In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients.
An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.
Orphazyme's hopes of getting EU approval for its Niemann-Pick disease type C (NPC) therapy arimoclomol after a rejection by the US FDA last year are dwindling fast.
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year