“This approval is an important step forward for patients with extensive-stage small cell lung cancer in Europe. It enables broader access to a new maintenance treatment option in a setting where the need for improved outcomes remains high, and reflects our commitment to bringing innovative therapies to patients with serious diseases and limited treatment alternatives.”

"Today’s approval marks a major milestone for Immedica, but an even greater one for people living with ARG1-D and their families in Canada. As the first and only disease-modifying treatment for ARG1-D, LOARGYS provides healthcare professionals with a treatment option specifically designed to address this ultra-rare disease"

The MHRA approval of Efmody is an important step forward for patients living with adrenal insufficiency in the UK. By expanding access to this innovative treatment, we hope to support patients in achieving a better quality of life, with less fatigue.

2025 was a year of growth, momentum and strong execution. With an expanded global footprint, a growing portfolio and a dedicated organization, Immedica has never been better positioned to deliver lasting impact for patients with rare diseases, healthcare systems and society at large

The extension of Efmody indication represents an important milestone for patients living with adrenal insufficiency, who face a significant daily treatment burden

Small cell lung cancer remains a highly challenging disease with significant unmet medical need. The CHMP’s positive opinion represents an important step towards bringing this new treatment option to patients in Europe.

“Today’s FDA accelerated approval of Loargys is an important milestone for Immedica and for patients and families affected by ARG1-D in the U.S. This outcome is the result of collaborative efforts across the entire ARG1-D community including advocacy groups, researchers, and clinicians. We are proud to be able to deliver a treatment option for patients and families who have long awaited progress."

The completion of the pediatric study on Loargys underscore our commitment to addressing unmet needs in rare metabolic diseases. Early initiation of a disease-modifying treatment in ARG1-D is a high unmet medical need and not only has the potential to alter the trajectory of disease, but also to give families greater confidence about the future."

"We appreciate the FDA’s acceptance of our BLA resubmission and the constructive dialogue throughout the review process. The assignment of a PDUFA target action date represents an important step forward for pegzilarginase. We remain focused on advancing this application with the goal of making a potential new treatment available to patients living with arginase 1 deficiency, a rare and serious condition with significant unmet medical need"

This acquisition supports our ambition to reach more patients living with rare diseases while continuing to build a sustainable and focused business. Alkindi and Efmody are well-established therapies that play an important role in the long-term management of adrenal insufficiency and congenital adrenal hyperplasia, and we are committed to ensuring continuity of care for patients. At the same time, the portfolio brings attractive, growing and durable revenues and strengthens our platform for long-term growth

We are very pleased to announce the approval of Ravicti in Japan, which represents an important milestone in our commitment to improving global access to treatments for patients with rare diseases.

The approval in Oman is the first in the MENA region and supports Immedica’s ambition to broaden access to essential therapies for rare diseases in markets where unmet medical need remains high

The submission of pegzilarginase to Health Canada represents an important step in our mission to bring transformative therapies to patients with rare diseases worldwide

The long-term data on Loargys underscore our commitment to addressing unmet needs in rare metabolic diseases. These results demonstrate not only sustained biochemical control, but also clinically meaningful and durable improvements in mobility and spasticity for patients living with ARG1-D whom previously had few effective treatment options.

"We are pleased to expand the availability of Ravicti to patients in these three regions. This approval underscores our commitment to advancing rare disease care and ensuring that patients have access to the treatments they need."

We thank Biologix for the collaboration and their efforts to make ganaxolone available to patients with CDD in the region. With the commercial rights now returned to us, we look forward to pursuing regulatory approval and exploring new opportunities to ensure that patients in the MENA can access this important treatment

2024 was a transformative year for Immedica, marked by milestones that only a few years back were unimaginable to us. We took significant steps toward our goal of becoming a global leader in rare diseases. These efforts are not only reshaping our business—they are making a real difference in the lives of patients worldwide

Being named Top 2 of Sweden’s Best Workplaces is a testament to our efforts in creating an environment where people thrive, grow, and feel included. We are extremely proud of our company, which is made up of dedicated employees who contribute every day to building a strong and positive atmosphere for each other and in the end for the patients we serve

All our Therapeutic Areas hold equal importance, and our goal is to maximize their potential. This refined structure enables us to strengthen our performance and continue delivering meaningful impact for people living with rare diseases

The acquisition of Marinus represents a transformative step in Immedica’s journey to further strengthen our position as a leading rare disease company. By adding ZTALMY to our portfolio, we significantly strengthen our capabilities and expand our presence in the United States, marking a new chapter in our mission to deliver impactful therapies for underserved patient populations

This submission marks a significant step toward bringing this important treatment addition to patients with UCD in Japan. We are pleased to see this progress together with our partner OrphanPacific, as we continue our commitment to addressing unmet medical needs in rare diseases.

