It is not practical to develop a single product like NNZ-2591 to...

It is not practical to develop a single product like NNZ-2591 to treat both rare and common disorders. It needs to be one or the other. The reason lies in the grossly different pricing structures for the two different markets being addressed.

For the rare diseases chosen thus far, NNZ-2591 can be expected to attract dosing prices similar to that of trofentide ($375,000 p.a.). Regulatory bodies, healthcare insurers, Medicare, etc accept that level of pricing because of the nature of the targeted disease.

If made available as a treatment for autism its pricing for this application would need to be that acceptable for a common disease, which obviously would be grossly less than the above figure.

Doctors treating Phelan-McDermid or Pitt-Hopkins patients then would be given the option of writing off-label prescriptions for NNZ-2591 at the lower price, and the higher pricing structure would be destroyed.

Logistical problems also would arise with commercialisation into a massive market like autism. For these reasons Neuren has stated its intention to proceed only along the less ambitious rare disease pathway.