The group indicated it had obtained orphan drug status for Zeneo® Midazolam from the FDA for the treatment of “status epilepticus”, i.e. epileptic crises of over five minutes.
Orphan drug designation is granted by the FDA to novel therapeutics for diseases or conditions affecting fewer than 200,000 patients in the US or greater than 200,000 patients if there is no reasonable expectation that the production cost of the drug will be covered by its sales. Such a designation allows the drug developer to be eligible for a 7-year period of US marketing exclusivity upon approval of the drug, as well as, in some cases, tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees. As a reminder, market approval for Zeneo® Midazolam should be asked in FY19 for both the US and Europe.
As such, the news does not change our estimates but it is positive given the benefits expected from this designation and it was unsurprisingly welcomed by the market.