The rare disease landscape is becoming increasingly sophisticated, demanding not only scientific innovation but also new approaches to access, evidence generation and patient support. Recordati Rare Diseases has built its growth by combining targeted business development with a lean, entrepreneurial organisation that remains closely connected to local healthcare systems. Luigi Longinotti, SVP EMEA, reflects on that journey, the opportunities ahead and why Europe continues to play a pivotal role in shaping the future of rare diseases.
What has your journey with Recordati Rare Diseases been, and what does your role encompass today?
I joined Recordati in 2014 as Corporate Portfolio Management Director for Orphan Drugs, with responsibility for portfolio management across business development, strategic marketing and pipeline management. At the time, the rare diseases business generated less than EUR 100 million in revenue, so it was still a relatively small niche. We had a clear ambition to build a global rare diseases platform, supported by an entrepreneurial culture and a team willing to pursue new opportunities. Many projects did not succeed, but we learned quickly, and those that did have played a defining role in shaping Recordati Rare Diseases into the organisation we are today.
For the past six years, I have been based in Paris, leading our EMEA region, one of Recordati’s three global regions alongside the US and Canada and the Rest of the World. EMEA is headquartered here because Recordati’s acquisition of Orphan Europe in 2007 laid the foundations for today’s rare diseases business. From Paris, I oversee all the functions involved in bringing our treatments to patients, spanning commercial, medical affairs, market access and all the other teams that support the business.
Today, we have a direct presence in more than 50 countries across EMEA, including almost every European market as well as the Middle East through our regional hub in Dubai. Rare diseases require a dedicated focus and a specialised mindset, which is why we operate with local organisations led by country managers who understand the specific needs of each healthcare system while remaining closely connected to the broader regional organisation.
To what do you attribute Recordati Rare Diseases’ sustained growth over the past decade?
Our growth reflects a strategy that has remained remarkably consistent since Recordati entered the rare diseases space through the acquisition of Orphan Europe in 2007. From the outset, we knew that becoming a leading player meant continuing to bring new products into our portfolio while building on what has always been our greatest strength: staying close to patients and healthcare professionals, supporting diagnosis and education, and helping innovative treatments reach the people who need them. That requires deep local expertise because every market has its own healthcare system, regulations and access pathways, and understanding those differences is a core part of what we do.
We began with our metabolic franchise, treating truly ultra-rare conditions where products such as Carbaglu (carglumic acid) make a life-saving difference despite serving only a handful of patients across Europe. We also made mistakes along the way, but we learned from them. The first real acceleration came with the acquisition of Signifor (pasireotide) and Isturisa (osilodrostat) from Novartis in 2019, which established our endocrinology franchise and marked an important step in the evolution of both our portfolio and our business. Throughout that growth, we have remained a lean organisation because our value lies in understanding our markets and staying close to patients and healthcare professionals. As organisations become larger and more layered, they inevitably lose some of that proximity, and we have worked hard to avoid that.
The acquisition of EUSA Pharma in 2022 represented another important milestone, adding a differentiated portfolio in rare and niche oncology while allowing us to leverage the platform and geographic reach we had built over many years. That geographic expansion has been a major driver of our growth because, in rare diseases, patients may be few, but they are everywhere. Our responsibility is to ensure that, wherever those patients are, they have the opportunity to benefit from the treatments they need.
What makes Europe such a strategic region for Recordati Rare Diseases, and where does it continue to hold a competitive advantage in rare diseases?
Europe remains the core of our business because it is where everything started. Although the US has become our largest region in recent years, Europe continues to play a fundamental role, not only because of its scale but because of the richness created by its diversity. The different healthcare systems, cultures and ways of working generate an exceptional breadth of medical expertise, and what we learn across Europe is often something we can replicate in other regions. That diversity creates ideas, capabilities and experience that continue to shape our business globally.
