Pharmaceutical Industry Update: Regulatory Surprises, Legislative Changes, and Clinical Advancements

Unexpected FDA Approval and Regulatory Controversy

In a surprising turn of events, KalVista Pharmaceuticals' Ekterly has received FDA approval for the treatment of hereditary angioedema. This approval comes despite reported attempts by FDA Commissioner Marty Makary to reject the application. The drug's journey to market was marked by delays and controversy, highlighting the complex landscape of drug approvals in the current regulatory environment.

Meanwhile, Health Secretary Robert F. Kennedy Jr. faces another lawsuit, this time from a group of medical organizations seeking to reverse his recent decision to remove COVID-19 shots from routine immunization guidelines for healthy children and pregnant women. This legal challenge underscores the ongoing debates surrounding vaccine policies and public health strategies.

Legislative Changes and Industry Implications

President Donald Trump has signed the One Big, Beautiful Bill into law, introducing significant tax reforms with implications for the biopharmaceutical sector. Notably, the law includes expanded IRA exemptions for orphan drugs, potentially incentivizing further research and development in rare disease treatments.

The pharmaceutical industry is also closely watching the implementation of Kennedy's extensive HHS overhaul. Despite initial widespread layoffs, several positions have already been reinstated, indicating a fluid situation within the department. These changes are likely to have far-reaching effects on healthcare policy and industry regulations.

Clinical Advancements and Patient Advocacy

The second half of 2025 promises several key milestones in drug development. Eli Lilly's oral obesity candidate, orforglipron, is approaching a highly anticipated Phase III readout. Additionally, the TIGIT space continues to evolve, with four therapies showing promise despite previous setbacks in the field.

Patient advocacy is also making waves, as evidenced by BrainStorm Cell Therapeutics' support for a Citizens' Petition requesting FDA approval of its cell therapy NurOwn for ALS treatment. This development highlights the ongoing challenges in bringing innovative therapies to market, especially in areas of high unmet medical need.