With Blood and Growth Disorder Bids Panning Out, Novo Nordisk Plots Path to Rare Disease Leadership
Novo Nordisk has been a significant player in the field of rare diseases for over 45 years. However, this sector has often taken a back seat to the company’s more mainstream endeavors in treating diabetes and other chronic conditions. But the company is shifting gears under the leadership of Ludovic Helfgott, the Executive Vice President for Rare Disease, who recently outlined the company’s plans and ambitions in an interview with Fierce Pharma.
Shifting Focus to Rare Diseases
Novo Nordisk’s renewed focus on rare diseases particularly gained momentum when Lars Fruergaard Jørgensen became CEO in 2017. Helfgott notes that Jørgensen saw potential in the rare disease unit, referring to it as an “uncut gem.” When Helfgott joined the company from AstraZeneca in 2019, he embarked on a mission to establish a “biotech within Novo Nordisk,” aiming for substantial leadership in the rare disease segment. Under Helfgott, the company has made significant strides in enhancing its research capabilities, leading to a burgeoning workforce in rare disease research—from eight to roughly 90 employees.
Recent Developments and Strategic Acquisitions
Novo Nordisk’s journey into rare diseases has seen strategic investments, including a notable $1.2 billion commitment to build a production facility in Denmark dedicated explicitly to rare diseases. The company’s acquisitions have also grown in recent years; the $1.1 billion takeover of Forma Therapeutics, focusing on sickle cell disease, and the acquisition of 2seventy bio’s hemophilia A program in 2024 are key examples of this expansion.
The focus on blood disorders—specifically hemophilia A and B—has led to several successful treatments, including Esperoct, NovoEight, and Rebinyn. Recently, Novo received FDA approval for its once-daily hemophilia treatment, Alhemo. This innovative treatment has a unique subcutaneous delivery mechanism, enhancing patient accessibility compared to traditional infusions.
Innovative Solutions for Sickle Cell Disease
Beyond hemophilia, Novo’s forward-looking strategy includes a new candidate named etavopivat, which recently advanced into phase 3 testing for sickle cell disease (SCD). Helfgott emphasized that patients with SCD face the dual challenges of painful vaso-occlusive crises and organ damage from recurrent crises. The phase 2 data presented by Novo indicated that the highest studied dose of etavopivat significantly reduced annualized VOC rates, showcasing promising efficacy compared to placebo.
Key Areas of Development
As Novo prepares to solidify its standing in the rare disease arena, the primary areas of focus will remain on blood and growth disorders. The company is also exploring opportunities in the “hemato-renal area,” which addresses the intersection of rare blood and kidney conditions. Recently, Novo secured FDA approval for its RNA interference drug, Rivfloza, targeting primary hyperoxaluria type 1 (PH1), which causes the overproduction of oxalate and, ultimately, kidney damage.
A Future Focused on Strategic Partnerships
As the rarity of these diseases presents both challenges and opportunities, Novo is not ruling out mergers and acquisitions to enhance its competencies in blood, hemato-renal, and endocrine disorders. The company has already demonstrated its interest in establishing collaborations; for instance, a partnership with NanoVation Therapeutics for lipid nanoparticle technology targeted at rare genetic diseases serves as an illustration of how Novo plans to leverage external capabilities to further its drug development pipeline.
The Path Ahead
The ambitious roadmap laid out by Novo Nordisk signals a strategic pivot toward a more pivotal role in the rare disease sector. The company is not only seeking to refine its existing therapeutic offerings but also to continue expanding its capabilities through research, acquisitions, and partnerships.
In conclusion, as Novo Nordisk continues to bolster its rare disease initiatives, the firm aims to refine its focus on blood and growth disorders while exploring new opportunities in emerging areas. With consistent investment and innovation, Novo is well-positioned to become a prominent leader in the rare disease market, promising significant advancements for patients worldwide.