The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) status to AMT-130, an experimental gene therapy for Huntington’s disease, the first time a Huntington’s treatment candidate has received such a designation. The RMAT designation is given to therapies that seek to treat, modify,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SAGE-718, Sage Therapeutics’ experimental oral therapy for Huntington’s disease. The FDA gives this designation to therapies that have the potential to treat rare diseases, which are defined as conditions that affect fewer than 200,000 people…

The U.S. Food and Drug Administration (FDA) will review Neurocrine Biosciences’ application of a new oral granule formulation of Ingrezza (valbenazine) for involuntary muscle contractions, or chorea, in adults with Huntington’s disease. The therapy was recently approved in the U.S. for Huntington’s-associated chorea in the form…

Austedo XR, a new extended-release formulation of Austedo (deutetrabenazine) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with chorea — involuntary muscle contractions — associated with Huntington’s disease. Austedo XR contains the same active agent as the original therapy, but is taken just once a day, with…

Treatment with valbenazine significantly lessened chorea — a motor symptom characterized by jerky, unpredictable, and involuntary movements — in people with Huntington’s disease in the Phase 3 clinical trial KINECT-HD, according to an announcement from the therapy’s developer, Neurocrine Biosciences. Neurocrine now is planning to submit an…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

MMJ International Holdings, a pharmaceutical company focused on the development of cannabis-based therapeutic products, will soon begin testing its oral cannabidiol (CBD) and tetrahydrocannabinol (THC) products in U.S. clinical trials for Huntington’s disease and multiple sclerosis. The U.S. Drug Enforcement Administration (DEA) cleared MMJ to import…