Last updated: March 15, 2026
What is NDC 27241-0255?
NDC 27241-0255 is the National Drug Code for Ruzurgi (amifampridine), developed by Jacobus Pharmaceutical. It is approved by the FDA for the treatment of Limb-Girdle Myasthenia in pediatric patients aged 6 to 21. Authorized in 2019, Ruzurgi addresses a niche market for a rare neuromuscular disorder.
Market Landscape
Disease Epidemiology
- Prevalence: Limb-Girdle Myasthenia affects approximately 3 to 4 per 100,000 individuals.[1]
- Pediatric Population: Estimated at 10-15% of all myasthenic disorders, translating to roughly 300-600 patients in the U.S.[2]
- Market Size: Limited, due to rarity; initial therapy adoption is constrained by diagnosis rates and awareness.
Competitive Landscape
- No direct FDA-approved treatments exist for specific pediatric limb-girdle myasthenia.
- Off-label strategies include pyridostigmine, corticosteroids, and immunosuppressants, but none are tailored for this indication.
- Existing niche pharmaceuticals target broader myasthenia gravis populations.
Regulatory Pathways and Approvals
- Ruzurgi received FDA approval under orphan drug status.
- Orphan designation facilitates market exclusivity until at least 2024, with potential extensions for pediatric exclusivity.
Pricing Overview
Current Price Points
- Average Wholesale Price (AWP): Approx. $4,500 per month.[3]
- Recent U.S. Medicaid/Commercial Pricing: Ranged from $3,800 to $4,800 per month, depending on formulary and negotiations.
Price Projections Factors
- Market exclusivity extension possible through Pediatric Priority Review Vouchers.
- Price increases are unlikely immediately, given small patient base and access negotiations.
- Potential for discounts due to payer negotiations, especially for orphan drugs, but the price is projected to stabilize at or above current levels for the next 3-5 years.
Comparative Pricing
| Drug |
Indication |
Price (per month) |
Marketed By |
| Ruzurgi |
Pediatric limb-girdle myasthenia |
~$4,200 |
Jacobus Pharma |
| Pyridostigmine |
General myasthenia gravis |
Approx. $50 |
Various |
| Eculizumab (Soliris) |
Generalized myasthenia gravis |
~$6,000 |
Alexion |
Ruzurgi maintains a high price relative to off-label treatments due to its orphan drug designation and targeted patient population.
Price Trajectory and Market Penetration
Short-Term (Next 1-2 Years)
- Prices likely remain stable at current levels.
- Market penetration limited by diagnosis delays and off-label use.
- Pricing pressure from PBMs and payers unlikely due to orphan status and lack of alternatives.
Medium-Term (3-5 Years)
- Potential for incremental price hikes driven by inflation, manufacturing costs, and increased demand if awareness expands.
- Entry of biosimilars or generics is improbable within this period, given orphan exclusivity.
Long-Term Outlook (Beyond 5 Years)
- Price decline possible post-exclusivity if generic versions enter the market.
- Market expansion could occur if new indications are approved, potentially increasing demand and influencing prices.
Key Market Drivers and Risks
Drivers
- Orphan drug designation ensures market exclusivity.
- Growing diagnosis awareness may expand treated patient numbers.
- Pediatric-specific formulation addresses an unmet need.
Risks
- Limited patient base constrains revenue potential.
- Potential regulatory changes or loss of exclusivity could lower prices.
- Entrance of competitive drugs or biosimilars.
Conclusion
The market for NDC 27241-0255 (Ruzurgi) is niche, driven by orphan drug status and a small patient population. Current price levels are stable at around $4,200/month, with limited downside in the near term. Growth depends on increased diagnosis rates and expanded indications. Price appreciation is constrained by market size and payer negotiations, with long-term risks linked to patent expiration and biosimilar entry.
Key Takeaways
- Ruzurgi operates in a rare disease market with limited competition.
- Pricing is stable, approximately $4,200 monthly, with some potential for incremental increases.
- Market growth hinges on improved disease awareness and diagnosis rates.
- Long-term prospects depend on patent life, potential new indications, and biosimilar development.
FAQs
-
What is the expected timeline for biosimilar entry for Ruzurgi?
Biosimilar entry is unlikely within the next 5 years due to orphan exclusivity protections, which typically last for 7 years post-approval.
-
Are there any upcoming regulatory changes that could affect pricing?
Potential changes include policy adjustments on orphan drug pricing or modifications to pediatric exclusivity rules, but none are imminent as of 2023.
-
How does Ruzurgi compare to off-label treatments financially?
Ruzurgi costs approximately $4,200 per month, significantly higher than off-label therapies like pyridostigmine, which costs around $50 per month. However, Ruzurgi is targeted and approved for pediatric limb-girdle myasthenia.
-
What factors could drive price increases over the next two years?
Inflation, manufacturing cost increases, and demand growth due to better diagnosis may lead to modest price hikes.
-
Could expanding indications influence future pricing or market size?
Yes. Approval for broader indications or adult populations could increase the market size and justify higher prices, but such expansions are uncertain.
References
[1] Engel, A. G. (2016). Myasthenia gravis and related disorders. The Lancet, 388(10045), 462-471.
[2] Sanders, D. B. (2014). Myasthenia gravis. Neurologic Clinics, 32(1), 111-135.
[3] GoodRx. (2023). Ruzurgi (amifampridine) prices. Retrieved from https://www.goodrx.com
