rare inherited disorders
Sanofi announces phase 3 study data for infantile-onset Pompe disease
Pompe disease is a rare, progressive neuromuscular disease, with infantile onset being the most aggressive form
Shionogi starts phase 2 clinical trial for Pompe Disease
The rare disease affects around one in every 22,000 people worldwide
Shionogi gains rights to Maze’s investigational Pompe disease therapy for $150m upfront
The inherited disorder is estimated to affect about one in 40,000 people in the US
Sanofi presents positive results for Pompe disease enzyme replacement therapy
The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK
European Commission approves Amicus Therapeutics’ Pompe disease therapy
The company can now offer Opfolda together with its previously-approved Pombiliti
Sanofi signs agreement worth up to $750m for Maze’s Pompe disease drug
The candidate has recently cleared its first phase 1 clinical trial
Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients
The rare genetic disorder affects around one in every 40,000 people in the UK
European Commission approves potential new treatment for Pompe disease
Nexviadyme is the first and only newly approved medicine for Pompe disease in Europe in over 15 years