ION373: The first drug for AxD in clinical trial.
Drug Development
Ionis Pharmaceuticals, Inc. develops treatments target-focused on RNA. Ionis previously developed a drug to successfully treat SMA, a neuro-muscular disorder, and they have developed a new drug, ION373, for AxD, that uses the same intrathecal delivery method.
European Medicines Agency (EMA) has granted orphan drug designation to ION373 for the treatment of people with Alexander Disease. This designation provides regulatory and financial incentives to develop therapies for life threatening or chronically debilitating conditions affecting not more than five in 10,000 people in the European Union (EU) and for which there is no satisfactory method of diagnosis, prevention or treatment. Upon approval, drugs that have been granted orphan designation by the EMA receive market exclusivity for 10 years in the EU.
Zilganersen granted U.S. FDA Fast Track designation for people living with Alexander disease
October 1, 2024
Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease, an ultra-rare neurological condition
Ionis completes enrollment in pivotal trial evaluating zilganersen in people living with Alexander disease
July 18, 2024
Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition
Ionis initiates pivotal clinical study of novel antisense medicine to treat patients with Alexander disease
April 20, 2021
Innovative therapy targeting the root cause of the disease has received orphan drug designation by the U.S. FDA and European Medicines Agency- ION373 is one of Ionis' wholly owned rare disease medicines the company plans to commercialize