Tag: Gene therapy

In the United States, approximately 20 million people have some form of age-related macular degeneration (AMD), and about 200,000 new cases of wet AMD are diagnosed annually [1.10.2]. While anti-VEGF therapy has been a revolutionary treatment, researchers are actively exploring alternatives to reduce treatment burden. So, what is the alternative to anti-VEGF?

As of 2025, the most expensive medication in the U.S. is Lenmeldy, a gene therapy priced at $4.25 million per dose [1.2.7]. This article answers **what is the most expensive medication in the US?** and explores the groundbreaking science and complex economics behind these costly treatments.

In 2023, the cholesterol-lowering drug atorvastatin was the most prescribed medication by volume in the U.S., with over 115 million prescriptions dispensed. However, answering **what is the highest prescription** is more complex, as the term can refer to the highest number of prescriptions, the highest revenue, or the highest individual price, revealing different top contenders in each category.

While Zolgensma has been a life-altering gene therapy for infants with Spinal Muscular Atrophy (SMA), experts widely agree that **Why is Zolgensma not a cure?** is a critical question for managing patient expectations. The single-dose treatment halts disease progression but cannot reverse pre-existing neurological damage, a key distinction from a complete cure.

With a reported list price of up to $2.6 million in 2025, Zolgensma therapy is one of the most expensive single-dose treatments in the world. This article explains how much does Zolgensma therapy cost for patients and the healthcare system, exploring the complex factors that influence the final price and outlining available financial assistance.

With a list price of over $2 million per dose, Zolgensma is one of the most expensive medications in the world. This staggering cost raises a critical question for families affected by spinal muscular atrophy (SMA): who pays for Zolgensma? The answer involves a complex combination of health insurance coverage, manufacturer assistance programs, and innovative payment models.

Over the last decade, biologics have become the fastest-growing class of therapies in the United States, accounting for 40% of U.S. pharmaceutical sales. Understanding what are the four types of biologics is crucial for comprehending these advanced medical treatments that target specific disease pathways with high precision.

Costing $425,000 per eye, Luxturna (voretigene neparvovec-rzyl) is a one-time gene therapy widely recognized as the most expensive eye drug. This revolutionary treatment targets a rare, inherited form of vision loss and represents a significant advancement in the field of ophthalmic medicine.

In December 2023, the U.S. Food and Drug Administration (FDA) approved the world's first CRISPR-based therapy, Casgevy, marking a historic shift in treating genetic blood disorders. This groundbreaking approach directly modifies a patient's own cells, which is central to understanding what is the active ingredient in Casgevy.

In clinical trials, a single infusion of valoctocogene roxaparvovec reduced the mean annualized bleeding rate by 83.8% compared to prior Factor VIII prophylaxis [1.10.3]. This one-time gene therapy is transforming treatment, but how is valoctocogene roxaparvovec administered to achieve such significant results?