Top 10 most expensive drugs in the world: Novartis gene therapy drugs rank first

Recently, GoodRx, a US drug price tracking website, published a list of the 10 most expensive drugs. Novartis's spinal muscular atrophy gene therapy drug Zolgensma ranked first with US$2.125 million (approximately 13.8 million yuan), while the newly approved premature aging drug Zokinvy and the new tumor drug Danyelza successfully entered the list with 103.2. Ten thousand U.S. dollars and 977.6 thousand U.S. dollars ranked second and third on the list.

On the whole, the list is mainly based on drugs for the treatment of rare diseases. It is expected that the global rare disease market will continue to grow in the future.

Top 10 most expensive drugs in the world

Ranking Drug Name Company Annual Usage Expenses (ten thousand U.S. dollars)

1.Zolgensma / Novartis 212.5

For the treatment of spinal muscular atrophy (SMA) under 2 years old

2. Zokinvy / Eiger Biopharmaceuticals 103.2

Used to treat progeria

3. Danyelza / Y-mAbs Therapeutics 97.76

For the treatment of relapsed/refractory high-risk neuroblastoma

4. Myalept / Aegerion Pharmaceuticals 89.99

Dietary supplemental replacement therapy for patients with leptin (leptin) deficiency and complications of congenital or acquired systemic lipodystrophy

5. Luxturna / Spark Therapeutics 85.00

For the treatment of inherited progressive retinal diseases caused by REP65 gene mutations

6. Folotyn / Acrotech Biopharma 79.38

For the treatment of patients with refractory/relapsed peripheral T-cell lymphoma

7. Brineura / Biomarin Pharmaceuticals 73.03

Enzyme replacement therapy for the treatment of CLN2 (known as neuronal lipofuscinosis)

8. Blincyto / Amgen 71.26

For the treatment of Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukemia (rrALL) patients

9.Ravicti / Horizon Therapeutics 69.59

For long-term management of UCD adults and children who cannot be treated with dietary protein restriction and/or supplementation alone

10.Soliris / Alexion 67.83

Used to treat paroxysmal nocturnal hemoglobinuria in adults and children, and subsequently approved for atypical hemolytic uremic syndrome