The latest Bell Potter Report considers that one of the positives of Neuren waiting for completion of its paediatric trial before partnering trofinetide is that it further de-risks the asset and is likely to attract interest from more than one party, which could enhance deal terms. The report also considers that the excellent response to enrolment to date suggests the potential commercial value of pursuing a paediatric label for trofinetide.
Last August I posted on the FDA's Rare Paediatric Disease Priority Review Voucher program, pondering whether Neuren might consider pursuing a paediatric label for trofinetide. I didn’t take the idea much further when it was pointed out that the program was set to finish this year. As the situation stands at the moment, the program is set to wind up at the end of this month.
HOWEVER…a proposal was put forward to extend the rare paediatric disease priority review voucher program as part of the 21st Century Cures legislation. This proposal received high levels of bipartisan support when it passed through the US House of Representatives in July last year. It was then passed (20-2) by a Senate Committee in March this year.
The Advancing Hope Act of 2015 proposes that the priority review voucher program for rare paediatric diseases be reauthorised through 2022, and that vouchers designated before 2022 be allowed to be awarded through 2027. Under the rules of the program, a drug sponsor intending to request a voucher for a rare paediatric disease drug would be required to notify the FDA of their intention to do so when submitting their NDA.
Although it was originally hoped that the Act would pass through the US Senate this June, prior to the Senate summer recess, it didn’t. Now with the legislators back in town, the aim is to push the Act through this month.
To recap how the program works, a drug company awarded a rare paediatric disease priority review voucher can use it in one of two ways. Either the voucher can be used to shorten the review of any future drug in its own pipeline to just 6 months, or it can be sold to another drug company allowing that company to speed the review of a new drug filing in its pipeline. The vouchers are considered a valuable commodity. Those sold to date have fetched prices ranging from $67 million to $350 million.
Assuming both success in Neuren’s current paediatric trial and the US Senate finally legislating to extend the priority review voucher program, would this not be an attractive incentive for any partner considering pursuing a paediatric label for trofinetide? Might this be another reason for Neuren choosing to wait a little longer before partnering?
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