Tag: Orphan drugs

Over 7,000 rare diseases collectively affect more than 30 million Americans, yet historically, most have had no approved treatment. Orphan drugs are specialized medications developed to treat, prevent, or diagnose these rare conditions, addressing a critical, unmet medical need.

Hereditary angioedema (HAE) medication costs can exceed hundreds of thousands of dollars per patient annually, with some treatments carrying a list price of over $600,000 per year. The question, **how much does hereditary angioedema medication cost?**, is complex, as the financial burden is influenced by the type of treatment, insurance coverage, and available patient assistance programs.

Initially launched with an annual wholesale price of approximately $32,500, the cost of Epidiolex can be a significant barrier for many patients. The complex reasons behind **why is Epidiolex so expensive** involve a multifaceted journey from lab to market, driven by stringent regulation and economic factors.

With a price tag of $4.25 million per one-time treatment, Lenmeldy, a gene therapy for a rare genetic disorder, holds the record for **what is the most expensive drug ever sold**. This staggering cost for a single dose highlights the unprecedented pricing of modern gene therapies and raises important questions about healthcare affordability and access.

Fabry disease affects up to 1 in 40,000 births, and for a specific subset of these patients, Migalastat, marketed as Galafold, offers a targeted therapeutic option. This oral medication functions as a pharmacological chaperone, addressing the root cause of the disorder in individuals with certain genetic mutations by stabilizing their own deficient enzyme.