Ann: ATL1102 Toxicology Protocol submitted to US FDA, page-31

Take a look at the timeline on page 14 of the investor presentation post above itsa.

They need 16 arms from each group to have had 24 weeks of dosing. Phase 2/3 trial finishes after 52 weeks of dosing. This is 52 weeks per arm.

So my understanding would be that they would need all the boys in the trial to start dosing on day 1 to achieve what you are saying. This would be very difficult to achieve with rare disease.

There is a mechanism that allows sales of drugs prior to approval. But i have not seen this referred to in any ANP announcement. They do mention persuing sales in FDA land off the back of EMA approval, as well as FDA approval from that data, but i have not seen any mention of this for EMA land. Have I missed it?

Expansion trial is normal to support an accelarated approval. This is where some approved Gene therapies are having problems.