Fabry gene therapy candidate granted FDA’s orphan drug status
Developer uniQure now testing AMT-191 in Phase 1/2 trial in US
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to AMT-191, an investigational one-time gene therapy being developed by uniQure to treat Fabry disease, the company announced.
This status is granted to treatments developed for rare diseases affecting fewer than 200,000 people in the U.S. It offers several benefits, including tax credits, fee waivers, and seven years of market exclusivity following potential approval.
uniQure is now testing AMT0191 in a Phase 1/2a clinical trial in the U.S.
“This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of delivering meaningful benefit given the suboptimal effectiveness of current chronic treatments,” Walid Abi-Saab, MD, uniQure’s chief medical officer, said in a company press release. “We look forward to rapidly generating clinical proof-of-concept data and providing initial data in 2025.”
Clinical trial in Virginia still recruiting participants
Fabry disease is caused by mutations in the GLA gene that lead to a deficiency in alpha-galactosidase A (alpha-Gal A), an enzyme needed to break down certain fats in the body. Without this enzyme, fats accumulate in cells, causing a range of symptoms.
AMT-191 uses a lab-modified, harmless adeno-associated virus 5 (AAV5) to deliver a healthy copy of the GLA gene to the liver. The liver then produces the alpha-Gal A enzyme, which helps break down the accumulated fats. The gene therapy is administered as a single infusion directly into the bloodstream, or intravenously.
The company started testing AMT-191 this summer in an open-label clinical trial (NCT06270316) in Virginia. This trial will involve up to 12 men, divided into two groups, who will receive either a low or high dose of AMT-191.
The study is recruiting participants at the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax. Patients will continue their usual enzyme replacement therapy until the criteria for withdrawal are reached, and will be followed for two years.
The trial will evaluate whether AMT-191 is safe and well tolerated. It also will look for proof of concept by measuring early signs that the gene therapy is working. To do this, researchers will measure the levels of the alpha-Gal A enzyme in the body.
About the Author
