argenx (ARGX) investor relations material

FDA leadership and regulatory outlook

• Recent departure of FDA Commissioner Dr. Makary signals a planned leadership reset amid White House frustration over ongoing controversies and lack of process predictability.

• Acting leadership is expected to maintain the status quo, with key divisions like CDRH seen as stable, but cell and gene therapy oversight remains contentious.

• The absence of a Senate-confirmed Commissioner may freeze major policy moves and slow regulatory innovation, especially in rare diseases.

• Upcoming PDUFA negotiations could be complicated by leadership uncertainty and shifting views on FDA-industry relations.

Drug pricing, MFN models, and industry agreements

• Recent industry agreements on drug pricing have bought time from legislative action but create long-term vulnerabilities, especially around prospective MFN policies.

• There is bipartisan potential for codifying MFN elements, with future negotiations likely to extend agreements and increase oversight.

• Demonstration programs like GUARD and GLOBE face implementation challenges due to complex carve-outs and legal uncertainties.

• Section 232 tariffs and onshoring agreements are used as leverage, but widespread exemptions dilute their impact.

China’s role in biopharma and U.S. policy response

• China’s rapid growth in clinical trials and drug development is attributed to regulatory agility, large patient pools, and government support.

• U.S. policymakers are increasingly concerned about supply chain security and the political risks of relying on Chinese data and manufacturing.

• There is bipartisan momentum for policies to slow or regulate the shift to China, but industry and Congress face challenges in crafting effective responses.

• The trend toward Chinese dominance is seen as difficult to reverse, with future U.S. policy likely to focus on onshoring and safeguarding innovation.