Another defining strength of Europe is the quality of its healthcare systems and the real-world evidence they generate. In rare diseases, clinical trials are often necessarily limited because patient populations are so small, which makes real-world evidence essential to demonstrating the full value of a treatment, supporting lifecycle management and exploring new opportunities for patients. One of the best examples is Isturisa (osilodrostat). Its recent indication expansion in the US, which represents one of our biggest growth opportunities today, was supported by real-world evidence generated by physicians in France. That is just one example, but it reflects what we see across much of our portfolio, where Europe’s clinical expertise continues to drive innovation and create value well beyond the region itself.
Europe also benefits from an exceptional rare disease community. Strong networks of physicians collaborate across borders rather than within individual countries, creating an environment where expertise and innovation can spread quickly. Our role is to support that community, facilitate collaboration wherever we can, and help ensure that patients benefit from the knowledge it generates. At the same time, we are encouraged by the initiatives underway at European Commission level to reduce bureaucracy and make access more consistent across Member States. While these efforts are still at an early stage, they are moving in the right direction. Ultimately, Europe’s greatest strength lies in its ability to bring together different healthcare systems, scientific expertise and clinical experience, and turn that diversity into better outcomes for patients.
How does Recordati Rare Diseases work with the wider healthcare ecosystem to improve diagnosis, access and patient care beyond developing medicines?
This is a responsibility we all feel very strongly, and I think it is one of the defining characteristics of our company. There is a real sense of urgency, combined with an entrepreneurial spirit that drives each of us to find solutions for patients. The challenge is that there is no single solution, and nothing can simply be replicated from one disease or one country to another. Every product is different, every patient population has different needs, and every healthcare system presents its own realities. Our role is therefore to understand those differences, identify the barriers that matter most, and work alongside healthcare professionals and the wider ecosystem to help address them. Sometimes that means improving diagnosis, sometimes facilitating access, and sometimes supporting the practical aspects of patient care, but it always starts with understanding what patients need in a particular country and how we can best contribute.
A good example is Cushing’s syndrome. In France, where the healthcare system allows almost all patients to be diagnosed, our focus is on supporting physicians and ensuring patients receive the most appropriate treatment. In neighbouring Germany, however, diagnosis rates remain much lower, largely because there are fewer endocrinologists per capita. As a small company, we cannot create more specialists, but we can work with the German Endocrine Society to raise awareness of the issue, engage with policymakers and support initiatives that strengthen specialist care over the long term. At the other end of the spectrum is high-risk neuroblastoma, where the challenge is entirely different. For children with high-risk neuroblastoma who can receive ambulatory infusion under local regulations, something as simple as designing a backpack to carry the infusion pump allows them greater freedom instead of remaining in a hospital bed throughout treatment. Those examples may seem very different, but together they reflect what our role really is. Whether we are helping address a long-term structural challenge within a healthcare system or developing a simple practical solution that improves a child’s daily life, our objective remains the same: to remove barriers wherever we can and help patients receive the care they need.
How can Europe strike the right balance between healthcare sustainability and ensuring patients have timely access to innovation?
I think this is one of the biggest challenges Europe will face in the years ahead. Across the region, there is a genuine willingness from physicians and healthcare systems to bring innovative treatments to patients, but access is still managed largely on a country-by-country basis, which inevitably creates inequalities. Patients in one country may receive a treatment quickly, while others face long delays or even have to travel because of local legislation, and that is frustrating for everyone involved. In this context, the Joint Clinical Assessment is a very positive step because it has the potential to streamline the assessment of innovation and facilitate access across Europe. At the same time, it is entirely reasonable that healthcare systems reward treatments that represent genuine innovation and deliver meaningful value for patients, while recognising that not every medicine can sustain the same price. The challenge is to apply that principle consistently and without unnecessary delays because, in some countries, patients wait so long that by the time an innovative medicine reaches the market, much of its value has already been lost. I think the direction has to be for all stakeholders to work together to raise the bar in defining what truly constitutes innovation and what genuinely adds value for patients, while having the courage to support that innovation even as governments introduce necessary cost-containment measures. Germany’s recent decision to increase the statutory manufacturer rebate reflects the pressures healthcare systems are facing, but the answer cannot simply be lower prices. It has to be a collaborative effort to improve the way we work, create greater value for patients and ensure that innovation remains both accessible and sustainable.
