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Key takeaways:
Zolgensma is the most expensive
drug in America, with an average
one-time cost of $2.1 million.
Annual
cost
based on
length of
Drug Manufacturer therapy
Novartis Gene
1 Zolgensma $2,125,000
Therapies, Inc.
Eiger
2 Zokinvy $1,073,760
BioPharmaceuticals
Y--mAbs
3 Danyelza $1,011,882
Theraptuics, Inc.
Amryt
5 Myalept Pharmaceuticals, $929,951
Inc.
Acrotech
7 Folotyn $842,585
Biopharma
BioMarin
8 Brineura $755,898
Pharmaceuticals
Horizon
10 Ravicti $695,970
Therapeutics
Source: GoodRx
1. Zolgensma – $2,125,000
Zolgensma received FDA approval in May
2019. It has remained the most expensive
drug in the U.S., with a one-time cost of $2.1
million for a course of treatment.
2. Zokinvy – $1,073,760
Zokinvy, an orphan drug, was approved by the
FDA in November 2020. Its cost is staggering,
at over $1 million a year. It is the first and only
treatment approved for Hutchinson-Gilford
progeria syndrome, a rare genetic disease
that causes premature aging.
3. Danyelza – $1,011,882
The FDA approved Danyelza in November
2020 to treat neuroblastoma in the bone or
bone marrow of pediatric and adult patients.
Danyelza was under an accelerated approval
program and is considered an orphan drug
and breakthrough therapy.
4. Kimmtrak – $975,520
The newest drug to make the list, Kimmtrak
was approved by the FDA in January 2022 to
treat metastatic or surgically untreatable
uveal melanoma, a form of eye cancer.
Kimmtrak is administered via IV infusion by a
healthcare provider, and the typical person
will use one vial per week, every week, as a
maintenance dosage. A single vial costs
$18,760, which brings the yearly cost of
treatment to $975,520.
5. Myalept – $929,951
Myalept is one of the only drugs on this list
that is dispensed at the pharmacy. It is used
to treat leptin deficiency in patients with
generalized lipodystrophy, which causes
abnormal fat distribution in the body.
6. Luxturna – $850,000
At $850,000 a year, Luxturna is the sixth-most
expensive drug on the list. Luxturna is a gene
therapy that treats an inherited form of retinal
dystrophy, a condition that causes vision loss
and often even complete blindness.
7. Folotyn – $842,585
Folotyn is approved to treat peripheral T-cell
lymphoma, a rare blood cancer that can cause
death. A healthcare professional must
administer Folotyn, and people are typically
given 135 vials of the drug annually.
Manufacturer Acrotech Biopharma increased
the list price for a vial by 3% in January 2022
— from $6,058 to $6,241 Q bringing the total
annual cost to $842,585.
8. Brineura – $755,898
Brineura is a first-in-class medication that
treats a specific form of Batten disease called
late-infantile neuronal ceroid lipofuscinosis
type 2 kCLN2l. Specifically, Brineura helps
treat the slow loss of walking ability in
pediatric patients 3 years and older.
9) Blincyto – $754,720
The ninth-most expensive drug, Blincyto, has
an annual list price of $754,720.
Manufactured by Amgen, Blincyto is used to
treat a rare form of acute lymphoblastic
leukemia, a cancer of the blood and bone
marrow.
Methodology
Previous reports
September 2021
March 2021
August 2020
March 2020
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