FDA Approves Aqneursa to Help Improve Muscle Control in Ataxia-Telangiectasia

The FDA approved Aqneursa (levacetylleucine) to treat muscle control problems, known as ataxia, in adults and children who weigh at least 33 pounds (15 kilograms) and have a rare genetic disorder called ataxia-telangiectasia (A-T). A-T is a condition passed down in families that causes a progressive loss of balance, body control, and speech. Before this approval, there were no approved treatments specifically for ataxia in people with A-T. Aqneursa was previously approved to treat nervous system symptoms in another rare disease called Niemann-Pick disease type C. 

Aqneursa is a modified form of an amino acid. The exact way it works in the body to help treat A-T is not fully known. But it helps improve movement, balance, and coordination in people living with this condition. 

Approval of Aqneursa was based on a clinical trial with 73 adults and children ages 4 years and older with A-T. In the trial, patients took either Aqneursa or a placebo (an inactive treatment) for 12 weeks and then switched to the other treatment for another 12 weeks. Researchers used a special rating scale to measure muscle control and coordination, including walking, sitting, balance, and speech. 

When patients were taking Aqneursa, they had better movement and balance scores compared to when they were taking the placebo. This showed a real improvement in daily neurological function. The drug was generally well tolerated. The main safety findings in the trial included falls, skin cuts, and urinary tract infections. 

Aqneursa comes as granules in a packet that are mixed with water, orange juice, almond milk, or yogurt before drinking. It can also be given through a gastrostomy feeding tube (G-tube) using water only. You should not mix the powder with hot liquids. The mixture must be taken right after mixing. 

Before starting Aqneursa, your healthcare provider will check to make sure you are not pregnant. Animal studies show that Aqneursa may harm a fetus. Females who can become pregnant should use effective birth control during treatment and for seven days after taking their last dose. If you are pregnant, plan to become pregnant, or are breastfeeding, talk with your healthcare provider to discuss the risks and benefits. 

The most common side effects reported during clinical trials for A-T were falls, skin cuts, and urinary tract infections. Tell your healthcare provider about all medicines you take. You should avoid taking Aqneursa with certain supplements containing N-acetyl-DL-leucine or N-acetyl-D-leucine, as they can prevent Aqneursa from working properly. Your healthcare provider may also need to monitor you more closely if you take certain other prescription medicines that interact with Aqneursa.