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.
ESMO 2024 was chock full of oncological data, diverse presentations providing hope across the cancer spectrum from breast and lung to unmet needs in colorectal and bladder.