical treatment approach. NMO is estimated to affect about five in every 100,000 people. TG Therapeutics is also assessing TG-1101 in patients with relapsing-remitting MS (RRMS)Ā in a Phase 2 clinical trial (NCT02738775) in the U.S. that is currently recruiting patients. Print This Page About the Author Patricia Silva, PhD PatrĆcia holds a PhD in medical microbiology and infectious diseases from the Leiden University Medical Center, Netherlands, and completed a postdoctoral research fellowship at the Instituto de Medicina Molecular, Lisbon, Portugal. Her work in academia was mainly focused on molecular biology and the genetic traits of infectious agents such as viruses and parasites. PatrĆcia earned several travel awards to present her work at international scientific meetings. She is a published author of several peer-reviewed science articles. Tags FDA, NMOSD, TG Therapeutics, TG-1101, ublituximab
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