Competitors
It has dosing schedules of every 4 or 8 weeks
What is Donidalorsen?
3502 Takhzyro patients (As of May 2025)
what is the number of patients on Takhzyro in MCE regions (GEM & EUCAN)?
HAE is a rare genetic disorder affecting this system in the body
What is the contact pathway?
Can be taken up to 3 times during an attack
What is Firazyr?
I am a clotting factor and the target of Andembry.
What is Factor XIIa?
The top-selling LOC in FY24
What is Germany?
There are 8 known mutations of this indication
what is HAE-nC1INH indication?
It has a half life of 14 days
what is Takhzyro?
In the KONFIDENT study, the median time to beginning of symptom relief was 1.61 hours.
what is Sebetralstat?
28%
What is FY24 MCE Takhzyro value sales growth?
Despite being genetic, HAE can sometimes occur without a family history due to this type of mutation
What is a De-Novo mutation?
This medication type replaces or enhances C1-inhibitor levels to prevent or treat HAE attacks
What are C1-inhibitor therapies ?
Donidalorsen is designed to selectively bind and degrade this
What is mRNA for prekallikrein (PKK) ?
The first country to plan to launch the PEN device (Sep-2025)
What is Sweden?
Type II HAE differs from Type I by producing this form of defective protein instead of low levels
What is dysfunctional C1-inhibitor?
This treatment can be administered IV or SC
What is C1 esterase inhibitor?
The mean number of investigator-confirmed attacks per month was 0.27 compared to 2.01 with placebo.
What is Andembry?
The LOC with the highest volume growth in FY24
What is Saudi Arabia?
He defined a hereditary form in a 24-year old woman with episodic attacks, from five successive generations of family members who also suffered from attacks
What is Sir William Osler ?
Early treatment during an HAE attack will prevent serious complications
What is the acute treatment?