Most Expensive drugs in the US

Cost per
dose or year
1) Hemgenix CLS Behring, uniQure $3.5 mio
2) Skysona Bluebird bio $3.0 mio
3) Zynteglo Bluebird bio $2.8 mio
4) Zolgensma Novartis $2.25 mio
5) Myalept Chiesi Farmaceutici $1.26 mio
6) Zokinvy Eiger Biopharmaceuticals $1.07 mio
7) Danyelza Y-mAbs Therapeutics $1.01 mio
8) Kimmtrak Immunocore $975.250
9) Lixturna Spark Therapeutics $850.000
10) Folotyn Acrotech Biopharma $842.585

SWIPE NEXT

Source: Fierce Pharma

 1) Hemgenix

Cost per dose: $3.5 million

Compay: CSL Behring, uniQure

Disease: Hemophilia B
● Hemgenix is a one-time gene therapy approved by the FDA to
treat adults with hemophilia B who currently use factor IX
prophylactic therapy.
● Patients with moderate to severe hemophilia B can cost
healthcare systems more than $20 million over a lifetime, CSL
says.
● CSL is commercially responsible for the drug after licensing
rights from uniQure last May for $450 million.
● CSL noted that the $3.5 million price tag does not reflect
anticipated discounts that it will provide for Hemgenix,
including value-based agreements with commercial payers.

SWIPE NEXT

Source: Fierce Pharma

 2) Skysona

Cost per dose: $3 million

Compay: Bluebird bio

Disease: Cerebral adrenoleukodystrophy
● Bluebird arrived at its price for Skysona through a “data-
driven” process that took into “consideration the positive
clinical outcomes, quality of life improvements, health system
cost savings and potential societal impact of patients and
families.”
● Bluebird does not offer an outcome-based deal with Skysona
due to the complexity of the disease.
● Skysona is approved for boys between ages 4 and 17 and
bluebird expects to treat approximately 10 patients per year.
● Manufacturing a dose of Skysona takes 55 to 60 days and
bluebird expects to have 40 to 50 qualified treatment centers
online by the end of 2023.
SWIPE NEXT

Source: Fierce Pharma

 3) Zynteglo

Cost per dose: $2.8 million

Compay: Bluebird bio

Disease: Transfusion-dependent thalassemia
● Zynteglo is for beta thalassemia patients who require regular
red blood cell transfusions, with 1,300 to 1,500 people with the
disease in the US.
● The Institute for Clinical Economic Review (ICER) endorsed
bluebird’s price, placing the value of a dose of Zynteglo at
$2.77 million.
● Bluebird has a refund policy of 80% of the cost of the
treatment if patients don’t achieve independence from
transfusions.
● Authorizations for coverage of Zynteglo have taken only two
weeks and no payer has yet to deny coverage.

SWIPE NEXT

Source: Fierce Pharma

 4) Zolgensma

Cost per dose: $2.25 million

Compay: Novartis
Disease: Spinal muscular atrophy (SMA)
● Zolgensma is a one-time gene therapy treatment designed to
last for a lifetime.
● Zolgensma is approved for infants with Type 1 SMA.
● Novartis has sought to offset the sticker shock by offering
flexible payment models, such as spreading the payment over
five years.
● This approach has secured favorable coverage policies for
Zolgensma covering about 97% of patients on commercial
insurance and 86% on Medicaid.
● Long-term data shows that Zolgensma is producing lasting
effects, with all 10 phase 1 trial patients maintaining their
previously achieved milestones.
SWIPE NEXT

Source: Fierce Pharma

 5) Myalept

Cost per year: $1.26 million

Compay: Chiesi Farmaceutici

Disease: Leptin deficiency
● Myalept is a recombinant form of human leptin used to treat
leptin deficiency in patients with congenital or acquired
generalized lipodystrophy.
● Myalept was approved by the FDA in 2014 and has changed
hands many times since then.
● It is currently owned by Chiesi Farmaceutici, who acquired it
from Amryt Pharma in 2021.
● There are no cheaper alternatives to Myalept, as it is the only
approved treatment for the rare condition.
● In 2021, Myalept collected full-year sales of $141 million.

SWIPE NEXT

Source: Fierce Pharma

 6) Zokinvy

Cost per year: $1.07 million

Compay: Eiger BioPharmaceuticals

Disease: Hutchinson-Gilford progeria syndrome
and processing-deficient progeroid
laminopathies
● Zokinvy is a farnesyltransferase inhibitor that targets the toxic
buildup of progerin or progerinlike farnesylated proteins.
● Zokinvy was developed by Merck & Co. and licensed by Eiger
BioPharmaceuticals in 2018. Merck previously offered the
drug for free to progeroid researchers.
● Eiger scored a rare pediatric disease review voucher after
Zokinvy’s approval, which was sold for $95 million and split
with the Progeria Research Foundation.
● Zokinvy is not a cure for the diseases it is used to treat, and
most patients die before the age of 15 from heart disease or
strokes. SWIPE NEXT

Source: Fierce Pharma

Comment below
to receive the link
to the full ppt
👇👇👇