Drug intelligence / Profile preview

AV001

Development stage
Approved
Lead developer
Avadel Pharmaceuticals
Modality
Recombinant Proteins and Enzymes, Small Molecules, Cell Therapies
Administration
Intravenous, Oral
01

Overview

AV001 is a code name associated with multiple distinct therapeutic products, primarily from **Avadel Pharmaceuticals** and **Airway Therapeutics**. In the context of Avadel, AV001 refers to **Nouress**, a cysteine hydrochloride injection approved by the FDA in 2019 as an additive to total parenteral nutrition (TPN) for neonatal patients to meet sulfur-containing amino acid requirements. Some documentation also links the AV001 designation to Avadel's **Lumryz** (FT218), an extended-release, once-nightly oral formulation of sodium oxybate approved for treating cataplexy or excessive daytime sleepiness in patients with narcolepsy. Additionally, **Airway Therapeutics** uses the code AV001 (also known as **AT-100**) for a recombinant human surfactant protein D (rhSP-D) being evaluated in clinical trials for the prevention of bronchopulmonary dysplasia (BPD) in very preterm infants and for the treatment of severe respiratory viral infections. The drug's mechanism of action varies by product: cysteine hydrochloride serves as a nutritional supplement; sodium oxybate acts as a GABAB receptor agonist; and rhSP-D functions as an immunomodulatory lung surfactant protein.

Brand names
NouressLumryz
Other names
cysteine hydrochloridesodium oxybaterecombinant human surfactant protein DrhSP-D

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