With Blood and Growth Disorder Bids Panning Out, Novo Nordisk Plots Path to Rare Disease Leadership: Exec
Novo Nordisk has been a key player in the rare disease space for over 45 years, albeit often overshadowed by its flagship diabetes and chronic disease operations. Recently, the company has expressed a resolute commitment to enhance its standing in rare diseases, particularly in blood and growth disorders, according to Ludovic Helfgott, the company’s EVP of rare disease. As Novo Nordisk gears up for transformative drug launches and progresses with clinical trials, including those targeting sickle cell disease (SCD), the company aims to significantly improve its market position and therapeutic offerings in this niche area.
Shifting Priorities Under New Leadership
The driving force behind Novo Nordisk’s enhanced focus on rare diseases can be traced back to the appointment of CEO Lars Fruergaard Jørgensen in 2017. Under Jørgensen’s leadership, the company recognized the potential of its rare disease unit, which Helfgott refers to as “an uncut gem.” When Helfgott joined the company from AstraZeneca in 2019, his mission was clear: to foster a “biotech within Novo Nordisk” focused on innovative therapeutic solutions for rare disorders.
The evolution of the rare disease unit marks a shift from previous neglect, with substantial investment in research and development (R&D) capabilities reflecting this renewed focus. The establishment of a $1.2 billion rare disease production facility in Denmark symbolizes Novo’s commitment to refining its R&D prowess in the rare disease sector.
Expanding Research and Acquisitions
To drive its rare disease ambitions forward, Novo has shifted its internal R&D strategy from an infrequent endeavor to a robust program. Under Helfgott’s leadership, the early research team focused on rare diseases has expanded from eight to approximately 90 members. This internal growth is essential for the company to identify and evaluate potential high-value assets in the rare disease space effectively.
A prime example of Novo’s strategic moves is the acquisition of Forma Therapeutics in 2022 for $1.1 billion, which fortified its portfolio in sickle cell disease. Further, the company’s continued investment in external partnerships, such as the acquisition of 2seventy bio’s hemophilia A program in 2024, showcases its intent to broaden its capabilities in rare disease treatments.
Product Launches and Innovation
Novo’s approach to rare diseases has thus far concentrated primarily on blood and endocrine disorders. The company has successfully introduced several treatments for hemophilia A and B, including Esperoct, NovoEight, and Rebinyn. Most recently, the approval of their once-daily hemophilia injection, Alhemo, is a noteworthy achievement. This tissue factor pathway inhibitor (TFPI) antagonist is tailored for patients over the age of 12 who experience complications from factor inhibitors, representing a new treatment modality in the hematology space.
Helfgott emphasizes the importance of medical education in the U.S. surrounding Alhemo’s launch. The drug’s convenient subcutaneous delivery method, which diverges from traditional infusion therapies, positions it favorably compared to existing hemophilia treatments.
Future Horizons: Sickle Cell Disease and Beyond
As Novo looks to extend its leadership in rare diseases, it has set its sights on SCD, with etavopivat, developed from the Forma Therapeutics acquisition, transitioning into phase 3 trials. This candidate aims to address the critical issues faced by SCD patients—specifically painful vaso-occlusive crises (VOCs) and long-term organ damage caused by recurrent episodes. Positive preliminary findings from phase 2 studies indicate that etavopivat can potentially reduce annual VOC rates significantly, validating its promising therapeutic profile.
Novo Nordisk’s commitment to rare diseases remains steadfast, with an ongoing focus on blood and growth disorders, including an expanding interest in the emerging “hemato-renal area”—a niche that connects rare blood and kidney disorders. The successful FDA approval of Rivfloza, an RNA interference drug for the treatment of primary hyperoxaluria type 1 (PH1), exemplifies Novo’s traction in this domain.
Strategic Growth through M&A and Collaborations
Novo Nordisk is not shying away from mergers and acquisitions to bolster its rare disease portfolio. The company’s external collaborations, including a notable agreement with NanoVation Therapeutics to utilize innovative lipid nanoparticle technology for two base-editing therapies, reflect its strategy to access new modalities and elevate research capabilities in rare genetic diseases. Such strategic partnerships are crucial for deepening Novo’s understanding and solutions in rare and cardiometabolic conditions.
Conclusion
As Novo Nordisk directs its efforts toward becoming a leader in the rare disease landscape, the company is laying a strong foundation through investment in R&D, strategic acquisitions, and innovative product launches. Their commitment not just to blood and growth disorders, but also to pioneering avenues in the hemato-renal space, positions Novo as a formidable player that investors should watch closely. As drug launches materialize, and more research data becomes available, the company’s trajectory will be pivotal in reshaping its reputation from chronic disease management to a recognized authority in rare therapeutic options.