From a portfolio perspective, what has changed over the years, and what is driving the continued growth of your haematology and oncology franchise?
Our metabolic franchise was where everything started, but it is also the oldest part of the portfolio and was largely built around small molecules. As those products gradually faced generic competition, the business naturally evolved. Endocrinology became the next important growth driver and continues to represent a significant part of both our sales and future expansion, particularly because products such as Signifor (pasireotide) and Isturisa (osilodrostat) are still being launched in a number of markets. More recently, haematology and oncology has become our largest franchise, reflecting both the evolution of our portfolio and the opportunities we have created over the past few years. Qarziba (dinutuximab beta), our treatment for high-risk neuroblastoma, is a good example. Future growth is extending access to countries where these therapies have not yet been available, particularly across North Africa and parts of the Middle East.
The addition of Enjaymo (sutimlimab), following the acquisition of the global rights from Sanofi, has opened another exciting chapter for our haematology franchise. Coming from a business development background, I have always been used to assessing the potential of new products. In this case, however, the experience with patients and physicians has highlighted an even greater impact than we had initially anticipated.. From the outset, the response from physicians has been extremely positive because they have seen the impact it has on patients’ daily lives. Whenever you have a treatment that delivers that kind of value, our role is simply to be there, support the healthcare community, facilitate access wherever we can, and ensure that patients are able to benefit from it.
How do you foster the entrepreneurial culture that has become such a defining characteristic of Recordati Rare Diseases?
Entrepreneurship, or in our case ‘intrapreneurship,’ is something I try to reinforce every day across the organisation because, in rare diseases, it is not simply part of our culture; it is essential to the way we operate. There is no standard solution that can be replicated from one disease or one country to another, so the only way we can continue supporting patients and healthcare professionals is by empowering people to make decisions, equipping them with the knowledge, expertise and support they need, and ensuring they know the company stands behind them. That is why we try to push decision-making to the lowest possible level of the organisation, trusting talented people who combine technical expertise with a genuine sense of purpose, because without that purpose it is difficult to make a real difference for patients.
Just as importantly, people need to feel they can make mistakes. We learn a great deal from them, and creating an environment where people feel supported to test new ideas, always within the right compliance framework, is fundamental to maintaining that entrepreneurial spirit. Ultimately, what matters is giving our teams the confidence to keep looking for better ways to support patients and healthcare professionals.
As Recordati celebrates its centenary, how would you like Recordati Rare Diseases to be perceived, and what would success look like over the next few years?
Reaching 100 years is an extraordinary milestone for any pharmaceutical company, and I think it says a great deal about who we are and the kind of company we want to remain. We come from a family-owned business with a long-term perspective, focused on building a lasting legacy rather than delivering short-term results, and that mindset continues to shape our ambition to become a leading global player in rare diseases. Although the portfolio has evolved considerably over the years, our DNA has remained remarkably consistent, built around an entrepreneurial leadership style, a commitment to doing the right thing and a determination to support both patients and healthcare professionals wherever we can.
When we speak again in a few years, I hope we will be talking about how we have continued to accelerate both our growth and, more importantly, the number of patients we are able to reach. One of the most meaningful ways we measure our progress is by looking at how many patients benefit from our treatments through geographic expansion and new therapies. In rare diseases, every patient counts, and our ambition is to continue bringing meaningful innovation to patients wherever they are in the world.
What defines the opportunities you pursue as you continue expanding the portfolio?
Our ambition is to continue growing through business development. We have already added two therapies this year for ultra-rare metabolic disorders affecting only a handful of patients, but that is precisely why they matter. Whether there are 20 patients in one country or many more worldwide, our objective is always the same: to ensure they can benefit from innovation as quickly as possible.
That same philosophy guides how we evaluate every opportunity. The first question is always the value a treatment can bring to patients because, while there is no shortage of incremental innovation, we are looking for products that make a genuinely meaningful difference. We have built a very strong business development organisation and have been fortunate to secure some excellent deals over the years, always guided by the same principle that has shaped our business from the beginning: if a treatment delivers real value for patients, it creates the strongest foundation for everything that follows, from physician confidence to market access and long-term success.
