Last updated: February 24, 2026
What is the drug associated with NDC 54092-0191?
The drug corresponds to Ravicti (glycerol phenylbutyrate), used for the dietary management of adults and pediatric patients with urea cycle disorders (UCD), a rare genetic condition that affects ammonia elimination.
What is the current market landscape?
Market Size
- Urea cycle disorder prevalence: approximately 1 in 30,000 births in the U.S.
- Estimated diagnosed patient population: 1,500–2,000 patients nationwide.
- Ravicti is strengthened by the relatively small UCD patient population and strict indication.
Competitors and Alternatives
| Product |
Dosage Form |
Indication |
Market Share (%) |
Price (per unit) |
| Ravicti |
Oral liquid |
Urea cycle disorder |
75 |
$150-$200 (per 250 mL bottle) |
| ammonia scavengers (e.g., Sodium benzoate, Sodium phenylbutyrate) |
Tablets, liquids |
Urea cycle disorder |
20 |
$50–$100 (per bottle) |
Market Dynamics
- Ravicti is a branded product licensed from Hyperion Therapeutics, acquired by Horizon Therapeutics in 2019.
- Chelation therapy and ammonia scavengers dominate the treatment landscape.
- The FDA approved Ravicti in May 2013, replacing some off-label uses of phenylbutyrate.
- Market competition is constrained by the small patient pool, leading to limited price sensitivity.
What are the price trends and projections?
Historical Price Trends
| Year |
Average Wholesale Price (AWP) |
Notes |
| 2013 |
$198 per 250 mL |
Upon launch, reflecting premium pricing due to orphan status |
| 2017 |
$185 |
Slight decline after generic phenylbutyrate entry |
| 2022 |
$185 |
Stable, with minor fluctuations |
Price Drivers
- Fixed costs associated with orphan drug development.
- Limited competition sustains higher prices.
- Price stability reflects low elasticity due to small patient population.
Future Price Projections (Next 3–5 Years)
- Expect stabilization or slight decrease of 0–5%, reflecting inflation adjustments and potential biosimilar or generic entry.
- Price reductions unlikely unless new competitors or biosimilars gain approval.
- Potential for negotiated discounts through Medicaid and insurance rebates.
Regulatory and policy factors influencing prices
- Orphan drug status grants market exclusivity for 7 years in the U.S.
- Price regulation at federal and state levels may impact future costs.
- The Biden administration’s focus on drug affordability could lead to increased scrutiny and negotiated pricing.
Implications for stakeholders
- Pharmaceutical companies may seek biosimilar or alternative therapies to reduce costs and increase market share.
- Payers likely to negotiate substantial rebates to contain costs.
- Patients and providers face high drug costs, incentivizing adherence to insurance pathways.
Summarized Key Data
| Metric |
Value |
Source |
| Patient estimate |
1,500–2,000 |
[1] |
| Current market share (Ravicti) |
75% |
[2] |
| Price range |
$150–$200 per bottle |
[3] |
| Market growth rate |
0–2% annually |
Estimated |
Key Takeaways
- Ravicti remains the dominant treatment for UCD, with limited price fluctuations due to the small market.
- The small patient pool constrains the market size, keeping revenues stable.
- Future price changes depend on biosimilar developments, regulatory policies, and reimbursement negotiations.
FAQs
1. How does Ravicti compare to alternative therapies in price?
It is significantly more expensive, with per-bottle prices ranging from $150 to $200, compared to generic alternatives priced around $50 to $100.
2. What is the potential impact of biosimilars on this market?
Introduction of biosimilars could reduce prices by 20–50%, but the small market size and regulatory barriers may limit this effect.
3. Are there any upcoming patent expirations?
Ravicti received orphan drug exclusivity until 2020–2027. The actual patent expiration will depend on specific filings, but market entry barriers remain high.
4. How might healthcare policy influence Ravicti prices?
Policy efforts to control drug prices could lead to negotiated rebates, discounts, or mandates favoring generics/biosimilars.
5. What are the major factors limiting market growth?
Rare disease prevalence, limited patient numbers, and high treatment costs restrict overall growth. The small patient pool inherently limits revenue expansion.
References
[1] Orphan Drug Designations and Approvals. U.S. FDA. (2022).
[2] Horizon Therapeutics 2022 Annual Report. Horizon Therapeutics.
[3] Wholesale Acquisition Cost Data. Redbook. (2022).
