Clinical TrialsThe core of a potential FDA filing in ENPP1 deficiency is expected to come from the randomized ENERGY-3 trial in pediatric ENPP1 patients aged 1-13.
Mixed Program UpdatesINZY 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.
Regulatory 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.