I am excited to join Immedica at such a pivotal stage in the company’s growth trajectory. Immedica has already established itself as a leader in the rare disease space, and I look forward to leveraging my experience to support the company's ambitious goals of bringing novel therapies to underserved patient populations across the world.

We are pleased that the FDA has accepted the re-submitted BLA of pegzilarginase and granted priority review. This is a significant step towards providing a much-needed treatment option for patients with ARG1-D in the U.S. For this rare and debilitating condition there are limited therapeutic options, and we are hopeful that pegzilarginase, if approved, could offer a valuable alternative for managing the disease. We look forward to continuing to work with the FDA to potentially bring this treatment to those in need.

This study provides crucial insights into the challenges faced by ARG1-D patients and their families, reaffirming the need for continued innovation. As we expand our health economic function, we are focused on generating and analyzing relevant data to support the value of our products. This growing capability allows us to translate these findings into actionable steps that inform better clinical practices, improve healthcare decisions, and ultimately enhance the lives of those impacted by rare diseases

"We are thrilled to have Joe on board. His vast industry knowledge and successful track record in strategic deal-making will be key as we continue to drive growth and expand Immedica’s footprint in the rare disease and specialty care sectors."

This collaboration with KKR and Impilo marks an exciting step forward in our mission to deliver innovative treatments to more patients with rare diseases. With their support, we are well-positioned to enhance our growth and continue addressing critical unmet medical needs across the globe

The approval of Loargys for patients with ARG1-D is addressing a high unmet medical need in this disease, providing the first disease-modifying treatment. However, Immedica is committed to serve all patients with the disease and to investigate the ability to treat patients as early as possible, before any effects of the disease in the crucial first years of life, a particularly important period of child development

I’m excited to join Immedica at this pivotal time and lead the development of a U.S. commercial organization. I look forward to working closely with our European colleagues and U.S. employees to develop Immedica into a global leader in rare disease

This expansion into the U.S. is a pivotal moment for Immedica as we continue our mission to deliver life-changing therapies to people living with rare diseases

These data are of high importance for Loargys as a new treatment option in arginase 1 deficiency providing long-term and real-world outcomes data

With exceptional entrepreneurship, solid functional skill sets and capabilities fostered by a strong ‘can-do’ attitude, the team have time after time proven that we can overcome any obstacle together and achieve great things.

The Immedica team is one of the best that we have encountered in a space that we have been following for some time. We are deeply impressed by the accomplishments of Immedica under Impilo’s ownership and the establishment of a rare disease player with a highly promising pipeline. We look forward to working with the management team alongside Impilo to accelerate growth further

KKR, with its experience in rare disease and specialty pharma in Europe and the U.S., as well as its global presence and network, is an ideal strategic partner for the next stage of Immedica’s evolution. We are excited to continue our engagement in Immedica, together with KKR, supporting management to realize our joint vision of becoming a leading ultra-rare disease platform and making medicines available to patients with high unmet medical needs

I am excited to continue our partnership with Impilo and welcome KKR as a new strategic partner for the next step in Immedica’s journey, Since our inception in 2018 backed by Impilo, Immedica has made significant strides towards becoming a leader in the rare disease sector, providing innovative therapies to patients with severe unmet medical needs. I look forward to working alongside both KKR and Impilo as we further our mission, continue to build our organisation, and expand our reach globally"

"We are extremely proud to be recognized as one of Sweden's Best Workplaces™ by Great Place to Work®. It is a testament to our commitment to creating an inclusive and enjoyable work environment for all our employees,"

We raise awareness for Rare Disease Day, since it’s crucial to address the widespread issue of underdiagnosis and misdiagnosis, notably in conditions like ARG1-D. These rare diseases often remain overlooked, leaving patients and families to confront their challenges alone.

"This is a very important approval for the people living with ARG1-D and their families across the whole of United Kingdom and proof of the huge unmet medical need in this ultra rare disease. We will now focus on getting Loargys available to patients in the UK as soon as possible."

Today’s approval marks a major milestone for Immedica, but an even greater one for the people living with ARG1-D and their families.

Today’s announcement demonstrates significant progress towards providing the first disease modifying treatment for ARG1-D. The unmet medical need in this vulnerable patient population is huge and we look forward to the decision by the European Commission

“This approval by ANMAT is an important milestone for the people living with UCDs in Argentina and addresses the significant unmet medical need in this patient population”

We are very pleased with this new agreement with Aeglea, giving us the global rights to pegzilarginase. People with arginase 1 deficiency lack sufficient treatment options today and we believe that pegzilarginase, where awarded marketing authorizations, has the potential to benefit these patients as the first disease modifying treatment