Khaled Elrefae of Recordati Rare Diseases discusses how the company is expanding its rare disease footprint across Saudi Arabia, the GCC, and the wider MENA region. He highlights the region’s high unmet medical needs, the strategic steps taken to accelerate product launches, and the role of partnerships, diagnostics, and genomics in improving patient access. Elrefae also reflects on Recordati’s approach to balancing local and global collaborations.
When we last spoke at the beginning of 2024, you had just stepped into your new role. What has happened since then?
When we last spoke, we discussed our aim to take a more direct role in managing our activities across the region while also strengthening local capabilities. We also talked about reviewing our current partnerships to ensure we are collaborating with the right local organisations that can support faster execution and improved access for those we serve Since then, we have made strong progress against that strategy. We have established our regional headquarters in Saudi Arabia, with the official inauguration planned for April. In parallel, we have built a full local organization in Saudi Arabia, covering regulatory, quality, medical, and commercial functions. This infrastructure has been critical in accelerating product launches and ensuring our therapies reach patients without unnecessary delays.
We have also advanced our portfolio across key therapeutic areas, including endocrinology, oncology, and metabolic diseases. Several products have been launched not only in Saudi Arabia but also across the GCC, including Kuwait and Qatar, with additional markets following.
Overall, compared with where we were a year ago, we have successfully built a solid regional infrastructure in KSA and the GCC and significantly accelerated the rollout of our portfolio across these markets.
How did Recordati Rare Diseases’ global double-digit growth in 2025 translate to the MENA region, and what were the main growth drivers across your key therapy areas?
Recordati Rare Diseases delivered double-digit global growth 11.8% as compared to full year 2024. The MENA region made a strong contribution to that overall performance.
This growth was broad-based across all our key therapy areas, including haematology-oncology, endocrinology, and metabolic diseases. A major driver was the successful launch of innovative therapies in several priority markets, which significantly expanded patient access and strengthened our presence across the region.
Another important milestone was bringing our oncology portfolio fully in-house in KSA and the GCC. By integrating these products into the Recordati Rare Diseases portfolio, we gained greater control over execution and accelerated growth in this segment. At the same time, we continued to work closely with strong local distributors, which enabled us to launch products beyond KSA and the GCC and extend our regional footprint.
Overall, these results reflect both the strength of our portfolio and our clear commitment to delivering meaningful value to patients across the MENA region.
What were the key drivers behind this standout 50 percent growth?
The first and most important driver was our people. When you want to deliver a major step change, you have to start by assessing your local capabilities and asking whether you have the right resources and skills in place. We did that honestly and focused on closing the gaps. Saudi Arabia is a strategic market for us, and there was no question that we needed a direct and strong presence there. Over the past year, we built a high-quality local team across regulatory, quality, medical, and commercial functions, largely by investing in strong local talent.
The second key driver was accelerating product launches. We ensured that we had the right focus and the right level of resources in our priority markets, particularly Saudi Arabia and the Gulf. This allowed us to move faster and execute more effectively across our portfolio.
Finally, we continued to build on our infrastructure and leverage the expertise of our local partners, especially beyond KSA and the GCC. The combination of strong people, successful new launches, and effective collaboration with local partners was the main reason behind our strong growth last year.
What has your experience been over the past year accelerating launches for very small patient populations across diverse markets?
My experience over the past year has been very aligned with the company’s ambition to focus on what truly matters to patients. In rare diseases, we are often working with very small patient populations, but our commitment is clear: to focus on the few and ensure they receive innovative therapies as quickly as possible. Accelerating access in rare diseases requires a high level of focus, coordination, and purpose, and that has been central to everything we do.
A strong example of this commitment is the recent strategic collaboration and licensing agreement announced by Recordati Rare Diseases with Moderna. This partnership aims to develop and commercialize worldwide mRNA-3927, an investigational therapy for propionic acidemia, a rare inherited metabolic disorder that often presents in early childhood. Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, in collaboration with Recordati, and if approved, Recordati will lead global commercialization. If approved, this could be the first disease-modifying treatment option on the market for this severe disease. . This illustrates how we continue to strengthen our position in rare diseases by investing in meaningful innovation.
At the regional level, accelerating launches of our existing portfolio remains a key priority. We are leveraging our experience and capabilities to ensure efficient execution across KSA, the GCC, and the wider MENA region. In parallel, we are exploring local partnerships with companies that do not yet have a presence in the Middle East, where we can support them by using our established infrastructure and expertise to bring rare disease therapies to patients.
Overall, by combining global innovation with strong regional execution and collaboration, we are taking the next step in transforming how rare disease treatments are launched and accessed across diverse markets in MENA.
As you prepare for this new chapter, what are the key steps you are taking to prepare the organisation, build new capabilities, and support this evolving partnership model?
