Which drug has the highest price? Unpacking the Cost of Revolutionary Gene Therapies

October 10, 2025 •

3 min read

With a price tag of $4.25 million for a single treatment, Lenmeldy, a gene therapy for metachromatic leukodystrophy (MLD), is the highest-priced drug in the U.S.. This staggering figure brings critical attention to the complex market of specialized medicines, forcing us to ask: which drug has the highest price and why?.

Quick Summary

Lenmeldy, a gene therapy for MLD, is currently the highest-priced drug on the market at $4.25 million. This reflects the high cost of developing one-time, curative treatments for ultra-rare genetic disorders and the small patient populations they serve.

Key Points

Lenmeldy is the most expensive drug: The gene therapy Lenmeldy holds the record for the highest price, at $4.25 million for a one-time treatment for metachromatic leukodystrophy (MLD).
Gene therapies are the cost leaders: The highest-priced drugs are almost exclusively one-time gene therapies for ultra-rare diseases, offering a potentially curative treatment rather than ongoing symptom management.
High R&D and small patient populations drive costs: The massive investment required for research and development, combined with the very limited number of patients for each therapy, necessitates an extremely high price per treatment.
Value is tied to long-term impact: Companies justify high prices by comparing the cost to a lifetime of managing the disease with traditional therapies, emphasizing the potential for long-term clinical benefits and reduced healthcare utilization.
Financial solutions are emerging: To manage the financial burden, manufacturers offer patient assistance programs, and some payers and companies engage in outcomes-based payment models and installment plans.
Affordability remains a challenge: Despite payment programs and innovative models, the accessibility and affordability of these multi-million-dollar therapies for patients and the broader healthcare system remain a serious concern.

The Reign of Lenmeldy and Other Ultra-Costly Therapies

Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics is the world's most expensive drug, a one-time gene therapy for metachromatic leukodystrophy (MLD) costing $4.25 million. MLD is a rare and fatal genetic disease causing nervous system breakdown, and Lenmeldy aims to halt its progression. Its price, approved in 2024, highlights the value of potentially curative treatments for devastating conditions.

Other costly gene therapies and rare disease treatments include:

Kebilidi (PTC Therapeutics): $3.95 million for AADC deficiency.
Hemgenix (CSL Behring): $3.5 million for hemophilia B.
Elevidys (Sarepta Therapeutics): $3.2 million for Duchenne muscular dystrophy.
Skysona (bluebird bio): $3 million for cerebral adrenoleukodystrophy (CALD).
Zynteglo (bluebird bio): $2.8 million for beta-thalassemia.
Zolgensma (Novartis): $2.32 million for spinal muscular atrophy (SMA).

Understanding the Factors Behind Sky-High Prices

High drug costs result from complex factors like the risks and expenses of R&D and manufacturing for rare diseases.

The Research and Development Challenge

Treatments for ultra-rare diseases like MLD have tiny patient populations, making high per-dose pricing necessary to recover R&D costs. Drug development is also high-risk, with many failures. Furthermore, gene therapies require complex, specialized, and labor-intensive manufacturing.

The 'Curative' Value Proposition

The high cost of many gene therapies is linked to their potential to offer a one-time cure, potentially saving more than a lifetime of managing chronic conditions. Companies argue this value, including improved quality of life, justifies the price.

Comparison of Ultra-Expensive Drugs in the US


Drug (Manufacturer)	Cost per Dose/Year	Condition Treated	Type of Therapy	Price Justification	Status
Lenmeldy (Orchard Therapeutics)	$4.25 million (one-time)	Metachromatic Leukodystrophy (MLD)	Gene Therapy	Single-dose, potentially curative for a fatal genetic disorder.	Approved 2024.
Kebilidi (PTC Therapeutics)	$3.95 million (one-time)	AADC Deficiency	Gene Therapy	First targeted treatment for an ultra-rare, fatal genetic disease.	Approved 2024.
Hemgenix (CSL Behring)	$3.5 million (one-time)	Hemophilia B	Gene Therapy	One-time treatment reduces or eliminates the need for expensive regular infusions.	Approved 2022.
Elevidys (Sarepta Therapeutics)	$3.2 million (one-time)	Duchenne Muscular Dystrophy (DMD)	Gene Therapy	Aims to slow the progression of a severe inherited degenerative disease.	Approved 2023.
Skysona (bluebird bio)	$3 million (one-time)	Cerebral Adrenoleukodystrophy (CALD)	Gene Therapy	Aims to slow a fatal neurodegenerative disease in boys.	Approved 2022.
Myalept (Chiesi Pharma)	$1.3 million (annually)	Generalized Lipodystrophy	Non-Gene Therapy	Replaces leptin in a rare condition, used daily for long-term treatment.	Approved 2014.
Zokinvy (Eiger BioPharmaceuticals)	$1.2 million (annually)	Progeria Syndrome	Non-Gene Therapy	First and only treatment for a rare premature aging disease.	Approved 2020.

Navigating Affordability and Accessibility

The high cost of these therapies raises concerns about accessibility. Manufacturers and insurers are exploring ways to ease the financial burden:

Patient Assistance Programs: Many companies offer programs to help eligible patients with costs.
Outcomes-Based Agreements: Payments may be linked to treatment effectiveness.
Installment Payment Plans: Some therapies offer payment options spread over several years.

Despite these efforts, obtaining insurance coverage can be difficult, and strict requirements may apply, making access a challenge for many.

Conclusion

The answer to which drug has the highest price currently points to Lenmeldy, a $4.25 million gene therapy for MLD, reflecting the trend of high costs for one-time, potentially curative treatments for ultra-rare diseases. These prices are driven by high R&D risks, small patient populations, and complex manufacturing. Balancing the promotion of innovation for rare diseases with the need for affordability and accessibility remains a significant challenge for the healthcare sector. For more on drug pricing, consult resources like Fierce Pharma.

Frequently Asked Questions

As of recent reports, Lenmeldy (atidarsagene autotemcel) is the most expensive drug, costing $4.25 million for a single treatment.

Lenmeldy is a gene therapy used to treat children with metachromatic leukodystrophy (MLD), a rare and fatal genetic disorder affecting the nervous system.

While gene therapies dominate the top of the price charts due to their one-time, curative nature, other drugs for rare conditions can also be very expensive due to small patient populations and high manufacturing costs. Myalept and Zokinvy are examples of costly non-gene therapies.

The high price is driven by immense research and development costs, complex and specialized manufacturing processes, and the limited number of patients who need the treatment for these ultra-rare diseases.

Drug manufacturers argue that the price reflects the potentially curative nature of the one-time treatment and the long-term healthcare savings compared to managing a lifelong, devastating disease. However, this pricing is a major point of debate among patients, payers, and healthcare economists.

For eligible patients, the cost can be covered through specialized insurance policies, patient assistance programs, outcomes-based payment agreements, or installment plans offered by manufacturers.

Yes. As the biopharma industry continues to invest in gene and cell therapies for rare diseases, new treatments with potentially higher price tags are expected to enter the market.