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Norgine Expands Rare Disease Portfolio with Strategic Acquisition of Theravia

  • Norgine has entered a definitive agreement to acquire Theravia from Mérieux Equity Partners, adding key rare disease treatments SIKLOS® for sickle cell disease and ORPHACOL® for genetic bile disorders to its portfolio.

  • The acquisition aligns with Norgine's growth strategy, creating a European-based rare disease champion with six core products that will serve as key growth drivers in the medium-to-long term.

  • This transaction follows Norgine's recent deals with Fennec Pharma for PEDMARQSI® and X4 Pharma for mavorixafor, establishing the company as a partner of choice for commercializing rare and specialty pharmaceuticals in Europe.

Norgine and Theravia announced today that they have entered into a definitive agreement under which Norgine will acquire Theravia, an international pharmaceutical company specializing in treatments for patients with rare and debilitating conditions. The acquisition from Mérieux Equity Partners represents a significant advancement in Norgine's strategy for sustainable growth in the rare disease space.
The transaction will add Theravia's complementary portfolio of rare disease medicines to Norgine's existing offerings, further establishing the company's position as a preferred partner for commercializing rare and specialty pharmaceuticals across Europe.

Strategic Portfolio Expansion

Theravia brings several established products in the rare disease space to Norgine's portfolio, including SIKLOS®, used to treat adults and children with sickle cell disease, and ORPHACOL®, a medicine for patients with genetic disorders affecting bile production by the liver. These products span both rare hematology and rare hepatology therapeutic areas, complementing Norgine's existing rare and specialty portfolio.
"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 of 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."
With this acquisition, Norgine will now have six core products in its rare disease portfolio: PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS®, and ORPHACOL®. This expanded franchise achieves a critical scale that positions it as a key growth driver for the company in the medium-to-long term.

Building a Rare Disease Champion

The development of Theravia has been supported by its majority shareholder Mérieux Equity Partners, a healthcare-dedicated investment firm based in France. The merger brings together complementary expertise and geographical presence that will strengthen Norgine's position in the rare disease market.
Franck Hamalian, Chief Executive Officer of Theravia, commented on the acquisition: "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."

Expanding Therapeutic Reach

The acquisition adds significant depth to Norgine's rare disease capabilities:
SIKLOS® is currently marketed in 17 countries worldwide and is used to prevent recurrent, painful vaso-occlusive crises in patients with sickle cell syndrome. These crises occur when blood vessels become blocked by abnormal red blood cells, restricting blood flow to organs and potentially leading to acute chest syndrome, a life-threatening condition.
ORPHACOL®, available in 23 countries globally, contains cholic acid and is used to treat patients with genetic abnormalities that prevent them from producing bile. The therapy addresses a critical need for patients who lack specific liver enzymes necessary for producing primary bile acids, preventing liver damage and potential liver failure.

Building on Recent Strategic Moves

This acquisition follows Norgine's recent deals that demonstrate its commitment to expanding in the rare disease and specialty medicine sectors:
In January 2025, Norgine partnered with X4 Pharma for mavorixafor, a selective CXCR4 receptor antagonist approved in the U.S. for WHIM syndrome, a rare primary immunodeficiency. X4 has submitted a Marketing Authorization Application to the European Medicines Agency, with Norgine set to handle commercialization in Europe, Australia, and New Zealand following regulatory approvals.
Norgine has also secured rights to PEDMARQSI®, a novel formulation developed to prevent cisplatin-induced hearing loss in pediatric cancer patients. The medicine received marketing authorization from the European Medicines Agency in May 2023 and from the UK's MHRA in October 2023.

Looking to Future Growth

The transaction remains subject to obtaining customary regulatory approvals from competent authorities. Norgine has expressed its intention to build on this acquisition as it continues to strengthen its platform for future growth opportunities.
"With our integrated approach – strong commercial capabilities, deep medical and regulatory expertise, in-house manufacturing, and robust supply networks – we are well-positioned to deliver high-quality, transformative medicines to patients with rare diseases across our markets," added van der Kamp.
The acquisition aligns with Norgine's 120-year legacy of innovation and patient-focused care, as the company continues to expand its reach in delivering specialized treatments to the more than 25 million patients it serves annually across Western Europe, Australia, and New Zealand.
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