As we prepare for this next chapter, our priority has been to clearly define and strengthen our value proposition. We are focused on ensuring that we can present a compelling and credible platform for global partners who are looking to collaborate with Recordati Rare Diseases in the region.
The first step has been looking inward. We have worked to ensure the organization is ready, both structurally and operationally. Over the past year, we demonstrated our ability to accelerate our portfolio across KSA and the GCC, supported by strong and well-aligned local distributor partnerships. At the same time, we invested in building internal capabilities that allow us to launch and operate directly, including establishing a fully functioning affiliate in Saudi Arabia and evolving our regional structure.
We have also expanded our physical and organizational footprint. This includes setting up a new office in Saudi Arabia, which will be fully integrated in the first half of 2026, and continuing to strengthen our presence across the GCC and the UAE. These steps are essential to supporting more complex partnership models and future launches.
Finally, we are staying connected with key partners in the region as part of our ongoing business development efforts. This includes keeping an eye on potential areas for collaboration that may support our long‑term ambitions. Together, these efforts are ensuring that we are well prepared to support evolving partnerships and sustain long-term growth in rare diseases across the region.
Our approach is very clear and focused. Within our rare diseases business, we have focused therapeutic areas where we have deep expertise and a strong strategic fit. Our portfolio today is concentrated in haematology, oncology, endocrinology, and rare metabolic diseases.
This clarity helps us manage the dynamic with current partners. Continue to collaborate closely with our local partners, including beyond KSA and the GCC, where these relationships remain important to our success.
Given your experience launching these products, what makes this region an attractive opportunity in rare diseases?
From a patient perspective, the MENA region represents a significant opportunity. The region has a population of more than half a billion, and there is a high incidence of rare diseases, particularly in KSA and the GCC. Many of these conditions are linked to regional genetic and demographic factors, and there is a substantial unmet medical need.
For us, this creates an important role in improving patient access. We have the expertise to accelerate launches and work with local stakeholders to provide practical solutions. For example, in Egypt, we recently implemented a patient support program to address affordability challenges, ensuring that patients can access therapies in a timely manner.
Beyond patient support, demonstrating value to healthcare systems is critical. Through market access initiatives and health economics studies, we show how our therapies are cost effective and impactful within hospitals and national healthcare systems.
Overall, there is great potential in the region. While challenges exist, we are committed to collaborating closely with governments, healthcare providers, and payers to deliver the right access solutions and bring innovative treatments to patients efficiently.
Another issue often raised is delays in diagnosis and difficulty identifying patients. Once a product is on the market, finding the right patients can be a major hurdle. How do you address these challenges?
The first step is to have a clear understanding of the patient journey. We need to identify the bottlenecks in each disease area, who is treating patients, who is referring them, and who sees them first. By mapping this journey, we can pinpoint where interventions are needed and where diagnostic challenges arise.
Once we understand these hotspots, we work to strengthen diagnostic capabilities. This includes collaborating with Centres of Excellence in Europe and the US to bring expertise and best practices to local hospitals. We also leverage technology, including AI algorithms, to help identify and diagnose patients more efficiently.
In rare diseases, everything comes back to finding the right patients. By combining deep knowledge of the patient journey with improved diagnostic infrastructure and technology, we can increase the speed and accuracy of diagnosis and ensure that patients receive the care they need.
To better understand the regional environment, we are seeing progress in national genomic programs. How is Recordati Rare Diseases engaging with these initiatives?
Genomics is a promising area because it allows for a deeper understanding of disease, helps map current and future health burdens, and can guide the selection of the right treatments for specific patients. It also creates the potential to develop more tailored therapies in the future.
That said, we are not fully there yet. While governments are making significant efforts in national genomic programs, there remain gaps in how data is analysed, interpreted, and applied. Analytical methods vary, and the integration of genomic information into clinical practice is still limited.
From our perspective, the key opportunity is to leverage genomics in clinical trials. By embedding this information into trial design, we can focus on the right patients and generate better outcomes.
Ultimately, the value lies not just in generating data, but in connecting the dots linking genomic insights to clinical decision-making. This will require continued collaboration between governments, healthcare providers, and pharmaceutical companies. We see this as a space that will continue to evolve, and we are committed to engaging actively to help translate genomic data into actionable strategies for patient care.
Given the global shortage of trials despite the high disease burden, what can you tell us about the current ecosystem, potential improvements, and your company’s engagement in this area?
Working in MENA and representing Recordati Rare Diseases, one of our key priorities is to collaborate closely and leverage global clinical trials to benefit patients in the region. Through our medical team, we have strengthened our capabilities in Saudi Arabia and the GCC over the past year to better support this effort.
We are actively seeking opportunities to bring global trials to the region, which allows us to contribute patient populations and engage more closely with local stakeholders. This also creates the potential to integrate genomic data into clinical practice, improving patient selection and outcomes.
While we are still in the early stages, this is a clear focus for us to support patient access in MENA.
