The journey of the medicine, Caplacizumab – from a publicly funded scientific breakthrough to a high-cost pharmaceutical product controlled by a multinational corporation – illustrates the contradictions of the existing drug development system. 

It is a story of public investment, private capital, industrial consolidation, and the persistent question: Who ultimately benefits from medical innovation?

At the heart of this story lies a dilemma that defines the pharmaceutical landscape. On the one hand, venture capital, biotech start-ups, and pharmaceutical corporations are undeniably necessary under the current neoliberal system to bring new treatments to market. 

On the other, the logic of profit maximization, patents, and monopolistic pricing often ensures that life-saving medicines remain inaccessible to those who need them most.

Caplacizumab is a medicine developed to treat a rare and serious blood clotting disorder called acquired thrombotic thrombocytopenic purpura (aTTP), which can lead to blood clots in small blood vessels throughout the body.

Its development is a scientific success. But it is also a cautionary tale about the uneasy relationship between public good and private gain. 

What if there were an alternative that did not rely on the inevitable transition from public research to private ownership? The emerging vision of Public Pharma presents a radically different pathway, challenging the assumption that industrial monopolization is the only way forward.

Caplacizumab: A publicly funded breakthrough

The development of Caplacizumab lies in the publicly funded research of the Flanders Institute for Biotechnology (VIB), where scientists explored the potential of Nanobody® technology – antibody fragments derived from camelids. 

These tiny, stable molecules were an academic curiosity before they became the backbone of life-saving drugs such as Caplacizumab (trade name Cablivi).

This discovery was a testament to the power of curiosity-driven research. Without the support of public grants, institutional funding, and the intellectual freedom afforded to researchers, Nanobody® technology might never have seen the light of day. 

However, scientific discovery, no matter how groundbreaking, is not enough. Translating molecules into medicines requires capital, infrastructure, and expertise in clinical development.

This is where the state played a second vital role, not just as a funder of early research but as a creator of an environment where biotechnology start-ups could thrive. 

Belgium, recognizing the economic and medical potential of biotech, provided tax incentives, payroll deductions for research staff, and R&D grants to support companies willing to take risks.

These policies laid the groundwork for the creation of Ablynx in 2001—a spin-off from VIB tasked with commercializing Nanobody-based therapies. The company embodied the promise of academia-industry partnerships under the current pharmaceutical eco-system: scientific excellence paired with private investment.

Private capital: Necessary compromise or structural failure?

Ablynx, despite its academic origins, was not a philanthropic endeavor. From the moment it was founded, it needed funding beyond what government grants could provide. Venture capitalists like Gimv and Sofinnova recognized its potential and were willing to take some financial risk of investing in early-stage biotech.

Their bet paid off. Caplacizumab, developed over nearly two decades, demonstrated remarkable efficacy in treating acquired thrombotic thrombocytopenic purpura (aTTP). Clinical trials confirmed a dramatic reduction in the time required for platelet recovery and a significant decrease in relapses.

These results were not inevitable. They required substantial financial investment, regulatory navigation, and strategic decision-making. But the presence of private capital also meant that the endgame was never purely about patient access; it was about return on investment.

This is precisely where the structural failure of the system becomes evident. The reliance on venture capital means that, no matter how much public funding supports early-stage research, the final product is destined to end up as a private asset. Public resources de-risk innovation for investors, but they do not retain any claim over the resulting medicines.

When innovation becomes an asset

By 2018, Caplacizumab was on the cusp of regulatory approval. For Ablynx’s investors, this meant one thing: it was time to sell. Sanofi, one of the world’s largest pharmaceutical corporations, acquired Ablynx for €3.9 billion, securing exclusive rights to the company’s entire Nanobody® platform.

From a business perspective, the acquisition was a success. From a scientific perspective, it was validation. But from a public health perspective, it raised concerns. Caplacizumab, once a product of public funding and venture-backed risk-taking, was now firmly in the hands of a multinational corporation with an obligation to maximize shareholder value.

What did this mean for patients? It meant that Caplacizumab, now marketed as Cablivi, would be priced at nearly $8,000 per vial in the US with a full treatment course costing around $270,000. 

Even in Belgium—the country that helped fund its discovery—government agencies had to negotiate reimbursement schemes to make it accessible to patients. This medicinal product costs still over €5,000 per vial. 

And so the cycle continued: public institutions fund research, venture capital funds development, pharmaceutical corporations acquire and monopolize, and governments end up paying exorbitant prices to access the very medicines they helped create.

‘Public Pharma’ as an alternative 

If the story of Caplacizumab is emblematic of a system where public investment leads to private gain, what would an alternative look like?

The Public Pharma for Europe (PPfE) Coalition offers a concrete vision for breaking this cycle. The coalition argues that the current profit-driven pharmaceutical model is inherently dysfunctional by prioritizing profit over health, restricting innovation, and keeping essential medicines out of reach. 

Instead of a system where the state merely de-risks investments for private enterprises, Public Pharma calls for full public leadership in the research, development, production, and distribution of medicines.

Under such a system, the development of Caplacizumab might have taken a different path. Instead of transitioning from a publicly funded lab to a venture capital-backed startup and ultimately into the hands of Sanofi, the entire process – from research to commercialization – could have remained in public hands. 

This would not mean a return to slow-moving bureaucracy but rather a new model of state-led pharmaceutical infrastructure, one that prioritizes affordability, access, and transparency.

A Public Pharma approach would have ensured:

• Retention of public ownership: Instead of selling off promising biotech startups, public institutions could maintain ownership stakes, ensuring that profits are reinvested into further research rather than extracted by shareholders.
• Affordable pricing: Without the need to maximize returns, drug prices could be set based on production costs and equitable access rather than speculative market value.
• Health sovereignty: Countries and regions would not be at the mercy of multinational corporations for access to life-saving medicines.
• Democratic oversight: Instead of decisions being made behind closed doors by corporate executives, governance structures could involve public participation and transparency.

The PPfE Coalition asserts that governments should no longer limit their role to mitigating risks for the private sector. Instead, they should take full responsibility for pharmaceutical development to ensure medicines are developed for people, not profits.

Flawed but inevitable?

Caplacizumab’s journey raises difficult questions. Should we reject private capital, knowing that under the current economic paradigm few governments can afford to fund clinical development at scale? Should we reject industry, knowing that corporations provide the infrastructure for global manufacturing and distribution?

The problem is not that industry or investment exist – it is that the terms of engagement overwhelmingly favor the few over the many. If governments and public institutions play a crucial role in early research, why do they relinquish all control at the moment of commercialization? If taxpayers fund innovation, why do they then pay again—often at exploitative prices—to access the resulting treatments?

Caplacizumab is a triumph of scientific ingenuity, but it is also a reminder that scientific breakthroughs alone are not enough. Without structural changes, they will continue to follow the same path: from the lab to the marketplace, from the public to the private, from a breakthrough for humanity to an asset for shareholders.

The PPfE Coalition offers a bold alternative that ensures that life-saving medicines remain what they were always meant to be: a public good, not a private asset.

David Franco is a scientist and public health activist based in Leuven, Belgium. As a member of the People’s Health Movement, he focuses on the Public Pharma for Europe (PPfE) initiative, where he supports grassroots struggles for health justice and equity. David holds a PhD in Pharmaceutical Sciences from the Free University of Brussels (VUB).

Image Credits: VIB.