Norgine strengthens rare disease portfolio with acquisition of Theravia

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines

Transaction further establishes Norgine's position as a partner of choice for commercialising rare and specialty pharmaceuticals in Europe

PARIS, April 15, 2025 /PRNewswire/ -- Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specialising in cutting-edge treatments for patients with rare and debilitating conditions.

Recent deals with Fennec Pharma for PEDMARQSI^® and X4 Pharma for mavorixafor demonstrate Norgine's strong emphasis on driving growth in rare disease and specialty medicines through acquisitions and in-licensing.

Theravia's innovations have resulted in successful launches of life-saving medications which have changed patient outcomes worldwide. Theravia has several products in the rare disease space, including SIKLOS^®, for adults and children with sickle cell disease, and ORPHACOL^®, a medicine for adults and children who have a genetic disorder that affects bile production by the liver. These products span both the rare haematology and rare hepatology therapeutic areas and are complementary to Norgine's existing rare and specialty portfolio. The development of Theravia in the past few years has been supported by its majority shareholder Mérieux Equity Partners, a healthcare dedicated investment firm based in France.

"This acquisition is a unique opportunity for Norgine to bolster our growth trajectory, as well as our rare disease portfolio." said Janneke van der Kamp, Chief Executive Officer, Norgine. "With our strong legacy and proven track record of successfully bringing innovative treatments to patients, we believe we are well placed to ensure the Theravia medicines reach their full potential."

Franck Hamalian, Chief Executive Officer, Theravia, added: "The joining of our companies brings together the skills, know-how and geographical presence that will create a European-based champion of rare and ultra rare disease. Our combined product portfolios and Norgine's legacy and experience will enable broader access to medicines for patients with high unmet medical needs."

With the acquisition of Theravia, Norgine will now have six core products in its rare disease portfolio (PEDMARQSI^®, eflornithine, mavorixafor, AGILUS^®, SIKLOS^® and ORPHACOL^®) thereby comprising a franchise of critical scale with the ability to be a key growth driver in the medium-to-long-term.

Norgine looks forward to building on this transaction as we continue to strengthen our platform for future acquisitions and in-licensing opportunities to drive growth.

The transaction remains subject to obtaining customary regulatory approvals from the competent authorities.

Notes

About Theravia
Theravia is an international pharmaceutical laboratory specializing in rare or neglected diseases. Formed through the merger of Addmedica and CTRS, Theravia is dedicated to addressing the unmet medical needs of patients with these challenging conditions.

About SIKLOS^®
SIKLOS^® is used in adults, adolescents and children over two years of age who have sickle-cell syndrome, a genetic disease where the red blood cells become rigid and sticky and change from being disc-shaped to being crescent-shaped (like a sickle). It is used to prevent recurrent, painful vaso-occlusive crises that happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to an organ. They can include acute chest syndrome, a life-threatening condition when the patient has sudden chest pain, fever, hard breathing or signs of fluid in the lungs on an X-ray.

SIKLOS^® is marketed in 17 countries around the world.

About ORPHACOL^®
ORPHACOL^® is a medicine containing cholic acid, a substance found in the bile, which is used to digest fats. It is approved in the US and Europe used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. The therapy is used in patients who do not have enough of two specific liver enzymes which makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure.

ORPHACOL^® is marketed in 23 countries around the world.

About PEDMARQSI^®
PEDMARQSI^® is a novel formulation of anhydrous sodium thiosulfate, specifically developed and manufactured for the prevention of cisplatin-induced hearing loss in patients 1 month to <18 years of age. It is the first and only preventative treatment developed for cisplatin-induced ototoxicity to support children and adolescent patients with localised, non-metastatic solid tumour cancers.

It was granted marketing authorisation by the EMA in May 2023 (under the paediatric-use marketing authorisation (PUMA) programme) and the MHRA in October 2023.

About Mavorixafor
Mavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI^®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome, a rare primary immunodeficiency.

In January 2025, X4 submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor for the treatment of WHIM syndrome, for which it has been granted Orphan Drug Designation by both the EMA and the U.S. Food and Drug Administration.

X4 is also developing the medicine to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders. Norgine has responsibility for commercialisation in Europe, Australia, and New Zealand following regulatory approvals.

About Eflornithine
Eflornithine has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy. It is a targeted therapy that blocks an enzyme called ornithine decarboxylase (ODC), responsible for producing polyamines, which are important to tumour growth and development.

The medicine is approved by the FDA for a maintenance therapy for high-risk neuroblastoma in adult and pediatric patients. In April 2024, an application for approval was submitted by Norgine for the use of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. In January 2025, a marketing authorisation application was filed to the European Medicines Agency (EMA) for the medicine in high-risk neuroblastoma (HRNB).

About AGILUS®
AGILUS^® is a medicine used to treat malignant hyperthermia (rapid rise in body temperature caused by uncontrolled muscle contractions) in adults and children. Malignant hyperthermia is a serious reaction to certain medicines used for general anaesthesia during surgery or other medical procedures.

AGILUS^® is approved for use by the EMA for for the treatment of malignant hyperthermia in adults and children in combination with adequate support measures.

About Mérieux Equity Partners
Founded in 2009, Mérieux Equity Partners ("MxEP") is a leading European healthcare-specialized investment firm, with two dedicated platforms, Venture Capital and Buyout, supporting companies ranging from start-ups to established leaders. Benefiting from a longstanding expertise and a large network, MxEP invests in companies with ambitious growth projects and transformative products or services in healthcare. MxEP is AMF-accredited and currently manages c.€1.5bn of AuM.

About Norgine
Norgine is a uniquely positioned, specialty pharmaceutical and consumer healthcare company, with over €550 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across our core markets of Western Europe, Australia and New Zealand.

Norgine's integrated approach – strong commercial capabilities, as well as deep medical, regulatory and clinical expertise, in-house manufacturing, robust supply networks, and best-in-class enabling functions – ensures that Norgine can deliver high-quality, transformative medicines quickly and effectively to over 25 million patients annually.

Norgine is a nimble, innovative, and high-performing company that has been transformed by a relentless focus on operational excellence. This focus will enable us to secure the legacy of more than a century of innovation and doing the right thing by our patients, as we push the boundaries and take strides into therapeutic innovation.

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