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I can’t see why Acadia wouldn’t be eligible for orphan designation for NNZ-2591 in both Rett syndrome and Fragile-X syndrome.

Provided that (a) the condition being treated has a US population of <200,000 (b) a scientific rationale is provided to show that the drug might be able to treat the disease and (c) that this drug has not already been granted orphan designation for this indication, it should be possible.

While a Priority Review Voucher may not be up for grabs if Acadia goes ahead with NNZ-2591 in either Rett syndrome or Fragile-X (only one can be granted per drug), orphan designation would still confer the following significant benefits:

• Tax credits for some of the clinical trial costs.
• Waiver of PDUFA (drug approval application) fee, which is raised each year and is set at more than US$4.3m in 2025.
• Priority review- an orphan designated drug qualifies for a 6 month FDA regulatory review versus a standard drug review timeframe of 10 months or more.
• A marketing exclusivity period (protecting from generic competition) of 7 years in the United States and 10 years in the EU.