Ann: First patent in Japan granted for Neuren's trofinetide, page-14

I’d caution against expectations of massive revenues from off label sales. While it’s not known what trofinetide will cost if it gets to market, it won’t be cheap. Therefore, uptake is likely to be dependent on reimbursement. If trofinetide is approved in Rett syndrome and Fragile-x, that shouldn’t be a problem. However, I would expect many payers would refuse to fund the use of a high-cost drug in a non-approved indication (payers are not renowned for their generosity). Nor would the company be allowed to promote trofinetide in any non-approved indications, under the current rules.