Investigational drugs from Outlook Therapeutics and Vertex/CRISPR could become first-of-their-kind products if they win marketing approval.

The European Medicines Agency says it does not have enough data to justify the approval of Olumiant for hospitalized COVID-19 patients. The marketing authorization application for another product, Reata’s Imbarkyd for chronic kidney disease, has also been withdrawn.

With the planned $487m acquisition of sensorineural-focused Akouos, Lilly makes its second gene therapy M&A play in two years, on top of partnering/financing activity in the space.

Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.