Launch could be trickier than approval, not least because of problems diagnosing patients with primary haemophagocytic lymphohistiocytosis.
Swedish Orphan Biovitrum made waves yesterday by getting its rare disease asset emapalumab into the Ash late-breakers – and bagging approval soon afterwards.
Pain drugs look like they could face different outcomes, while targeted cancer agents will have a big month. Two biosimilar decisions also await.
Roche's Hemlibra has impressed, but uptake might not be as fast as some hope.
Experts expect Roche's Hemlibra to dominate the inhibitor patient population, but to be a big deal it will need to impress in non-inhibitor patients, too.