US FDA approval tracker: April 2022

Last month the US regulator tightened its grip over PI3K inhibitors by calling for randomised data instead of single-arm clinical studies. Ahead of an advisory committee meeting on the subject, TG Therapeutics pulled its own PI3K asset Ukoniq from sale and withdrew the filing of its combination product, U2. Elsewhere both Alnylam and Merck had Pdufa dates extended. For the latter, its 15-valent pneumococcal vaccine Vaxneuvance was heading for a decision in infants and children, a larger market than its already approved adult label. Despite the three-month delay Merck is still ahead of Pfizer in the younger age group, with that group's follow-on project, Prevnar 20, due to yield phase 3 in the second half of the year. Lastly Bristol Myers Squibb finally received the green light for mavacamten, now called Camzyos, in obstructive hypertrophic cardiomyopathy. The cardiac myosin inhibitor not only comes with a REMS and a black boxed warning, but a higher than expected price tag. With a list price of $89,500 a year it is well above the $15,000 figure ICER said would meet traditional thresholds of cost-effectiveness. Bristol has its work cut out to meet its $4bn-plus peak sales target, and justifying the $13.1bn it spent on Myokardia.