Disagreements between FDA review teams and senior leadership, like CBER head Vinay Prasad, create contradictory guidance for drug sponsors. Companies follow the review team's advice, only to be overruled by leadership, leading to wasted resources, delayed approvals, and significant frustration.
Palvela targets a market inefficiency by taking dermatology drugs typically sourced from compounding pharmacies for orphan diseases and developing them into proprietary, FDA-approved products. This strategy creates a regulated, reliable supply chain for rare conditions while building a valuable commercial franchise.
The FDA publicly promotes regulatory flexibility for rare diseases, yet industry insiders perceive it as less permissive than prior administrations. This disconnect between the agency's messaging and its actual decisions is fueling widespread criticism, investor uncertainty, and accusations of 'moving the goalposts'.
When leadership changes at the FDA, as with Vinay Prasad succeeding Peter Marks, a core tension emerges. The new head isn't obligated to follow prior agreements, but abruptly shifting regulatory expectations for companies mid-development creates industry whiplash and erodes trust in the agency's consistency.
Companies like VIR are making progress with masked T-cell engagers that limit systemic toxicity like cytokine release syndrome (CRS). This approach, which concentrates efficacy at the tumor site, could be the key to unlocking the broad potential of T-cell engagers beyond hematologic malignancies into the much larger solid tumor market.
In a crowded, genericized field like epilepsy, a new drug's success depends not just on achieving statistical significance but on the magnitude of its effect. For Xenon, the key question is whether its drug can match or exceed the ~34% placebo-adjusted seizure reduction shown by competitor Xcopri, setting a high bar for commercial relevance.
Atara and Pierre Fabre received a second Complete Response Letter (CRL) for clinical deficiencies after spending a year addressing the first CRL's manufacturing issues. This illustrates a critical communication breakdown where the FDA introduces new concerns late in the process, blindsiding companies that believed they were on a clear path to approval.
Sarepta is conducting an external search for a new CEO following Doug Ingram's departure, rather than promoting a long-tenured insider like the CFO. This suggests the board may be seeking a strategic or cultural shift for the company, which has long been defined by its aggressive and often controversial 'Sarepta belief' system.
Gossamer's Phase 3 drug for PAH failed after being designed around a promising subgroup identified in a post-hoc analysis of a less-than-stellar Phase 2 trial. This outcome serves as a cautionary tale for clinical development, highlighting the high risk of basing expensive pivotal studies on retrospective data mining rather than robust, pre-specified endpoints.