Last updated: February 23, 2026
What is NDC 00781-4004?
NDC 00781-4004 corresponds to a branded drug product marketed under the brand name Rydapt (midostaurin). It is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) positive for FLT3 mutations, and systemic mastocytosis (SM), including aggressive SM and SM with associated hematologic neoplasm. It is an oral kinase inhibitor developed by Novartis.
Current Market Size and Penetration
Indication-specific Market Dynamics
- AML with FLT3 mutations: Represents approximately 30% of AML cases in the U.S. (~10,000 incidence annuals), with increasing recognition of FLT3 mutations as a key therapeutic target.
- Systemic mastocytosis (SM): Rare disease, estimated prevalence of 1 in 10,000, with around 2,000–3,000 diagnosed U.S. patients.
Competitor Landscape
| Product Name |
Indication |
Market Share |
Notes |
| Rydapt (midostaurin) |
AML, SM |
Dominant in niche markets |
Only FDA-approved FLT3 inhibitor for AML and SM |
| Gilteritinib (Xospata) |
AML (FLT3) |
Growing |
Approved for relapsed/refractory AML |
| Midostaurin generics (unapproved) |
Off-label AML, other indications |
N/A |
No official generic, but compounding exists |
Market Penetration
- Rydapt benefits from exclusivity through patent protection, with the patent expiring around 2026 (anticipated, subject to legal challenges).
- Market penetration in AML is high within the target demographic, with prescriptions reaching approximately 4,000 annually in recent years.
- SM indication remains niche; prescriptions estimate around 500–1,000 annually, mostly in specialized centers.
Pricing Analysis
Current Price Points
| Pricing Parameter |
Value |
Notes |
| Wholesale Acquisition Cost (WAC) |
~$19,680 per 30 capsules (~60 mg total) |
Approximate list price for a 30-capsule pack |
| Cost per Day |
~$656 |
Estimated based on typical dosing (100 mg BID) |
| Monthly Cost |
~$19,680 |
Based on a 30-day month |
| Commercial Insurance Reimbursement Range |
~$15,000 – $18,000 per month |
Varies with co-pays, rebates, and payer negotiations |
Price Comparison with Competitors
| Drug Name |
Indication |
Average Monthly Price (USD) |
Notes |
| Rydapt |
AML, SM |
$18,000 – $20,000 |
High due to targeted niche markets |
| Gilteritinib |
AML (relapsed/refractory) |
$24,000 – $27,000 |
Slightly higher, broader indications |
Price Trends & Projections
- Patent expiry date: 2026; expected to potentially see generic entry within 1–2 years post-expiry.
- Price elasticity: Moderate; high cost limits access but key in niche indications where alternatives are limited.
- Reimbursement policies: Impact price stability; payers increasingly demand discounts and rebates for high-cost targeted therapies.
Revenue and Market Outlook
Revenue Estimates (2023–2027)
| Year |
Estimated Global Sales (USD) |
Key Drivers |
| 2023 |
~$350 million |
Established market penetration in AML and SM |
| 2024 |
~$375 million |
Growth in prescriber base, expanded access |
| 2025 |
~$400 million |
Peak sales before patent expiry |
| 2026 |
~$250 million |
Patent expiration, initial generic approvals expected |
| 2027 |
~$150 million |
Market erosion, generics entering slowly |
Impact of Patent and Generics
- Patent protection delays generic competition until 2026.
- After patent expiry, prices are projected to decline by 50–70%, depending on market entry speed and regulatory approvals.
Regulatory and Market Entry Strategies
Key Regulatory Milestones
- FDA approval date: April 2017.
- Pediatric exclusivity: Extended to April 2024.
- Potential biosimilar/generic approval: Anticipated post-2026, contingent on patent challenges and regulatory pathways.
Strategic Considerations
- Focus on expanding indications, e.g., newly emerging molecular targets.
- Engage in payer negotiations early to sustain margins as generic competition approaches.
- Investigate combination therapies to extend patent life and market appeal.
Key Takeaways
- NDC 00781-4004 (Rydapt) is a targeted therapy primarily used for AML with FLT3 mutations and systemic mastocytosis.
- The current U.S. market generates approximately $350 million annually, with potential growth driven by increased diagnosis rates.
- Pricing remains high, around $19,680 per month, influenced by limited competition and the niche nature of indications.
- Patent protection expires around 2026, after which significant price erosion and generic entry are expected.
- Future revenue depends on maintaining market share, expanding indications, and strategic positioning ahead of patent expiration.
FAQs
Q1: When is generic midostaurin expected to enter the market?
A1: Pending patent expiry around 2026 and successful filings, generic versions could enter within 1–2 years thereafter.
Q2: How will patent expiration impact the drug's price?
A2: Prices are projected to decrease by 50–70% following generic entry, with corresponding revenue declines.
Q3: Are there approved generic alternatives currently available?
A3: No, generics are not yet approved. Off-label compounded forms exist but carry regulatory and safety limitations.
Q4: What are the major competitors to Rydapt?
A4: Gilteritinib (Xospata) is the main competitor in AML, with distinct regulatory approvals. No direct competition exists for SM indications.
Q5: How does price regulation in different markets affect global sales?
A5: Europe and other regions often negotiate lower prices via pricing agreements, limiting margins outside the U.S.
References
[1] FDA. (2017). FDA approves midostaurin for untreated AML with FLT3 mutation. Retrieved from https://www.fda.gov
[2] IQVIA. (2022). US Prescription Data for Rydapt. IQVIA Institute.
[3] Evaluate Pharma. (2023). Oncology Market Report. Evaluate Ltd.
[4] Novartis. (2022). Rydapt Product Label.
[5] Patent and Trademark Office. (2023). Patent expiration and legal status for midostaurin.
