What is the name of the drug and what does it do?
Aqneursa (pronounced ak-NUHR-sah), generic name levacetylleucine approved on September 25, 2024, is a medicine used to treat neurological symptoms of Niemann-Pick disease type C (NPC). NPC is a rare genetic disorder that affects the body’s ability to process cholesterol and fats, leading to severe brain and nerve damage. Aqneursa is approved for use in adults and children who weigh at least 15 kilograms (33 pounds).
How does it work?
Aqneursa’s exact mechanism isn’t fully understood, but it is thought to help improve nerve function and stabilize neurological symptoms caused by NPC. By supporting the nervous system, it helps reduce the progression of disease-related symptoms like difficulty walking, swallowing, and speaking.
What did the research discover?
In a clinical trial involving 60 patients with NPC, Aqneursa was shown to improve neurological symptoms when compared to a placebo. The study measured changes using a functional test called the fSARA, which evaluates movement, speech, and balance. Patients treated with Aqneursa had better scores than those on a placebo, showing slowed progression of symptoms. The study also confirmed that Aqneursa works well in both children and adults, with most patients tolerating the medication well.
What are some of the side effects?
The most common side effects of Aqneursa include:
- Abdominal pain
- Difficulty swallowing (dysphagia)
- Upper respiratory infections
- Vomiting
Patients should report any unusual symptoms to their doctor, especially if they experience severe allergic reactions or other complications.
What are the dosage recommendations and how is it prescribed?
Aqneursa is an oral suspension taken up to three times daily, depending on body weight:
- 15–25 kg: 1 gram in the morning and 1 gram in the evening
- 25–35 kg: 1 gram in the morning, 1 gram in the afternoon, and 1 gram in the evening
- Over 35 kg: 2 grams in the morning, 1 gram in the afternoon, and 1 gram in the evening
To take Aqneursa, patients mix the contents of a packet with water, orange juice, or almond milk. It should be consumed immediately after preparation. For those who have a feeding tube, the medicine can also be administered through the tube using water.
Source:
U.S. Food and Drug Administration. Full prescribing information: [Label for NDA 219132]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/219132s000lbl.pdf. Accessed 2024 Nov 21.
