Norgine Expands Rare Disease Portfolio by Acquiring Theravia

Norgine, a specialty pharmaceutical firm, has made a significant move in its growth strategy by acquiring Theravia, a company focused on developing treatments for rare and severe diseases. This acquisition enhances Norgine's position as a leader in the European market for rare and specialty pharmaceuticals.

Announced on April 15, 2025, the agreement between Norgine and Theravia marks a pivotal step for both companies. Norgine is set to acquire Theravia, which has established itself as a key player in the pharmaceutical field with innovative solutions for rare conditions. The acquisition aligns with Norgine’s strategy to expand its portfolio of rare disease treatments, having previously collaborated with Fennec Pharma and X4 Pharma on PEDMARQSI® and mavorixafor, respectively.

Theravia's portfolio includes crucial medications such as SIKLOS®, designed for individuals with sickle cell disease, and ORPHACOL®, which aids those with a genetic disorder affecting bile production. These treatments address rare haematology and hepatology conditions, complementing Norgine’s existing offerings. Theravia's recent advancement was supported by Mérieux Equity Partners, a dedicated healthcare investment firm.

Janneke van der Kamp, CEO of Norgine, expressed enthusiasm about the acquisition, viewing it as an opportunity to enhance the company’s growth trajectory and expand its rare disease offerings. She emphasized Norgine’s proven ability to deliver innovative treatments to patients, anticipating that Theravia's medicines will reach their full potential under Norgine’s guidance.

Franck Hamalian, CEO of Theravia, shared a positive outlook on the merger, highlighting that the combined expertise and geographical reach of the two companies would create a formidable European entity in the realm of rare diseases. The collaboration is expected to improve access to vital treatments for patients with high unmet medical needs.

With the inclusion of Theravia's products, Norgine's rare disease portfolio now encompasses six core treatments: PEDMARQSI®, eflornithine, mavorixafor, AGILUS®, SIKLOS®, and ORPHACOL®. This expanded portfolio positions Norgine as a significant player in the rare disease market, poised to drive growth in the medium to long term.

Norgine anticipates that the acquisition will strengthen its platform for future growth through additional acquisitions and in-licensing opportunities. However, the transaction is still pending regulatory approvals from relevant authorities.

Theravia, formed from the merger of Addmedica and CTRS, specializes in addressing neglected diseases and has made significant contributions to the field. SIKLOS®, one of its products, is pivotal in managing sickle-cell syndrome by preventing painful vaso-occlusive crises. ORPHACOL® helps patients with genetic abnormalities in bile production, preventing potential liver damage. Both drugs are marketed globally, making crucial impacts on patient outcomes.

PEDMARQSI®, another innovative treatment, addresses cisplatin-induced hearing loss in young cancer patients, marking a significant development in pediatric oncology care. Meanwhile, mavorixafor targets WHIM syndrome, a rare immunodeficiency, with ongoing developments for other conditions like chronic neutropenia.

Eflornithine, also in Norgine’s pipeline, is under investigation for its use in neuroblastoma, a pediatric cancer, with applications submitted for approval across several regions. AGILUS®, approved for treating malignant hyperthermia, further enriches Norgine's therapeutic offerings.

Overall, the acquisition of Theravia by Norgine represents a strategic expansion that strengthens its position in the rare disease market and reinforces its commitment to providing transformative healthcare solutions across Europe and beyond.