The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Trethera Corporation’s experimental oral therapy TRE-515 for demyelinating optic neuritis, an eye condition that may progress to multiple sclerosis (MS) or occur during the disease course. The first-in-class therapy is expected to lessen the inflammation…
Orphan drug status
FFF Enterprises and Bionews Services, publisher of this website, announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone.
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to combinations of VTS-Aspirin plus fumarate therapy using Vumerity (diroximel fumarate) or Bafiertam (monomethyl fumarate) for easing flush in patients with relapsing-remitting multiple sclerosis (RRMS). In the U.S., orphan drug designation is given to…
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…
The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…
Here’s my Pick of the Week’s News, as published in Multiple Sclerosis News Today. Gilenya-like Therapy Shows Benefit in Secondary Progressive MS Patients in Phase 3 Trial Here’s a potentially encouraging development for anyone with SPMS. Patients with secondary progressive multiple sclerosis (SPMS) who were treated with BAF312 (siponimod),…
The U.S. Food and Drug Administration (FDA) has designated TG-1101 (ublituximab), a glycoengineered anti-CD20 monoclonal antibody by TG Therapeutics, an orphan drug to advance its development. The drug is a potential treatment for neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD), two relapsing-remitting autoimmune diseases with similarities to multiple sclerosis. Currently, there are…
Nutra Pharma, a US-based biotechnology a company specializing in the acquisition, licensing, and commercialization of pharmaceutical products and technologies for the management of neurological disorders, cancer, autoimmune, and infectious diseases, recently announced that it has filed an application with the US Food and Drug Administration (FDA) for orphan drug status…
The Food and Drug Administration granted Orphan Drug Designation to Inhibikase Therapeutics’, Inc. lead product, IkT-001Pro (imatinib), to treat progressive multifocal leukoencephalopathy (PML). According to the National Institute of Neurological Disorders and Stroke, PML affects the white matter of the brain usually through a virus known as Polyomavirus JC (JC…