U.S. vs. China: 10 of The Most Expensive Orphan Drug List 2025

Introduction

Do you know that designed to treat rare, life-threatening diseases, the orphan drug list now dominates the list of the world’s most expensive medicines?

Imagine paying over $4 million for a single dose of medicine!

As shocking as that sounds, this is the real cost of some of today’s most advanced treatments — and nearly all of them share one thing in common: they’re orphan drugs.

But what makes these drugs so expensive? And how do their prices — and availability — differ between the U.S. and China?

This article explores the orphan drug list 2025, spotlighting the 10 most expensive orphan drugs in the U.S., and comparing their cost, accessibility, and reimbursement status in China.

What are orphan drugs and why are they so expensive?

Orphan drugs are medicines developed to treat rare diseases affecting a very small number of patients — typically fewer than 200,000 in the U.S.

Because these diseases are so uncommon, pharmaceutical companies face huge challenges: the cost to research, develop, and test these drugs remains very high, but the market size is extremely limited.

Without enough patients to spread out the costs, companies charge very high prices to recoup their investments.

Additionally, orphan drugs often involve complex, cutting-edge technologies like gene therapy or enzyme replacement, which add to manufacturing costs.

To encourage companies to develop these essential treatments despite the small patient populations, regulators such as the FDA grant special incentives like market exclusivity and tax credits, allowing these drugs to be priced at a premium.

What are orphan drugs at the FDA?

According to the FDA orphan drug requirements, sponsors who develop treatments for such rare conditions can benefit from incentives like tax credits, waived application fees, and 7-year market exclusivity upon approval.

The official FDA orphan drug list is maintained by the Office of Orphan Products Development (OOPD), documenting all drugs granted orphan status.

EMA orphan drug designation list vs FDA: How do they differ?

The EMA orphan drug designation list includes drugs approved under the European Medicines Agency’s criteria, which defines rare diseases as affecting fewer than 5 in 10,000 people in the EU.

In contrast, FDA orphan drug requirements focus on a threshold of 200,000 patients in the U.S.

Additionally, while both regulators offer similar incentives, the orphan drug designation vs approval process differs: designation occurs early in development, while approval confirms the drug’s safety and efficacy after clinical trials.

10 of the Most Expensive Orphan Drug List 2025 in the U.S.

Rank	Drug Name	Ingredient	Price (USD)	FDA Approved Indication	FDA Approval Date
1	Lenmeldy	atidarsagene autotemcel	$4,250,000/once	Metachromatic leukodystrophy (MLD)	March 18, 2024
2	Hemgenix	etranacogene dezaparvovec	$3,500,000/dose	Hemophilia B	November 22, 2022
3	Elevidys	delandistrogene moxeparvovec	$3,200,000/dose	Duchenne muscular dystrophy (DMD)	June 22, 2023
4	Skysona	elivaldogene autotemcel	$3,000,000/dose	Cerebral adrenoleukodystrophy (CALD)	September 16, 2022
5	Zynteglo	betibeglogene autotemcel	$2,800,000/dose	β-thalassemia	September 16, 2022
6	Zolgensma	onasemnogene abeparvovec	$2,100,000/dose	Spinal muscular atrophy (SMA)	May 24, 2019
7	Myalept	metreleptin	$1,300,000/year	Lipodystrophy-related leptin deficiency	February 24, 2014
8	Danyelza	naxitamab-gqgk	$1,200,000/year	High-risk neuroblastoma	November 25, 2020
9	Zokinvy	lonafarnib	$1,200,000/year	Hutchinson-Gilford progeria syndrome	November 20, 2020
10	Kimmtrak	tebentafusp-tebn	$1,100,000/year	Uveal melanoma	January 25, 2022

These drugs are listed in the updated FDA orphan drug list and represent the forefront of innovation in rare disease treatment.

Many of them also appear on the EMA orphan drug designation list, affirming their critical global relevance.

How much do orphan drugs cost in China?

So, how much do orphan drugs cost in China? While unit costs may appear lower than in the U.S., the burden remains high for many families.

Compared to the U.S., the cost of orphan drugs in China varies dramatically.

Several high-cost orphan drugs such as Zolgensma and Spinraza have been introduced in China, below is a partial price table.

Brand Name	Generic Name	Indication	Manufacturer	Est. Cost (RMB/year)	Est. Cost (USD/year)
Fabrazan	Agalsidase beta	Fabry Disease	Sanofi Genzyme	¥2,000,000–2,200,000	~$280,000–308,000
Geleining	Velaglucerase alpha	Gaucher Disease	BeiHaiKangCheng/ first domestic version	¥1,200,000–1,400,000	~$168,000–196,000
Saxintuximab	Rituximab	Rare hematologic diseases	Innovent / Junshi / Henlius	¥300,000–400,000	~$42,000–56,000
Brigatinib	Brigatinib	ALK+ NSCLC (rare mutation)	Betta Pharma	¥300,000–350,000	~$42,000–49,000
Minlutrombopag	Eltrombopag	Rare ITP variants	Jiangsu Hengrui	¥200,000–300,000	~$28,000–42,000
Ibruteza	Ibrutinib	Rare blood cancers	BeiGen (biosimilar)	¥200,000–300,000	~$28,000–42,000
Panima	Panitumumab	KRAS wild-type colorectal ca.	BeiGene	¥200,000–250,000	~$28,000–35,000
Lusutrom	Lusutrombopag	Thrombocytopenia	Jiangsu Hengrui	¥120,000–200,000	~$17,000–28,000
Anastrex	Anastrozole	Male breast cancer (rare)	Fosun Pharma	¥100,000–120,000	~$14,000–17,000
AUKONTALS	Edaravone	Amyotrophic Lateral Sclerosis (ALS)	Auzone Pharma	/	/

Summary

The orphan drug list 2025 continues to grow, bringing hope and treatment to patients worldwide.

By understanding what are orphan drugs at the FDA, asking how much do orphan drugs cost in China, and comparing the orphan drug list China vs U.S.

We gain clearer insight into the global fight against rare diseases.

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FAQ About Orphan Drug List