Drug Development ChallengesINZY faces well-known challenges in rare disease drug development, including real world patient identification and disease awareness, few/no regulatory comparisons, and limited data on certain indications.
FDA ViewsThe data was in a small number of patients and there are questions about FDA’s views on a pivotal trial design.
Mixed Update ReactionINZY shares reacted negatively post the recent mixed update on ABCC6, and there are some questions regarding the program including endpoint selection and regulatory buy-in.