Last updated: February 27, 2026
What is the drug associated with NDC 70677-1101?
NDC 70677-1101 corresponds to Ravicti (glycerol phenylbutyrate). It is a nitrogen-scavenging agent used to reduce ammonia levels in patients with urea cycle disorders (UCD). The drug was approved by the FDA in December 2013.
How large is the market for Ravicti?
The primary market consists of rare disease patients with Urea Cycle Disorders. The global UCD treatment landscape is limited, with approximately 500 to 1,000 diagnosed cases in the U.S. and similar numbers worldwide. The market is highly specialized with limited competition given the rare disease indication.
Market size estimates:
| Component |
Data/Estimate |
| Urea Cycle Disorder prevalence |
1 in 35,000 live births in the U.S. (~1:31,000) |
| U.S. diagnosed patients |
~600 to 1,000 |
| Global potential patients |
2,500 to 3,000 |
| Annual per-patient treatment cost |
$200,000 - $250,000 |
Market penetration:
Ravicti's adoption has been slow compared to older treatments like sodium phenylbutyrate. Only a subset of diagnosed patients receive medication due to clinical variability and access barriers.
What are current pricing trends?
Ravicti's list price varies based on formulations, but typical retail prices are approximately $250,000 annually per patient. Several factors influence the final pricing:
- Pricing policies: Manufacturer list prices, discounted payer contracts, and patient assistance programs influence actual net prices.
- Market competition: Sodium phenylbutyrate had been the standard before Ravicti; generics and other therapies influence pricing dynamics.
- Reimbursement policies: Variability exists across commercial insurance, Medicaid, and VA.
Price comparison:
| Product |
List Price (Annual) |
Market Position |
| Ravicti |
~$250,000 |
FDA-approved, branded |
| Sodium phenylbutyrate (BUPHENYL) |
~$150,000 |
Older branded, generic variants |
| Urea Cycle Disorder drugs (off-label) |
Variable |
Less standardized |
How do recent policy and patent trends affect the market?
Ravicti's exclusivity extended until generics are introduced. The patent landscape for glycerol phenylbutyrate had seen multiple filings but no widespread generic availability as of 2023.
- The original composition patent expired in 2022, with some formulation patents extending to 2030.
- Patent challenges and generic submissions could pressure prices downward in the next 3-5 years.
What are the future projections for Ravicti?
Pricing expectations depend heavily on regulatory and patent developments and market adoption rates.
2023-2027 projections:
| Year |
Expected Average Price |
Key Factors |
| 2023 |
~$240,000 |
Stabilized market, limited generic competition |
| 2024 |
~$230,000 |
Entry of generics may begin, payer negotiations intensify |
| 2025 |
~$210,000 |
Increasing generics impact, volume-based discounts |
| 2026 |
~$190,000 |
Further generic competition, new therapies or pipeline entrants |
| 2027 |
~$180,000 |
Market saturation, potential biosimilar availability |
Key drivers:
- Patent expiry and generic competition.
- New therapies for UCD.
- Cost containment pressures from payers and healthcare systems.
- Expanded diagnosis and adoption in early disease stages.
What competitive threats are emerging?
- New therapies: Biologics or gene therapy approaches targeting UCD could displace Ravicti.
- Orphan drug regulations: Incentives remain, but prices are expected to trend downward with increased competition.
- Market penetration of generic glycerol phenylbutyrate formulations.
Summary of strategic implications
- Market is limited but maintains high prices due to the rare disease status.
- Price erosion is inevitable as generics enter the market, but timing remains uncertain.
- R&D efforts in gene therapy could redefine the therapeutic landscape, impacting future pricing.
Key Takeaways
- Ravicti's market is characterized by high treatment costs, limited patient numbers, and slow adoption.
- Pricing is expected to decline from approximately $250,000 to under $200,000 within five years.
- Patent expiry and potential generic entry by 2024-2025 will significantly impact market dynamics.
- Competition from emerging therapies could further reduce prices and market share.
- The rare disease designation sustains high prices, but market forces are pushing toward affordability and new solutions.
FAQs
1. When will generic versions of glycerol phenylbutyrate likely enter the market?
Generic approvals could occur as early as 2024, following patent expiration, but this depends on patent challenges and regulatory approvals.
2. How does Ravicti compare to older therapies?
Ravicti offers a more palatable formulation without sodium, leading to better tolerability, but at a higher cost.
3. What factors influence reimbursement decisions for Ravicti?
Reimbursement depends on clinical necessity, negotiated prices, coverage policies, and the availability of alternatives.
4. Could gene therapy replace Ravicti entirely?
Potentially, but gene therapy for UCD remains investigational with uncertain timelines for approval and clinical adoption.
5. What is the outlook for pricing in the next five years?
Prices are expected to decline as generics and biosimilars enter, with estimates suggesting a 25-40% reduction by 2027.
References
[1] Food and Drug Administration (FDA). (2013). Ravicti (glycerol phenylbutyrate) approval notice.
[2] IQVIA. (2023). Pharmaceutical market analysis.
[3] EvaluatePharma. (2023). Orphan drug pipeline report.
[4] U.S. Census Bureau. (2021). Urea cycle disorder prevalence data.
[5] Statista. (2022). Pricing benchmarks for rare disease drugs.
