Is Zolgensma the most expensive drug? An Exploration of Escalating Gene Therapy Costs

October 17, 2025 •

3 min read

In 2019, Novartis's Zolgensma was approved for treating spinal muscular atrophy (SMA) with a record-breaking $2.125 million price tag, quickly earning the reputation as the world's most expensive drug. This one-time gene therapy offered a groundbreaking treatment for a rare and devastating disease. However, in the years since, the field of gene therapy has continued to advance, and Zolgensma's time at the top of the price list has come to an end.

Quick Summary

Zolgensma, once the most expensive drug globally at $2.125 million, has been surpassed by newer gene therapies such as Lenmeldy and Hemgenix. These highly priced, single-dose treatments for rare genetic diseases raise questions about R&D costs, manufacturing complexity, small patient populations, and patient access. The high upfront cost is often weighed against the potential long-term savings compared to chronic, lifelong care.

Key Points

No Longer the Most Expensive: Zolgensma was famously the world's most expensive drug in 2019 but has since been overtaken by newer gene therapies.
New Price Records: The title of 'most expensive drug' has been claimed by others, notably Lenmeldy at $4.25 million per treatment.
Driven by Gene Therapy Costs: The high cost of Zolgensma and its successors is rooted in extensive R&D, small patient populations for rare diseases, and complex manufacturing processes.
Alternatives Exist for SMA: For treating Spinal Muscular Atrophy (SMA), alternatives like Spinraza (injections) and Evrysdi (oral) offer different cost structures and administration methods compared to Zolgensma's one-time infusion.
One-Time vs. Chronic Care: The immense upfront cost of Zolgensma is weighed against the potential for a long-term or permanent solution versus decades of managing a chronic condition.
Fueling Pricing Debates: The high price of gene therapies drives ongoing public and policy debates about fair pricing, access, and affordability, particularly for rare diseases.

The Rise and Fall of Zolgensma's Price Record

Approved by the FDA in May 2019, Novartis's Zolgensma (onasemnogene abeparvovec), a gene therapy for Spinal Muscular Atrophy (SMA), made headlines with its $2.125 million price for a single dose. This one-time treatment addresses the genetic cause of SMA, a rare and often fatal disease in infants. Novartis justified the price by comparing it to the lifelong costs of other SMA treatments and the value of a single curative therapy. Newer gene and cell therapies have since surpassed Zolgensma's price.

The New Class of Record-Breaking Gene Therapies

The gene therapy market has seen the introduction of therapies with prices exceeding Zolgensma's, targeting other rare genetic disorders. For more details on some of these expensive therapies, see {Link: Fierce Pharma https://www.fiercepharma.com/special-reports/most-expensive-drugs-us-2025}.

Factors Driving the High Cost of Gene Therapies

The high prices of gene therapies stem from factors unique to their development and application. These include high research and development costs, small patient populations, complex manufacturing, and the potential for a one-time cure. For a comprehensive look at the factors driving these costs, please refer to {Link: Fierce Pharma https://www.fiercepharma.com/special-reports/most-expensive-drugs-us-2025}.

Zolgensma vs. Other SMA Treatments: A Cost Comparison

Zolgensma is not the only treatment for SMA. The table below outlines these differences:


Feature	Zolgensma	Spinraza (Nusinersen)	Evrysdi (Risdiplam)
Mechanism	Gene replacement therapy; delivers a functional copy of the SMN1 gene.	Antisense oligonucleotide; increases production of SMN protein from the SMN2 gene.	Oral splicing modifier; increases production of SMN protein from the SMN2 gene.
Administration	One-time intravenous (IV) infusion.	Intrathecal injection (into the spinal fluid), requiring repeated doses.	Oral solution taken daily at home.
Eligibility	Children under 2 years of age at diagnosis.	Children and adults of all ages with SMA.	Children and adults of all ages with SMA.
Upfront Cost	$2.125 million (originally).	$750,000 for the first year, followed by $375,000 annually.	Depends on body weight; generally lower annual cost than Spinraza.
Long-Term Cost	The high upfront cost covers the entire course of the gene therapy.	Cost accumulates significantly over a patient's lifetime (e.g., ~$4 million over 10 years).	Ongoing daily treatment with accumulating costs over a lifetime.

This comparison highlights the different economic models and treatment approaches for SMA.

The Ongoing Debate Over Access and Affordability

The high cost of gene therapies fuels significant debate regarding access and affordability. While manufacturers emphasize R&D costs and long-term value, concerns remain about the financial burden on healthcare systems and global access disparities. Innovative payment models, such as outcomes-based pricing, are being explored. However, obtaining insurance coverage remains a challenge, often involving strict criteria and hurdles. This highlights the tension between promoting innovation and ensuring patient access to life-saving treatments.

Conclusion

While Zolgensma garnered attention as the most expensive drug upon its release in 2019, that distinction has since passed to newer gene therapies for other rare diseases. The rising cost of gene therapies is influenced by factors like extensive R&D, limited patient populations, and complex manufacturing. The debate continues regarding the balance between the high upfront cost of a potential 'one-time cure' and the financial impact on healthcare systems and equitable access globally. Zolgensma remains a notable example in the complex economic landscape of modern medicine.

Frequently Asked Questions

As of early 2024, Lenmeldy (atidarsagene autotemcel), a gene therapy for metachromatic leukodystrophy (MLD), is considered the most expensive drug, with a price tag of $4.25 million.

Zolgensma is a gene therapy used to treat Spinal Muscular Atrophy (SMA) in children under two years of age. It works by replacing the function of the missing or nonworking SMN1 gene with a new, healthy copy.

Zolgensma lost its title as other innovative gene therapies for different rare diseases were approved and brought to market with even higher list prices. The rapid development in the gene therapy space is continuously setting new pricing benchmarks.

Yes, other treatments for SMA include Spinraza (nusinersen), an injectable medication, and Evrysdi (risdiplam), an oral solution. Unlike Zolgensma's single dose, these treatments require ongoing administration.

Gene therapies carry high price tags due to several factors: the substantial cost and high-risk nature of research and development, the small patient populations they target, and the complex, specialized manufacturing and distribution processes involved.

Coverage for expensive gene therapies like Zolgensma can be complicated and often requires a rigorous approval process. While some plans may cover it, others have strict requirements, and patient financial assistance programs also exist.

The cost-effectiveness of a one-time treatment versus chronic care is a complex debate. While a single, high upfront payment may represent savings compared to decades of accumulating medical costs and care, it can also present a significant immediate financial burden and may not always provide a guaranteed lifelong cure.