The Top 10 Most Expensive Drugs in the U.S.: Starting From 1 Million

Photo byFreepik.com

Medical innovations have brought about treatments that can change the lives of patients with rare and life-threatening conditions. But these advancements come with price tags that are often as staggering as they are life-saving, raising questions about where to draw the line between ethics and profit.

Here’s a look at the ten most expensive drugs available in the U.S.:

10. Kimmtrak - $1.1 Million Annually

Kimmtrak (tebentafusp) is an immunotherapy developed by Immunocore for treating uveal melanoma, a rare and aggressive eye cancer.

Costing $1.1 million annually, it is designed for patients with the HLA-A*02:01 genetic marker. Kimmtrak enhances the immune system’s ability to target and destroy melanoma cells. Clinical trials demonstrated a median overall survival benefit of six months, making it a crucial treatment for this challenging cancer.

9. Zokinvy - $1.2 Million Annually

Zokinvy (lonafarnib) is a medication for Hutchinson-Gilford Progeria Syndrome (HGPS), a rare genetic disorder that accelerates aging in children.

With an annual cost of $1.2 million, Zokinvy works by inhibiting the accumulation of defective proteins that cause the disease. Clinical trials have shown that Zokinvy can extend the lifespan of children with Progeria by an average of 2.5 years, offering them a longer and better quality of life.

8. Danyelza - $1.2 Million Annually

Danyelza (naxitamab), an immunotherapy from Y-mAbs Therapeutics, is used to treat high-risk neuroblastoma, a type of cancer that primarily affects children.

Priced at $1.2 million per year, Danyelza targets cancer cells in the nervous system and activates the immune system to fight the tumor. The treatment has shown promising response rates in clinical trials, offering new hope for children battling this aggressive disease.

7. Myalept - $1.3 Million Annually

Myalept (metreleptin) is a therapy for generalized lipodystrophy, a rare condition affecting fat storage and metabolism. Manufactured by Amryt Pharmaceuticals,

Myalept costs approximately $1.3 million per year. This daily injection helps regulate metabolic functions, significantly improving the management of complications like severe insulin resistance and high triglyceride levels, which are common in lipodystrophy patients.

6. Zolgensma - $2.1 Million per Dose

Zolgensma (onasemnogene abeparvovec) is a revolutionary gene therapy developed by Novartis to treat Spinal Muscular Atrophy (SMA) in young children.

At a cost of $2.1 million for a one-time intravenous dose, Zolgensma replaces the defective SMN1 gene responsible for the disease. This therapy can halt the progression of SMA, offering a potentially curative treatment for a condition that is often fatal in early childhood.

5. Zynteglo - $2.8 Million per Dose

Zynteglo (betibeglogene autotemcel or beti-cel) is a gene therapy from bluebird bio designed to treat beta-thalassemia, a severe blood disorder.

Priced at $2.8 million for a single infusion, Zynteglo introduces a functional beta-globin gene into the patient’s stem cells, potentially eliminating the need for regular blood transfusions. Clinical trials have shown that most patients achieve transfusion independence, dramatically improving their quality of life.

4. Skysona - $3 Million per Dose

Skysona (elivaldogene autotemcel or eli-cel) is another gene therapy from bluebird bio, intended to treat cerebral adrenoleukodystrophy (CALD), a fatal neurological disorder affecting young boys.

At $3 million per dose, Skysona works by delivering a functional gene that produces the ALDP enzyme, crucial for breaking down harmful fatty acids. This treatment has shown a significant reduction in disease progression, offering a critical lifeline for affected patients.

3. Elevidys - $3.2 Million per Dose

Elevidys (delandistrogene moxeparvovec), developed by Sarepta Therapeutics, is aimed at treating children aged 4-5 with Duchenne Muscular Dystrophy (DMD).

With a one-time cost of $3.2 million, Elevidys delivers a gene that produces a functional, shortened version of the dystrophin protein, essential for muscle health. Though still under accelerated approval, Elevidys represents a significant advancement in treating this debilitating condition.

2. Hemgenix - $3.5 Million per Dose

Hemgenix (etranacogene dezaparvovec) by CSL Behring is a gene therapy for hemophilia B, a rare bleeding disorder.

Priced at $3.5 million for a single intravenous infusion, Hemgenix helps patients produce their own factor IX, a crucial blood-clotting protein. Clinical trials have shown a significant reduction in bleeding episodes, offering patients a chance to lead more normal lives without the burden of frequent infusions.

1. Lenmeldy - $4.25 Million per Treatment

Topping the list is Lenmeldy (atidarsagene autotemcel), the most expensive drug in the U.S., with a cost of $4.25 million for a single treatment.

Developed by Orchard Therapeutics, Lenmeldy is a gene therapy for children with metachromatic leukodystrophy (MLD), a severe genetic disorder that causes progressive neurological damage. By restoring the defective ARSA gene, Lenmeldy has the potential to halt the disease's progression, offering hope where there was once little.

Conclusion

These drugs exemplify the cutting-edge advancements in medical science, offering life-changing and, in some cases, life-saving treatments for rare diseases. However, their extraordinary costs highlight the ongoing challenge of balancing innovation with accessibility and ethics with profit.

As personalized medicine and gene therapies become more prevalent, the healthcare industry must navigate the complexities of ensuring these breakthroughs are available to all who need them.