argenx (ARGX) investor relations material

FDA leadership and regulatory outlook

• Recent departure of FDA Commissioner Dr. Makary signals White House frustration with ongoing turmoil and unpredictability at the agency, with further leadership changes expected soon.

• Acting leadership is expected to maintain the status quo, limiting major policy moves until a new Commissioner is confirmed, which may not occur until late in the year.

• The FDA's lack of process predictability has led to increased investor and political pressure, with the agency's stability and scientific independence now more politicized.

• Upcoming PDUFA/user fee negotiations could be complicated by the absence of a confirmed Commissioner and shifting political dynamics.

• The cell and gene therapy division remains an area of concern, with no clear direction for change.

Drug pricing, MFN, and demonstration programs

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

• The administration is leveraging demonstration programs (GUARD, GLOBE, BRIDGE, BALANCE) as negotiating tools, with many exemptions and ongoing complexity, especially for orphan and small drug companies.

• There is skepticism about the enforceability and effectiveness of these demos, with some experts predicting they may not be fully implemented.

• The administration is using trade policy (Section 232, 301 tariffs) as leverage to secure voluntary agreements from companies, with many exemptions diluting their impact.

• The intersection of drug pricing and China policy is intensifying, with supply chain and clinical trial data from China under increasing scrutiny.

China, biopharma competition, and supply chain

• China is rapidly advancing in clinical trials and drug development, driven by policy changes and a large patient population, raising concerns about U.S. and European competitiveness.

• U.S. policymakers are reacting with restrictive measures, but industry experts argue that the trend toward Chinese innovation is too far advanced to reverse.

• There is bipartisan appetite in Congress to address the perceived threat, but policy responses are often reactive and complicated by national security concerns.

• The industry faces challenges explaining the benefits and risks of Chinese clinical data and supply chains to U.S. policymakers.

• Future policy may focus on onshoring and regulatory reforms, but the global nature of biopharma innovation complicates unilateral U.S. action.