Eight years into its shift into rare diseases, Ferrer is moving into a new phase as its pipeline matures. CEO Mario Rovirosa reflects on the setbacks and lessons shaping that journey, as well as the growing pressures on Europe’s ability to sustain innovation and patient access. Throughout, Ferrer’s commitment to social justice remains closely intertwined with its ambitions as a pharmaceutical business.
How has Ferrer’s portfolio transformation progressed, and what have been the main successes and challenges along the way?
I think we have made significant progress in that transformation, although we have had both successes and failures along the way. We have not yet launched anything from the new pipeline that has been really meaningful for our results, but we are now in a very different position, with several assets reaching the later stages of development. We submitted in September a marketing authorisation application in Europe for nebulized treprostinil inhaled solution in idiopathic pulmonary fibrosis (IPF), for which the Phase III studies met their primary endpoints, showing significant preservation of lung function compared with placebo. This could represent an important change in the way patients with IPF are treated and, given the size of the opportunity, a major shift for Ferrer.
We are also nearing the end of a Phase II study with egalognastat, in progressive supranuclear palsy (PSP), with the readout expected in the first quarter of next year. The FDA has granted the programme Fast Track designation and is open to reviewing the Phase II data, so if the results are strong enough, there could be an opportunity to pursue an approval pathway directly with the FDA. With the EMA, we would most likely need a Phase III study. At the same time, we have just started two Phase III studies with pridopidine, for which we acquired co-development and commercialisation rights from Prilenia across Europe and several international markets. One is a pivotal study in amyotrophic lateral sclerosis (ALS), while the other is a confirmatory study in Huntington’s disease. The results are probably another two or three years away, but today we have several assets at a late stage.
Four years ago, the situation was almost the opposite. We were reaching the end of a Phase III study in ALS and were reasonably confident that we might have something, even if the data were not perfect. When we opened the data, however, there was no efficacy, so we closed the programme and suddenly found ourselves without anything at a late stage. Our goal has always been to maintain a balanced pipeline, with assets at both early and late stages, and when a late-stage programme fails, sometimes you have to go to the market and bring in something more advanced. That is what we did with pridopidine. Following the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on its European application in Huntington’s disease, we now need additional confirmatory data. With several programmes now at a late stage, we are looking for earlier-stage opportunities again to restore that balance.
We have had other setbacks along the way. With inhaled treprostinil in pulmonary hypertension associated with interstitial lung disease (PH-ILD), we obtained the European distribution rights after its successful US launch, but European regulators concluded that additional data would be needed before we could move forward with an application. These are the normal risks of drug development, particularly for us as relative newcomers to areas such as rare neurological diseases. We only started this strategy eight years ago, focusing on pulmonary vascular and interstitial lung diseases and rare neurological diseases, so we are still learning. We have accumulated a great deal of knowledge during that time, however, and I am much more confident today about the next data we will submit.
What has Ferrer learned from its experiences with the FDA and EMA, and how has this shaped its approach to development?
The first lesson is very simple: you need very good data, even when you are working in a disease with significant unmet need and no effective treatment available to patients. My personal impression is that the EMA is more cautious. With the FDA, safety comes first, but once that has been established, I think there is a greater willingness to accept some degree of risk and potentially grant approval while requiring additional studies afterwards. The EMA tends to expect not only a strong safety profile, but also compelling evidence of efficacy and a clear improvement over the standard of care.
We have had programmes where we failed because the data were simply not compelling or conclusive enough, and that has been an important lesson for us. That experience also shapes the kind of opportunities we pursue. We are unlikely to compete for what I would call the perfect assets because they are extremely expensive and generally go to big pharma. We therefore have to be comfortable with a certain degree of imperfection, perhaps an asset supported by one Phase III study rather than two, and recognise that this means working harder on registration, market access and pricing. When you have perfect data, all of those discussions become much easier.
