Tag: Orphan drug

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.

Affecting an estimated one in ten Americans, there are more than 7,000 rare diseases, and for these patients, an orphan drug provides a critical, often life-saving, treatment option. Historically, the small patient populations for these conditions made drug development financially unviable, leading to a significant gap in treatment options.

In 2024, the global orphan drug market reached an estimated revenue of nearly $190 billion, illustrating the significant financial impact of these medications. This success raises the question: **What is the highest grossing orphan drug?** The answer is more complex than it appears, as many top sellers have expanded indications far beyond their initial orphan status.

With an initial price tag exceeding $2.1 million per dose, Zolgensma became known as the world's most expensive medicine upon its approval in 2019. This staggering cost is the result of a complex interplay of factors, including its groundbreaking gene therapy technology, a small patient population, and the innovative pricing strategies employed by pharmaceutical companies.

According to the manufacturer, Biocodex, DIACOMIT (stiripentol) is an orphan medicinal product and is distributed exclusively through a specialty pharmacy, confirming it is a specialty drug. This classification significantly impacts how the medication is accessed and managed for patients with Dravet syndrome.

Hereditary Angioedema (HAE) is a rare genetic disorder affecting an estimated 1 in 50,000 people worldwide. For those managing this condition, knowing their treatment options is critical. So, **what is another name for Firazyr?** The other name for the brand Firazyr is its generic name, **icatibant**.

For the average patient, the annual list price of Cerezyme (imiglucerase) is approximately $200,000, though this figure can vary significantly based on dosage, disease severity, and individual factors. This high cost is typical for specialized orphan drugs designed to treat rare genetic disorders like Gaucher disease.

With an average annual cost potentially exceeding $600,000 per patient, the price of TAKHZYRO is staggering. An investigation into why is TAKHZYRO so expensive reveals a complex interplay of pharmaceutical economics, research, and market dynamics that drive up the cost of this life-changing, but incredibly expensive, medication for hereditary angioedema (HAE).

The annual wholesale acquisition cost for Orladeyo has reached over $44,000 per pack, placing a significant financial burden on patients. Understanding why is Orladeyo so expensive involves examining a multifaceted system, including its status as an orphan drug, the high cost of research, and strategic market positioning.

Miltefosine, the only oral treatment for leishmaniasis, was originally developed as an anticancer agent before being repurposed for parasitic diseases. Knowing how to obtain miltefosine requires navigating a specific set of procedures, including securing a medical prescription, understanding commercial availability, and adhering to strict safety protocols.