IntraBio Inc

Niemann-Pick Disease Awareness Month reminds us that while rare diseases may affect only a few individuals, together their voices create a powerful impact.

The INPDA International Niemann-Pick Disease Alliance and its member organizations play a vital role in amplifying those voices, uniting the community, and driving forward awareness.

At IntraBio, we are proud to stand alongside these organizations in raising awareness of Niemann-Pick disease and highlighting the importance of collaboration to improve the lives of those affected.

Today we celebrate one year since the FDA approval of AQNEURSA® (levacetylleucine) for Niemann-Pick disease Type C (NPC) on September 24, 2024.

This milestone marked an important step forward for patients and families living with NPC. Over the past year, we have been energized by the strong engagement from healthcare providers and the dedication of the NPC advocacy community. Most importantly, we are inspired by the voices of patients, families, and advocates who remind us what this community means.

We are proud to celebrate this first year together and remain committed to supporting patient access and advancing care for everyone affected by NPC in the US.

INDICATION
AQNEURSA® (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

IMPORTANT SAFETY INFORMATION
Do not take AQNEURSA if you:
· have not had a negative pregnancy test. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments
Before taking AQNEURSA, discuss with your doctor if you:
· are pregnant or planning to become pregnant. Based on findings from animal reproduction studies, AQNEURSA may cause harm to your unborn baby when administered during pregnancy. If you become pregnant while taking AQNEURSA, inform your doctor immediately to discuss potential risks and alternative treatments.
· require the need for effective contraception during treatment with AQNEURSA and for 7 days after stopping treatment to avoid pregnancy.
· are breastfeeding or plan to breastfeed. It is not known if AQNEURSA or its metabolites pass into human or animal milk or effects on breastmilk production
· are taking P-gp substrate. Your doctor will monitor you for related adverse reactions
· have any other medical conditions
· are taking any medications and supplements, including N-acetyl-DL-leucine or N-acetyl-D-leucine, as some of these may impact the effectiveness of AQNEURSA
The most common side effects of AQNEURSA include abdominal pain, dysphagia, upper respiratory tract infections and vomiting. Speak with your doctor if these side effects persist or worsen.

Take AQNEURSA exactly as prescribed by your doctor.

If a dose of AQNEURSA is missed, skip the missed dose and take the next dose at the scheduled time. Do not take 2 doses at the same time to make up for a missed dose.

AQNEURSA can be taken with or without food.

You are encouraged to report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please click here for Full Prescribing Information for AQNEURSA: https://lnkd.in/eZaW2t2R