As for our own capabilities, I would say that rather than having a traditional research and development department, we have a very strong “D”. We do not focus on discovery ourselves. Instead, we map the global landscape in our two therapeutic areas, understand who is developing what and at which stage, and approach companies when we identify an asset that fits our strategy. If there is an opportunity to license or acquire the rights, we bring the asset into Ferrer and take it forward through clinical and regulatory development. We have developed strong capabilities in executing that process and running these trials, although we are still learning with every programme.
Why is the US becoming more important to Ferrer, and how is this reshaping its international strategy?
Until now, we have been very focused on Europe, but the changing relationship between US and European pricing means we can no longer look at the two markets separately. Most of the assets we have looked at have also come from the US, where there are many start-ups developing very promising programmes. Historically, these companies often wanted to retain their US rights and find a partner for Europe, where navigating multiple markets, pricing systems and access pathways creates considerable complexity. Ferrer was well positioned in that model because we understood how to manage that complexity.
Today, that is no longer enough. Egalognastat, is a good example. We acquired exclusive worldwide rights from Asceneuron, including the US. A year ago, we might have considered finding a US partner while continuing to build our expertise in Europe, but today we find US a key market for us. .
This shift also creates a significant resource challenge for Ferrer because our resources are not unlimited. If the Phase II programme in PSP succeeds and we move forward with the IPF registration, preparing for both launches will require substantial investment, some of which will have to come from our existing business. The challenge is therefore not simply how we balance Europe and the US, but how we invest in these new opportunities while ensuring that the current portfolio continues to deliver until the new launches begin contributing.
How is Ferrer preparing its European footprint for the next wave of potential launches?
The opportunity in Europe is significant. The IPF market alone is worth around EUR 1.3 billion and has largely been built by Boehringer Ingelheim and Roche over the past decade, during which there has been relatively little innovation. If everything progresses as expected, we could launch in the near future, and that will require a significant expansion of our commercial presence. We already have sales capabilities in some countries, while in others, including France and the Italy, we need to build them. We have recently opened our UK affiliate and intend to establish a direct presence across the major European markets. Spain currently represents more than 40 percent of our business, but with these launches, Europe could eventually account for around 70 percent. If we are also successful in launching PSP in the US, our geographical mix could change completely over the next five years.
We will need new people to support that growth, but we are not looking to build very large structures. What matters is having the right profiles, which will be different from some of those we have traditionally recruited. There are people in large pharma companies who are attracted by the opportunity to build something rather than joining an organisation where everything has already been defined and their role is primarily to execute. We believe Ferrer can offer that opportunity, and our purpose and commitment to social justice are also important parts of the proposition. We are already building these teams in Germany, the Netherlands, Spain and UK, and so far we have not encountered major difficulties attracting the talent we need.
The launch sequence itself is largely determined by how quickly we can enter a market and the time required to negotiate pricing.
What differences does Ferrer see across international markets in terms of access, and where is it sourcing innovation today?
The complexity of market access varies considerably from one region to another, and it is ultimately a question of perspective. Europe can appear complex compared with the US, where despite dealing with different insurers, you are essentially operating within a single market. Europe involves multiple countries, pricing systems and access processes, but compared to the institutional market in Latin America, it can actually be more straightforward. We have been very successful in Latin America through the private market with our traditional portfolio, but rare disease products require access to public healthcare systems, where the process can take considerably more time and negotiation. Mexico, for example, requires you to navigate the national public system and its different institutions and procurement processes; Chile has a different route through the Ley Ricarte Soto for selected high-cost treatments; and in Brazil, after obtaining regulatory approval from ANVISA, broad public access through SUS requires going through the CONITEC incorporation process. So compared with Latin America, Europe can look relatively straightforward, while compared with the US, it is clearly more complex.
When it comes to sourcing innovation, we are not actively looking towards Asia today,. We know there is considerable innovation emerging from China, but when we attend major industry gatherings such as the Jefferies Global Healthcare Conference or the J.P. Morgan Healthcare Conference, we do not see many Asian companies working in the specific areas we are targeting. We see the same pattern when we map the global landscape: so far, we have not identified many Chinese, Indian or other Asian companies successfully advancing Phase II programmes in the fields most relevant to us, although we do see some opportunities coming from Australia.
