The High Cost of Hope: How Zolgensma Became the World’s Most Expensive Drug

ProPublica’s investigation into the gene therapy Zolgensma sheds light on the journey of this breakthrough drug from its inception to becoming the world’s most expensive treatment. The drug, which has shown promising results in treating spinal muscular atrophy (SMA), a fatal disease in children, comes with a staggering price tag of over $2 million per dose.

The story begins with Vincent Gaynor, whose daughter Sophia was diagnosed with SMA. The Gaynors, through their charity Sophia’s Cure, played a crucial role in funding the early development of Zolgensma. However, as the drug progressed towards commercialization, the dynamics shifted. Researchers like Brian Kaspar, who developed the therapy, became entangled with biotech startups like AveXis, which eventually sold to pharmaceutical giant Novartis for billions.

The investigation reveals how taxpayer money and charitable donations fueled the initial research behind Zolgensma, only to be overshadowed by profit-driven motives as the drug reached the market. Executives and investors reaped massive financial gains, while families like the Gaynors felt left behind.

The pricing of Zolgensma, at over $2 million, sets a new standard for gene therapies, with other treatments following suit. The high cost of these drugs adds to the growing burden of prescription drug expenses in the U.S., impacting insurance premiums and public healthcare programs.

Despite the life-saving potential of Zolgensma, the exorbitant prices have put the drug out of reach for many children in low- and middle-income countries. The story highlights the ethical and financial complexities of drug development, raising questions about the true cost of medical advances and the balance between innovation and accessibility.

As families like the Gaynors navigate the challenges of accessing expensive treatments, the broader implications of drug pricing and access continue to shape the healthcare landscape. The investigation underscores the need for transparency, accountability, and equity in the pharmaceutical industry to ensure that life-saving treatments are accessible to all who need them.