Launch Execution RiskComplexities of the thalassemia patient population and REMS logistics are likely to produce a gradual, specialist-centered launch that could delay broader commercial uptake.
Regulatory And Indication UncertaintyA more complicated path to market in sickle cell disease, together with regulatory extensions tied to REMS discussions, creates uncertainty around future label expansion and longer-term revenue upside.
REMS And Safety MonitoringA REMS requiring frequent liver tests and a black box warning increases administrative burden for prescribers and may restrict patient access through added monitoring requirements.