Last updated: February 25, 2026
What is the drug associated with NDC 62542-0101?
NDC 62542-0101 corresponds to Ravicti (glycerol phenylbutyrate), used as an alternative to sodium phenylbutyrate and sodium benzoate for the management of adult and pediatric patients with urea cycle disorders (UCDs). Ravicti received FDA approval in 2013 for this indication.
Market overview
Ravicti's market is confined to rare disease treatment, primarily targeting patients with UCDs. The overall prevalence of UCDs is approximately 1 in 35,000 live births. The drug's market potential depends on diagnosis rates, treatment adoption, and availability of competing therapies.
Market size and patient population
| Parameter |
Data |
| Urea cycle disorder prevalence |
1 per 35,000 live births (approximate) |
| Estimated diagnosed patients |
600–800 in the U.S. |
| Pediatric vs. adult population |
About 70% pediatric; 30% adults |
| Annual treatment-eligible patients |
400–600 in North America |
Estimations derived from Urea Cycle Disorders Foundation data.[1]
Competitive landscape
- Sodium phenylbutyrate (Buphenyl): Older, generic, lower cost but with tolerability issues.
- Ammonul (sodium phenylacetate and sodium benzoate): Used acutely, especially for hyperammonemia episodes.
- Glycerol phenylbutyrate (Ravicti): Approved non-injectable alternative with improved tolerability and convenience.
Pricing analysis
Current pricing
- List price of Ravicti ranges between $209,000 to $250,000 per year (as of 2022).
- Comparatively, Buphenyl costs approximately $80,000–$100,000 per year.[2]
- Ammonul's annual cost is roughly $50,000–$70,000, but used primarily during acute episodes.
Cost drivers
- Cost driven by the drug's formulation, patent exclusivity, and manufacturer pricing strategies.
- Insurance coverage and Medicaid rebates impact net prices.
- Distribution and handling costs are significant given the high-dollar value.
Price projections
| Year |
Projection (USD) |
Basis |
| 2023 |
$210,000–$255,000 |
Current market trends, inflation |
| 2024 |
$220,000–$265,000 |
Patent protections, limited generic entry |
| 2025 |
$225,000–$275,000 |
Anticipated patent exclusivity protection, market stability |
| 2026+ |
Plateau or slight decrease |
Potential generic or biosimilar entry, market saturation |
Influencing factors
- Patent and exclusivity: Ravicti’s patent expired in 2022 in some regions, but extended exclusivities or formulations could delay generic entry.[3]
- Generic competition: Entry of bioequivalent generics expected between 2024–2026, likely reducing prices by 20–40%.
- Regulatory developments: Approval of new formulations or alternative therapies could influence pricing.
Regulatory and policy considerations
- 2017 FDA guidance emphasizes patient access and affordability.
- The 340B drug pricing program affects pricing strategies for eligible hospitals.
- Medicaid rebates and insurer negotiations influence net prices.
Summary
Ravicti maintains a premium pricing position due to its orphan drug status and improved tolerability. Price increases are forecasted into 2024–2025, with potential dips if generic versions gain market share. The narrow patient population constrains volume growth, but increasing diagnosis and treatment rates could stabilize revenues.
Key takeaways
- The market for NDC 62542-0101 primarily involves rare UCD patients, with a small but stable treatment population.
- Current list prices range around $210,000–$255,000 annually.
- Patent expirations and generic entry forecast potential price reductions starting 2024–2026.
- Competition from older therapies and future biosimilar options will influence long-term pricing.
- Market growth depends on increased diagnosis, improved access, and regulatory factors.
FAQs
1. How does patent expiration impact Ravicti’s pricing?
Patent expiration opens the market to generics, typically leading to price decreases of 20–40%. The timing of generic entry depends on patent protections and regulatory approvals.
2. What are the main R&D trends affecting this market?
Development of oral formulations with improved tolerability and biosimilars could challenge Ravicti’s market share, influencing prices and sales.
3. Are there emerging therapies for UCDs?
Gene therapy and enzyme replacement therapies are under investigation, potentially disrupting the current market landscape.
4. How does payer coverage influence actual transaction prices?
Rebates, discounts, and negotiated rates significantly reduce net prices paid by insurers and government programs.
5. What is the global market outlook?
Limited due to the rarity of UCDs; North America and Europe account for most treated patients. Emerging markets may have access limitations impacting volume.
References:
[1] Urea Cycle Disorder Foundation. (2021). Disease overview and prevalence.
[2] Medispan. (2022). Average wholesale prices for Buphenyl and Ravicti.
[3] U.S. FDA. (2022). Patent data for glycerol phenylbutyrate formulations.
