Last updated: February 17, 2026
What is NDC 62135-0225?
NDC 62135-0225 represents Ravicti (glycerol phenylbutyrate), approved by the FDA for the management of urea cycle disorder (UCD), a rare genetic condition affecting ammonia detoxification. Introduced in 2013 by Valent BioSciences, Ravicti replaced sodium phenylbutyrate (NaPB) in some indications due to improved tolerability and dosing convenience.
Market Size and Patient Population
The Urea Cycle Disorder (UCD) market is categorized as a rare disease space with limited but stable demand:
- Estimated prevalence: 1 in 30,000 births in the U.S.
- U.S. diagnosed UCD patients: approximately 500-1,200 individuals (per ORPHA and NIH data).
- Treatment indication: Chronic therapy to prevent hyperammonemia episodes.
Key factors influencing demand:
- Growing awareness due to improved diagnostics.
- Increased diagnosis rates with newborn screening programs.
- Off-label use for other hyperammonemia-related conditions.
Competitive Landscape
Main competitors:
| Drug |
Type |
Approval Year |
Market Share (Estimated) |
Price (Estimated) |
| Ravicti |
Glycerol phenylbutyrate |
2013 |
Majority of niche market |
$300,000 per year |
| NaPB (Buphenyl) |
Sodium phenylbutyrate |
1996 |
Declining, due to tolerability issues |
$250,000 per year |
| Ammonul |
Ammonia-detoxifying agent |
1987 |
Less used, acute settings |
Varies, ~$50,000 per treatment |
Market share dynamics: Ravicti has gained market share over NaPB due to better tolerability and dosing flexibility, though cost remains a barrier. The small size of the patient population limits market expansion.
Pricing Trends and Cost Factors
- Ravicti's list price approximates $300,000 annually per patient.
- NaPB, its predecessor, was priced higher initially but has seen price reductions and generic availability.
- Cost is driven by:
- Orphan drug designation benefits.
- Complex manufacturing process.
- Limited patient population leading to high per-unit costs.
Pricing challenges:
- Payor resistance due to high costs.
- Preference for off-label use of cheaper alternatives when possible.
- Biosimilar or generic versions unopened, reducing competition.
Regulatory and Policy Environment
- Orphan Drug Status: Confers seven-year exclusivity in the U.S., through 2020s.
- Pricing and Reimbursement: Depending on PBMs and insurers, reimbursement levels vary. Some payers require prior authorization or step therapy.
- Potential Biosimilar Entry: No biosimilars authorized yet; patent expiration or patent challenges could alter pricing.
Market Trends and Forecasts
Short-term (1-3 years):
- Minimal growth in the existing niche market.
- Price stability driven by regulatory exclusivity.
- Slight shifts toward value-based reimbursement.
Medium to long-term (3-5 years):
- Possible market contraction if generic or biosimilar versions enter.
- Investments in gene therapy and novel treatments for UCD may impact demand.
- Increased diagnostic awareness could expand the patient base marginally.
Price projections:
| Scenario |
Price Estimate (per patient/year) |
Assumptions |
| Status Quo |
~$300,000 |
No biosimilar entry, stable demand |
| Moderate Competition |
~$250,000 |
Entry of biosimilar, price competition |
| Market Contraction |
~$200,000 |
Loss of exclusivity, increased competition |
Forecasts assume no radical price reductions and continued lifecycle management within the orphan drug framework.
Key Takeaways
- Ravicti (NDC 62135-0225) remains the dominant treatment in its niche with stable, high prices.
- The small patient population limits market size, with revenues primarily driven by pricing rather than volume.
- Market dynamics are sensitive to biosimilar entry, regulatory changes, and advancements in alternative therapies.
- Manufacturers should monitor patent statuses and emerging treatments to anticipate future price adjustments.
FAQs
1. What factors could lead to a decline in Ravicti's price?
Introduction of biosimilars or generic glycerol phenylbutyrate could pressure prices downward. Patent expiry and regulatory approvals for alternative treatments also influence pricing.
2. How does the UCD patient population impact market growth?
The small, stable population limits revenue growth; expansion relies on better diagnostics and potential off-label uses.
3. Are there emerging therapies that threaten Ravicti’s market?
Gene therapies and alternative ammonia detoxification strategies are under development but currently lack FDA approval for UCD.
4. What are reimbursement challenges for Ravicti?
High costs lead to prior authorization requirements, and payor resistance could limit access, affecting revenue stability.
5. Will regulatory changes affect the future of Ravicti?
Yes, especially if patent protections are challenged or if new therapies receive approval, impacting exclusivity and pricing.
References
- FDA. (2013). Ravicti Approval Letter.
- ORPHA. (2020). Urea Cycle Disorder Overview.
- IMS Health. (2019). Orphan Drug Market Data.
- NIH. (2021). Urea Cycle Disorder Fact Sheet.
- EvaluatePharma. (2022). Orphan Drug Market Forecasts.
