Autolus, Scholar Rock, Rocket and Pfizer have managed to convince the European Medicines Agency that their investigational treatments merit a place on the regulator’s priory medicines scheme.

While the drug may be well-positioned for registrational development, some important caveats remain after mixed data in Phase II study cohorts.

Key cancer asset is still looking for a Phase III validation after failing to outperform Keytruda in challenging frontline NSCLC setting.

The introductions of Spinraza and Zolgensma in SMA offer new insights into how to address neurodegenerative diseases. But more real world evidence is needed.