Last updated: March 2, 2026
What is the Drug with NDC 13668-0203?
NDC 13668-0203 is a product marketed as Odevixibat. It is a selective ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration (FDA) for treating progressive familial intrahepatic cholestasis (PFIC) in pediatric patients aged 3 months and older.
Current Market Position
Odevixibat (trade name: Bylvay) was approved in July 2021 by the FDA. It is among a limited class of drugs targeting rare cholestatic conditions with high unmet medical needs. No direct generic competition exists as of Q1 2023, positioning Odevixibat as a first-in-class therapy.
Market Size
- Prevalence of PFIC: Estimated at 1-2 per million children globally, with higher rates in regions with consanguineous marriages.
- US Market: Approximate annual incidence in the US is 150-300 cases.
- Global Market Potential: Estimated at 1,000-2,000 patients annually worldwide.
Adoption Factors
- FDA approval: Clear regulatory pathway for use in pediatric PFIC.
- Physician familiarity: Limited due to rarity; initial adoption mainly from tertiary care centers.
- Treatment landscape: No approved pharmacotherapy options other than supportive care; liver transplant remains the primary treatment.
Competitive Landscape
| Drug / Class |
Regulatory Status |
Indications |
Market Share |
Notes |
| Odevixibat (Bylvay) |
FDA-approved |
PFIC |
Nearly 100% (initial phase) |
First drug with specific approval for PFIC |
| Ursodeoxycholic Acid |
Off-label |
Cholestatic diseases |
Widely used |
No specific approval for PFIC |
| Liver transplant |
N/A |
Final treatment |
N/A |
Last-resort option |
No other pharmacologic treatments have received FDA approval for PFIC. Off-label use remains limited.
Pricing Analysis
Current Pricing Data
- Odevixibat (As per US wholesale acquisition cost (WAC), 2023): Approximately $300,000 - $400,000 annually per patient.
- Pricing Factors:
- Dosage is weight-dependent, typically starting at 1.0 mg/kg/day.
- Treatment duration is lifelong unless liver transplantation occurs.
- No generic or biosimilar options are available.
Cost Drivers
- Drug Manufacturing: High, due to small patient population and complex synthesis.
- Market exclusivity: Maintains high price levels under patent protections; patent expiry not expected before 2035.
- Approval status: Sole approved pharmacotherapy for PFIC promotes high pricing power.
Price Trends and Projections
| Year |
Estimated Price Range |
Factors Influencing Price |
| 2023 |
$300k - $400k |
Limited competition, rare disease pricing |
| 2025 |
$300k - $400k |
No biosimilar entrants, stable demand |
| 2030 |
$250k - $350k |
Potential entry of biosimilars or generics post-patent expiry |
Prices are unlikely to decline significantly before patent expiration, barring regulatory or patent challenges. Market access barriers and payer negotiations could influence actual net prices, potentially lowering effective costs by 10-20% in managed care settings.
Future Market Opportunities
- Expansion to other pediatric cholestatic diseases: Unlikely in the short term due to regulatory restrictions.
- Extended indications: Use in adults or other hepatic conditions would require additional trials.
- Pricing elasticity: Limited; high unmet need sustains premium pricing.
Summary
- NDC 13668-0203 (Odevixibat) commands premium pricing due to being the first FDA-approved treatment for PFIC.
- Market size remains small but high-value, with annual revenue potential in the range of hundreds of millions globally.
- Price projections indicate stability through 2025, with possible decline post-patent expiry around 2035.
- Competition is minimal; biosimilar or generic invasion anticipated after patent expiry.
Key Takeaways
- Odevixibat is a niche, high-price hepatology drug with limited but unmet demand.
- Market entry barriers are high; prices are expected to remain elevated until generic competition emerges.
- The global market potential remains constrained by disease rarity, but high per-patient revenues justify continued investment.
- Regulatory and reimbursement pathways heavily influence market penetration and price realization.
FAQs
1. When is patent expiry for Odevixibat expected?
Patent protection is anticipated to last until at least 2035, barring legal challenges. It ensures market exclusivity during this period.
2. Are there any approved biosimilars or generics for NDC 13668-0203?
No, as of Q1 2023, no biosimilar or generic has entered the market.
3. What factors could lead to price reductions before patent expiration?
Reimbursement negotiations, formulary inclusions, and new competing treatments could pressure prices downward.
4. Is the market for PFIC treatment expected to grow?
The disease's rarity limits growth, but increasing awareness and potential expansion of indications could marginally expand the market.
5. How does the annual treatment cost compare with other rare disease drugs?
Odevixibat’s cost aligns with other orphan drugs, which often range from $200,000 to over $500,000 annually depending on the indication.
References
[1] U.S. Food and Drug Administration. (2021). FDA approves Bylvay for pediatric patients with progressive familial intrahepatic cholestasis [Press release].
[2] IQVIA. (2023). United States Pharmaceutical Pricing Data.
[3] Global Liver Institute. (2022). PFIC epidemiology and treatment landscape.
[4] Milken Institute. (2021). Rare Disease Drug Pricing Analysis.
