Last updated: February 17, 2026
Overview
The drug associated with NDC 68462-0536 is Ravicti (glycerol phenylbutyrate), approved for the management of urea cycle disorders (UCD). It is marketed primarily as a specialty medication with a niche target patient population.
Market Size
The Urea Cycle Disorder (UCD) market is limited but growing. Estimated prevalence is approximately 1 in 35,000 live births, translating to about 2,700 diagnosed patients in the U.S.[1]. The treatment landscape includes Ravicti, Buphenyl (sodium phenylbutyrate), and off-label options, with Ravicti being the preferred choice due to dosing convenience.
Market Adoption & Competitors
Ravicti's market share is primarily influenced by:
- Physicians' familiarity with Ravicti versus Buphenyl.
- Insurance coverage and reimbursement policies favoring Ravicti.
- Patient adherence preferences, given Ravicti's liquid formulation.
The patent expiration or exclusivity period critically impacts market dynamics. Ravicti received FDA approval in 2013, with patent exclusivity extending uncertainly until at least 2030, barring generic entry.[2]
Price Point and Revenue
The average wholesale price (AWP) for Ravicti has historically been around $35,000 per year per patient. Adjusting for discounts, insurers typically pay $25,000–$30,000 annually.
Positioning assumptions:
- Market Penetration: Achieves 40% of diagnosed patients within the next 5 years.
- Patient Count: 1,000 active U.S. patients using Ravicti directly.
- Average Treatment Duration: Lifelong or long-term, averaging 20 years per patient.
Estimated annual revenue:
| Parameter |
Value |
| Number of patients |
1,000 |
| Annual cost per patient |
$30,000 |
| Total annual revenue |
$30 million |
Price Projections
- Short-term (1-3 years): Stable pricing at current levels, supported by limited competition.
- Mid-term (4-7 years): Possible price erosion (~10-15%) upon generic entry or biosimilar development.
- Long-term (8+ years): Prices could stabilize or decline further as biosimilar options appear, with estimates around $20,000-$25,000 per patient annually.
Price declines are contingent on regulatory timelines, market share shifts, and insurance reimbursement policies.
Regulatory and Intellectual Property Outlook
- Patent protections have been challenged, but current patents likely extend to at least 2030.[2]
- No biosimilar or generic versions are currently approved; however, patent litigations could change this.
Market Risks
- Entry of generics could reduce prices substantially.
- Changes in reimbursement policies for rare disease treatments.
- Introduction of alternative therapies or gene therapies.
Key Considerations for Stakeholders
Investors and pharma companies should watch patent expirations, insurance coverage policies, and developments in gene therapy for UCD, which could replace enzyme substitution therapies altogether.[3]
Summary
Ravicti remains a high-price niche drug for UCD management, with steady revenues supported by limited competition. Price projections suggest modest declines upon patent expiration, with long-term market size constrained by disease prevalence and treatment adherence.
Key Takeaways
- The UCD market is small but stable, with Ravicti’s high price supported by limited competition.
- Current annual revenue estimates stand at approximately $30 million in the U.S.
- Price decline is expected in 8-10 years, influenced by patent expiry and generics.
- Market expansion depends on diagnosis rates and insurance coverage improvements.
- Biosimilar entry could be a significant disruptor in long-term pricing.
FAQs
-
How soon could generic Ravicti enter the market?
Patent challenges may lead to generic entry around 2030, barring successful litigation.
-
What are the major competitors to Ravicti?
Sodium phenylbutyrate (Buphenyl) is the primary alternative, often less expensive but less convenient.
-
Could new therapies replace Ravicti?
Yes, gene therapy and enzyme replacement approaches are in early development stages and could disrupt the market.
-
How does insurance impact Ravicti pricing?
Insurance coverage often results in negotiated discounts from the wholesale price, reducing out-of-pocket costs for patients.
-
Is market growth expected beyond the current patient population?
Slight growth may occur due to increased diagnosis rates, but overall size remains limited by disease prevalence.
Sources
- National Organization for Rare Disorders (NORD). Urea cycle disorder overview.
- FDA database and patent filings.
- Recent clinical trials and biotech pipelines for UCD.
