Each dose costs up to $3.5 million, and new drugs for hemophilia are approved

Disposable gene therapy is known for its high price, and the US FDA recently approved an expensive new drug, which will cost as much as $3.
5 million
per dose.
The new drug, called Hemgenix, was developed by CSL Behring and Unique and is the first gene therapy
approved by the FDA for the treatment of moderate to severe hemophilia B.
Hemophilia B is a life-threatening degenerative disease in which patients are deficient in clotting factor IX (FIX)
due to genetic mutations.
Patients with the disease are prone to bleeding in joints, muscles, and internal organs, with pain, swelling, and joint damage
.
Current treatment includes lifelong prophylactic transfusion of clotting factor IX to temporarily replace or replace low levels of clotting factors
.
The approved Hemgenix treatment works by delivering a gene that produces the missing clotting factor to cells in the liver, enabling the patient to produce the ninth clotting factor
themselves.
According to clinical trial data, subjects could still stably express factor 9 after 18 months of treatment, resulting in a 54% reduction in the average annual bleeding rate
.
More importantly, after receiving a single injection, 94% of participants were exempted from the time-consuming and costly lifelong clotting factor injection
.
Jetbering bought the commercialization rights
to Hemgenix from uniQure, a U.
S.
-listed company, in 2020.
Jetbering has a focus on rare and serious diseases, and his innovative portfolio of medicines includes a range of recombinant and plasma products for the treatment of bleeding disorders, immunodeficiency disorders and chronic inflammatory demyelinating polyneuropathy, as well as hereditary angioedema and α-1 antitrypsin deficiency
.
Jetbelin commercialized
the gene therapy after acquiring global patent rights from Unique for $450 million in 2020.
According to uniQure's earlier estimates, there are approximately 16 million people with hemophilia B in
the United States and Europe.
It is worth mentioning that pricing has always been a topic that cannot be avoided by new drugs, and disposable gene therapy is known
for its high price.
Counting Hemgenix, the title of "the world's most expensive drug" has actually changed three times in just over three
months.
It is reported that Novartis' gene therapy Zolgensma has occupied the title of the world's "most expensive drug" for nearly two years
with a price of $2.
125 million.
In August and September this year, two gene therapies under Bluebird Biologics were approved by the FDA in succession, namely Zynteglo for the treatment of β-thalassemia ($2.
8 million) and Skysona for the treatment of early cerebral adrenoleukodystrophy ($3 million).

Despite the high pricing, the industry remains optimistic
about Hemgenix's future sales.
Referring to Novartis' third quarterly report, Zolgensma achieved sales of $1.
061 billion in the first nine months of this year, analysts say that Hemgenix is still likely to succeed
.
On the one hand, because existing hemophilia treatments are also expensive, on the other hand, hemophilia patients are in a state
of worry about bleeding for a long time.
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to anyone.