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.
Discontinuations and an unusual revelation about Tecentriq’s breast cancer data dominated the R&D part of the company’s third-quarter results call.
Celgene’s surprise termination of a clinical trial makes for a day of soul-searching among other followers of the anti-CD47 approach.
Next-generation projects, including gene therapies, could soon be shaking up the haemophilia market.
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.