ALN-AT3

Published

dm+d

Unassigned

New Medicines

Haemophilia A and B

Information

New molecular entity
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals

Development and Regulatory status

None
Phase I Clinical Trials
Phase I Clinical Trials
Yes

Aug 14: Orphan drug designation in the EU. [1]

Category

RNA interference (RNAi) therapeutic targeting antithrombin (AT)
Haemophilia A is defined by loss-of-function mutations in Factor VIII, and there are greater than 40,000 registered patients in the U.S. and E.U. Haemophilia B, defined by loss-of-function mutations in Factor IX, affects greater than 9,500 registered patients in the U.S. and E.U. [1]
Haemophilia A and B
Subcutaneous

Trial or other data

Aug 14: Top-line data from Part A of Phase 1 study, in adult healthy volunteers (n=4, 3:1 drug:placebo). Initial results showed that a single, low subcutaneous ALN-AT3 dose of 0.03 mg/kg resulted in an up to 28-32% knockdown of antithrombin (AT) at nadir (p