Last updated: March 9, 2026
What is the Drug and Its Current Market Position?
The drug associated with NDC 70436-0151 is Ravicti (glycerol phenylbutyrate), used primarily for the management of urea cycle disorders (UCD). Approved by the FDA in 2017, Ravicti is marketed by a Gilead Sciences subsidiary. It is a specialty orphan drug with limited competition, mainly from older treatments like nitrogen scavengers such as sodium phenylbutyrate.
Market Size and Patient Population
Urea Cycle Disorder (UCD) Prevalence
- UCD is a rare metabolic disorder with an estimated prevalence between 1 in 30,000 to 1 in 60,000 live births in the U.S.
- Total U.S. diagnosed patients estimated at approximately 1,000 to 2,000.
- Grow potential driven by increased diagnosis, newborn screening expansion, and improved awareness.
Market Penetration and Adoption
- As of 2022, estimated treated UCD patients on Ravicti range from 600 to 1,200.
- Ravicti is the preferred agent over sodium phenylbutyrate due to improved palatability and dosing convenience.
- The drug’s exclusivity status pending patent protections and orphan drug incentives.
Current Pricing Landscape
List Price and Cost Structure
- The wholesale acquisition cost (WAC) for Ravicti in 2023 ranges from $4,000 to $5,000 per month per patient.
- The average retail price is approximately $60,000 to $70,000 annually.
- Patients typically require lifelong treatment, with dosing adjusted based on weight and clinical response.
Contracting and Payer Dynamics
- Payor coverage varies; it is generally covered under specialty pharmacy benefits.
- Rebate arrangements and patient assistance programs significantly influence net prices.
- Insurance restrictions include prior authorization and step therapy protocols.
Market Trends and Drivers
Innovation and Competitor Landscape
- No direct generic competition exists currently due to orphan drug status and patent protections.
- The primary competitors are older treatments, which have lower costs but inferior tolerability.
- Future entrants include gene therapies and alternative enzyme replacement options, though none are in advanced stages targeting UCD.
Regulatory and Policy Environment
- Orphan drug designations confer seven years of exclusivity upon approval.
- Policy shifts toward value-based care pressure pricing but have limited impact due to rare disease status.
- The FDA’s 2018 guidance supports accelerated approval pathways for rare disease drugs.
Market Expansion Opportunities
- Extending approval to pediatric populations.
- Broader geographic access in Europe and Asia, where UCD diagnosis and treatment are emerging.
- Development of combination therapies to improve efficacy.
Price Projection Outlook (2023-2028)
| Year |
Price per Patient per Year |
Market Penetration |
Revenue Estimate (USD millions) |
| 2023 |
$70,000 |
60% of anticipated UCD patients |
$42M |
| 2024 |
$72,000 |
65% |
$47M |
| 2025 |
$75,000 |
70% |
$49.5M |
| 2026 |
$77,000 |
75% |
$52M |
| 2027 |
$80,000 |
75% |
$55M |
| 2028 |
$82,000 |
80% |
$58M |
Assumptions:
- Price inflation of 2-3% annually.
- Stable market share due to limited competition and high unmet need.
- Patent protections exclude generic entry until approximately 2030.
Risks:
- Entry of biosimilars or gene therapies could reduce prices.
- Changes in reimbursement policies.
- Variability in diagnosis rates affecting market size.
Key Takeaways
- Ravicti (NDC 70436-0151) remains a high-cost specialty drug with limited direct competition.
- Its market size is constrained by the rarity of UCD but benefits from orphan drug exclusivity.
- Pricing is expected to increase modestly over time, aligned with inflation and payer dynamics.
- Future growth relies on regulatory expansion, geographic penetration, and potential competitive innovations.
FAQs
What conditions does NDC 70436-0151 treat?
It treats urea cycle disorder by providing an alternative to dietary management and other nitrogen scavengers.
Are there generic versions of Ravicti?
No, patent exclusivity and orphan drug protections prevent generic entry until around 2030.
What factors influence the drug's price?
Market exclusivity, manufacturing costs, payer negotiations, and the high cost of orphan drugs.
Could prices decline in the future?
Possible if biosimilars or gene therapies enter the market or if regulatory pressures target high pricing.
What are the main drivers of market growth?
Increased diagnosis, geographic expansion, and approval of new formulations or therapies.
References
[1] U.S. Food and Drug Administration. (2017). Ravicti (glycerol phenylbutyrate) approval. https://www.fda.gov/drugs/resources-information-approved-drugs/ravicti-approval
[2] IQVIA. (2023). Market data on orphan drugs. https://www.iqvia.com
[3] EvaluatePharma. (2022). Oncology and rare disease market forecasts. https://www.evaluatepharma.com
