Tag: Gene therapy

With a list price of $4.25 million per dose, Lenmeldy became the most expensive prescription drug on the market after its FDA approval in early 2024. This staggering figure highlights how recent breakthroughs in gene therapy are redefining what is the most expensive prescription drug and sparking critical discussions about access and cost.

In the United States, hemophilia is estimated to affect between 30,000 and 33,000 males [1.9.1]. The question, 'What is the drug of choice for hemophilia?' does not have a single answer, as treatment is highly individualized based on the type, severity, and patient-specific factors.

Advancements in hemophilia treatment have transformed patient outcomes, with new options offering a significantly improved quality of life. For individuals with this genetic bleeding disorder, what are three treatments for hemophilia? The primary pharmacological approaches include traditional clotting factor replacement, innovative non-factor therapies, and, for some, a promising one-time gene therapy.

As of late 2024 and into 2025, the one-time gene therapy Lenmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy holds the title of the most expensive infusion drug, with a staggering list price of $4.25 million. This represents a new peak in the rapidly evolving landscape of ultra-high-cost pharmaceuticals, dominated by groundbreaking gene therapies for rare genetic diseases.

In a significant development for the rare disease community, the FDA approved the first-ever treatment for Friedreich's ataxia in early 2023, offering a major breakthrough after years with no disease-specific drugs. This milestone, along with other promising therapies currently under review, provides a clearer answer to the question, "What is the new medicine for ataxia?" and fuels optimism for future treatments across various forms of this debilitating condition.

Affecting approximately 1 in 40,000 to 60,000 births, Gaucher disease is the most common lysosomal storage disorder, caused by a deficiency of the enzyme glucocerebrosidase (GCase). Understanding the specific therapeutic strategies for this genetic condition is essential for managing symptoms and improving quality of life, which is precisely how you treat glucocerebrosidase deficiency.

Priced at $4.25 million for a single infusion, Kyowa Kirin's Lenmeldy is currently the most expensive drug substance in the world. This one-time gene therapy is a groundbreaking treatment for the ultra-rare genetic disorder metachromatic leukodystrophy (MLD), highlighting the high costs associated with advanced therapeutic innovation.

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?.

According to physician perception, new pharmaceuticals and biopharmaceuticals were the biggest contributors to improved health outcomes for eight debilitating diseases over a 20-year period. So, **what does a medical breakthrough offer** beyond incremental progress, providing revolutionary changes that can fundamentally alter a patient's life and the healthcare landscape? It offers unprecedented hope and transformative new possibilities.

With more than 7,000 rare diseases known worldwide, affecting an estimated 300 million people, Pfizer's focus on what is Pfizer rare disease represents a significant commitment to an area of high unmet medical need. For over thirty years, the company has worked to provide critical treatment options for patients with rare conditions, advancing research across areas like rare hematology, neurology, and cardiology.