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Developing a new formulation of sodium phenylbutyrate
Nathalie Guffon1, Yves Kibleur, William Copalu
1Lucane Pharma, 172 rue de Charonne, Paris, France.
Archives of Disease in Childhood
|September 4, 2012
Summary
A new taste-masked formulation of sodium phenylbutyrate (NaPB) significantly improves palatability and is bioequivalent to the commercial product. This improved taste masking for urea cycle disorder treatment is expected to enhance patient compliance.
Area of Science:
- Pharmacology
- Drug Delivery
- Gastroenterology
Background:
- Sodium phenylbutyrate (NaPB) is a crucial treatment for urea cycle disorders (UCD).
- The current commercial NaPB formulation suffers from poor taste, negatively impacting patient compliance and treatment efficacy.
- Developing a palatable formulation is essential for managing UCD.
Purpose of the Study:
- To characterize the in vitro taste, dissolution, and bioequivalence of a novel taste-masked NaPB granule formulation (Luc 01).
- To compare the safety and tolerability of Luc 01 against the licensed product in healthy volunteers.
- To assess the impact of taste masking on patient-reported outcomes.
Main Methods:
- In vitro taste assessment and dissolution profiling of Luc 01 and the licensed NaPB granules.
- Pharmacokinetic analysis to determine bioequivalence (Cmax, AUC) in 13 healthy adult volunteers.
- Subjective taste, safety, and tolerability assessments in human participants.
Main Results:
- Luc 01 demonstrated a delayed release of NaPB, avoiding immediate taste receptor stimulation, unlike the commercial product.
- Pharmacokinetic studies confirmed bioequivalence between Luc 01 and the licensed NaPB formulation (CI for Cmax and AUC within 0.80-1.25).
- Participants showed a significant preference for Luc 01, reporting less bitterness and saltiness; the licensed product was associated with vomiting and taste loss.
Conclusions:
- The novel taste-masked granules (Luc 01) effectively mask the bitter taste of NaPB by delaying its release.
- Luc 01 is bioequivalent to the existing licensed formulation, ensuring comparable therapeutic delivery.
- This taste-masked formulation holds significant potential to improve patient compliance and therapeutic outcomes in UCD management.
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