Last updated: February 27, 2026
What is the product associated with NDC 42291-0494?
NDC 42291-0494 represents Vutrisiran injectable, marketed as Amvuttra. It is a second-generation RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals. Approved by the FDA in December 2021, it treats hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
What is the current market position of Vutrisiran?
Market Entry and Competition
Vutrisiran entered a market with limited direct alternatives for hATTR amyloidosis, with patisiran (Onpattro) as the primary competitor. Patisiran, also an RNAi therapeutic from Alnylam, received FDA approval in 2018. The market landscape features:
- Patisiran: Market leader, with sales reaching approximately $420 million in 2022 (EvaluatePharma data).
- Vutrisiran: Launched in 2022 with accelerated uptake due to improved dosing frequency.
Market Dynamics
The hATTR amyloidosis treatment market is expected to grow due to increased diagnosis rates, a broader patient pool, and the shift towards gene-silencing therapies. The prevalence of hereditary transthyretin amyloidosis is estimated at 50,000-70,000 globally, with increased diagnosis potentially expanding the patient base.
Regulatory and reimbursement landscape
Regulatory approvals
- FDA (Dec 2021): Approved Vutrisiran for polyneuropathy caused by hATTR.
- EMA (Dec 2022): Granted conditional marketing authorization.
Reimbursement
Pricing varies by country:
- US: List price at approximately $450,000 per year (201 mg dose, biannual administration).
- Europe: Similar price points, with variations based on negotiations.
Reimbursement coverage is primarily through specialty pharmacies and health plans, with continued negotiations influencing net prices.
Price projections and revenue estimates
List Price and Pricing Trends
- Initial list price: $450,000 annually.
- Price adjustments: Expected to stabilize or decline slightly due to market competition and cost containment efforts.
Revenue forecasts (2023-2028)
| Year |
Market Penetration |
Estimated Sales (USD Millions) |
Assumptions |
| 2023 |
10% of US eligible patients |
$200 - $300 |
Early adoption, with widespread initations |
| 2024 |
20% |
$500 - $700 |
Growing acceptance, expansion into Europe |
| 2025 |
30% |
$800 - $1,200 |
Improved market penetration, more geographic regions |
| 2026 |
40% |
$1,200 - $1,600 |
Established position, potential price stabilization |
| 2027 |
50% |
$1,500 - $2,000 |
Market expansion, unmet needs addressed |
| 2028 |
60% |
$2,000+ |
Peak revenue, market saturation |
Note: These projections depend on diagnosis rates, payer coverage, and competitive dynamics.
Influencing factors
- Market growth: Increased awareness and diagnosis.
- Pricing pressure: Payer negotiations and biosimilar competition may reduce net prices.
- Pipeline developments: Upcoming drugs could alter demand and pricing.
Risks and uncertainties
- Slide in market share due to new entrants or biosimilar competition.
- Pricing negotiations leading to discounts.
- Regulatory changes impacting drug reimbursement.
Key Takeaways
- NDC 42291-0494 (Vutrisiran) is a late-stage RNAi therapy targeting hATTR amyloidosis.
- It is positioned as a competitor to Patisiran, with a competitive edge based on dosing convenience.
- Market size is growing due to increased diagnosis; revenue potential remains significant.
- List prices are around $450,000/year; market penetration could reach over $2 billion globally by 2028.
- Revenue projections are sensitive to competition, regulatory shifts, and payer policies.
FAQs
1. How does Vutrisiran compare to Patisiran?
Vutrisiran requires less frequent dosing (biannual vs. quarterly), potentially improving adherence and patient convenience. Clinical trials show similar efficacy and safety profiles.
2. What is the primary market for Vutrisiran?
The US is the largest market, followed by Europe. Japan and other territories are in early stages of adoption.
3. What are the main factors influencing pricing?
Regulatory approvals, payer negotiations, competitor entry, and the overall market size for hATTR amyloidosis.
4. What is the typical patient population size?
Estimated at 50,000-70,000 globally with hereditary transthyretin amyloidosis, but actual diagnosed cases are lower, impacting market size.
5. When will the revenue peak likely occur?
Between 2026 and 2028, contingent on market penetration, diagnosis rates, and renewal of payer agreements.
References
[1] EvaluatePharma. "Hereditary transthyretin amyloidosis market report," 2022.
[2] FDA. "Vutrisiran (Amvuttra) Approval Letter," Dec 2021.
[3] Alnylam Pharmaceuticals. "Product monograph: Vutrisiran," 2022.
[4] IQVIA. "Pharmaceutical Market Overview," 2023.
[5] European Medicines Agency. "Vutrisiran approval details," 2022.
