The serial acquirer of royalty interests is coming into its own, but the secret to its success lies in cystic fibrosis.
Real-world data on Translarna look good, but with a failed confirmatory trial and a troubled history US approval is far from guaranteed.
June will see adcoms aplenty, with Bluebird, Acadia, and the Covid vaccine makers Novavax, Pfizer/Biontech and Moderna all facing FDA committees.
Tirzepatide heads for its first FDA approval and Enhertu eyes an earlier setting, as Verrica and Axsome keep their fingers crossed.
The second quarter should bring pivotal data for Karuna and key cardiomyopathy results for Alnylam, while Ionis's eplontersen lags in amyloidosis.