Last updated: February 27, 2026
What is the approved indication for NDC 45802-0087?
The drug with NDC 45802-0087 is Givlaari (givosiran), used for the treatment of acute hepatic porphyria (AHP).
What is the current market landscape?
Market Size and Patient Population
- Prevalence of AHP: Approximately 1,000 to 3,000 identified cases in the U.S. (May 2021, FDA), with underdiagnosis potentially increasing this number.
- Target Population: Patients with recurrent attacks requiring prophylactic therapy. Estimates suggest about 20-25% of diagnosed patients receive Givlaari.
Competitive Environment
- Key competitors: Panhematin (hemin) and other supportive therapies, but no direct approved alternatives exist for prophylaxis.
- Market exclusivity: Givlaari received FDA approval in November 2019, with patent protections extending into the late 2020s.
- Pricing: Initial list price at launch was approximately $6,900 per vial, with treatment courses costing around $335,000 annually per patient (Wholesaler Price).
What are the current sales and usage trends?
- Sales data (2022): U.S. sales exceeded $250 million, with year-over-year growth rates near 40%.
- Market penetration: Approximately 300 to 400 patients use Givlaari, with increases expected as awareness rises.
What are price projections based on?
Influencing factors:
- Patent expirations: No immediate threats; exclusivity extends into the mid-2020s.
- Pricing policies: Payers negotiate discounts; list prices are higher than net prices.
- New indications: The expansion to pediatric populations or additional rare porphyrias could expand market size.
- Market access dynamics: Payer restrictions and prior authorization impact uptake.
Price trends:
| Year |
Estimated Average Price |
Notes |
| 2022 |
$6,900 per vial |
List price at launch, subject to discounts. |
| 2023 |
$6,900 - $7,000 per vial |
Slight price increase expected due to inflation and inflation-adjusted contracts. |
| 2024 |
$7,000 - $7,200 per vial |
Payer pressure may moderate list prices; net prices vary. |
| 2025 |
$7,200 - $7,500 per vial |
Potential stabilization or slight increase as market matures. |
| 2026+ |
Stable or declining slightly |
Patent protection scheduled to end mid-2020s, potential biosimilar competition unlikely due to niche orphan status. |
Price reduction possibilities:
- Discounts negotiated with payers can lower net prices by 10-20%.
- Manufacturer may implement patient assistance programs affecting out-of-pocket costs.
What are key takeaways?
- Givlaari remains the only FDA-approved therapy for AHP with a growing patient base.
- Sales growth will depend on increased diagnosis, expanded indications, and market acceptance.
- List prices could rise minimally into 2024, then stabilize or decrease slightly due to payer negotiations.
- No imminent biosimilar or generic competition is expected before late 2020s.
- Price sensitivity among payers may lead to sustained discounts impacting net revenue.
FAQs
1. Will Givlaari's price increase significantly in the next few years?
Likely minimal increases—expected to be constrained by payer negotiations and market dynamics.
2. Could biosimilar competition appear soon?
Unlikely before late 2020s due to orphan drug protections and manufacturing complexities.
3. What factors could influence patient uptake?
Increased awareness, better diagnosis, expanded indications, and payor coverage policies.
4. How does Givlaari's pricing compare to similar orphan drugs?
It is in line with other high-cost rare disease therapies, generally ranging from $300,000 to $500,000 annually.
5. What impact would healthcare policy changes have?
Potentially moderate pricing pressures; cost-containment measures could constrain long-term pricing.
References
- FDA. (2021). Givlaari (givosiran) approval. Retrieved from https://www.fda.gov
- IQVIA. (2022). U.S. pharmaceutical sales data.
- Manufacturer pricing and market access reports. (2022).
- Orphan Drug Designations and Patent Data. (2022).
- Peerspeak Market Analysis Reports. (2022).
