Author
Alpha1 Germany eV, as published in Alpha1 Journal 2/2023.
Dear Alphas,
As you may already know, recruitment is currently underway for the Phase 3 trial of the drug Fazirsiran.
Fazirsiran is currently the most promising treatment for liver disease associated with alpha-1 antitrypsin deficiency. This drug belongs to the class of siRNAs and reduces the production of the mutated alpha-1 antitrypsin without altering the genes. This is intended to halt the progression of the liver disease and allow for liver regeneration.
The Phase 2 trial has already yielded promising results: Several markers of liver damage improved significantly, and fazirsiran administration led to a reduction of more than 801 TP3T in the mutated protein in both blood and liver tissue. During the one-year observation period, lung function remained stable, and no serious adverse events occurred that would have necessitated discontinuation of the study. If you or your relatives carry the Pi*ZZ genotype, are affected by liver disease as part of alpha-1 antitrypsin deficiency, and do not have advanced lung disease, please contact us by phone (+49 241 80 36606) or email (alpha1@ukaachen.de). We would be happy to advise you on your individual case.
We would be delighted if you would help us bring the first drug for the treatment of alpha1-antitrypsin deficiency-associated liver disease to the clinic!
Greetings from Aachen!