I think this is largely a matter of time, and I am confident that Asian companies will become much more visible in these fields. For the moment, however, when we look globally for assets that fit our therapeutic focus and development capabilities, I would say the US remains our main source of innovation.
What drives Ferrer’s commitment to social justice, and how is that purpose embedded in the business?
To understand this, you first have to understand Sergi Ferrer-Salat, Ferrer’s President and owner. He inherited the family company and became President in 1998 following the death of his father, Carles Ferrer Salat, who founded Ferrer in 1959. Sergi has always been conscious that what he owns is largely a matter of luck: he was born into a wealthy family in Barcelona, while many equally capable people around the world have never had the same opportunities. He sees that inequality as profoundly unfair and believes that, because he has both the resources and a company, he has a responsibility to do something about it. For years, he supported organisations such as Greenpeace, WWF and Médecins Sans Frontières. After I had been at Ferrer for two years, I told him that the company itself should reflect those values, and that became the basis of Ferrer for good. “For good” is not simply about the medicines we develop; it is about using the business to contribute to a fairer society and a better planet. Eight years later, that positioning translates into decisions and projects across Ferrer every day.
We have embedded that purpose in the way the business is structured and managed. The Ferrer Foundations employ around 140 people and work across environmental projects, support for people in vulnerable situations, and opportunities for young people through education, music, culture and sport. Over the past five years, we have allocated an average of around 50 percent of our profits to social and environmental projects. We also have a dedicated Social Justice area that reports directly to me and is represented on the Management Committee, ensuring that these considerations form part of our business decisions rather than sitting alongside them. Our B Corp certification is another reflection of that commitment. We initially scored 100.6 points and reached 136.4 at our 2024 recertification, which at the time made Ferrer the highest-scoring pharmaceutical B Corp and placed us among the highest-scoring large B Corps globally. Achieving that score with factories and around 1,800 employees is not easy, so we take it very seriously.
Ultimately, we do this out of conviction, not because it is fashionable or because the political environment happens to support it. The shift away from social and environmental commitments in parts of the corporate world does not change our direction. We strongly oppose policies that move in the opposite direction, including many of those coming from the Trump administration, and I believe that sooner or later the reality of what is happening to the planet will force all of us to confront the consequences.
How can Europe remain competitive in pharmaceutical innovation, and how is Ferrer adapting to this changing environment?
Europe certainly has the talent, expertise and scientific capabilities to remain competitive in pharmaceutical innovation. The challenge is creating an environment that sufficiently encourages and finances that innovation. We see policies presented as supporting innovation which, when you look at the detail, reduce periods of data protection. Ultimately, however, somebody has to finance innovation, and today that is very clearly the US market. Whether you are a European or an American company, US prices have played a major role in financing pharmaceutical innovation.
The situation becomes particularly difficult if the US starts lowering its prices by referring to European prices, because that puts even greater pressure on Europe and ultimately risks limiting European patients’ access to innovation. If we are prepared to accept that medicines will reach Europe years later, as often happens in Latin America, that is one option; if we are not, then Europe needs to encourage innovation, address the pricing environment and avoid further reductions in protection periods. I strongly believe in European values and in preserving a certain way of doing business, because not everything should be acceptable simply because it generates a financial return. Europe has the people, expertise and capacity to innovate, but the current environment is making it increasingly difficult to develop the market as we did in the past, and ultimately the consequences will be felt by European patients.
For Ferrer, the answer is to adapt, remain flexible and keep fighting for our long-term sustainability. We are not here to maximise return on investment; we want to remain sustainable while doing something meaningful, which is why we chose to focus on rare diseases rather than simply continue manufacturing products such as paracetamol, of which there are already plenty in the world. At the same time, European authorities need to understand the context in which companies are making their decisions, because otherwise we will increasingly see companies choosing not to launch in Europe. This is a difficult balance when medicines represent significant public expenditure, particularly as large numbers of baby boomers retire over the next decade and place greater pressure on pensions and healthcare systems. Yet European patients still need access to innovative medicines; otherwise, we will simply have to accept that those medicines will reach Europe much later.
