Novo Nordisk Expands Rare Disease Focus, Aiming for Leadership in Blood and Growth Disorders

Novo Nordisk, the Danish pharmaceutical giant known for its diabetes treatments, is making significant strides in the rare disease space, with a particular focus on blood and growth disorders. The company's executive vice president of rare disease, Ludovic Helfgott, recently outlined Novo's ambitious plans to become a leader in this field, marking a strategic shift for the organization.

Novo's Rare Disease Strategy Takes Shape

Since CEO Lars Fruergaard Jørgensen took the helm in 2017, Novo Nordisk has been polishing what Helfgott calls an "uncut gem" within the company – its rare disease unit. The goal is to create "a biotech within Novo Nordisk," a vision that has led to substantial investments in research and development, as well as strategic acquisitions.

Helfgott, who joined Novo from AstraZeneca in 2019, has overseen a significant expansion of the company's rare disease research team, which has grown from just eight people to around 90. This internal growth has been complemented by external deals, including the $1.1 billion acquisition of Forma Therapeutics in 2022, which bolstered Novo's sickle cell disease pipeline.

Recent Approvals and Pipeline Progress

Novo Nordisk's efforts in rare diseases are beginning to bear fruit. In December, the company received FDA approval for Alhemo, a once-daily injection for hemophilia A or B patients with certain factor inhibitors. The drug's unique mode of action and convenient subcutaneous delivery are expected to be key selling points as Novo focuses on medical education to support the launch.

In the sickle cell disease arena, Novo has advanced etavopivat, acquired from Forma Therapeutics, into phase 3 trials. The drug showed promising results in phase 2, nearly halving patients' annualized vaso-occlusive crisis rates compared to placebo at the 52-week mark.

Expanding Beyond Blood Disorders

While blood disorders remain a core focus, Novo is also exploring the intersection of rare blood and kidney diseases, which it terms the "hemato-renal area." This expansion led to the FDA approval of Rivfloza in late 2023 for the treatment of primary hyperoxaluria type 1 (PH1), a rare disease causing kidney damage.

The company is not ruling out further acquisitions and partnerships to strengthen its position in blood, hemato-renal, and endocrine disorders. A recent $600 million collaboration with NanoVation Therapeutics exemplifies this strategy, giving Novo access to long-circulating lipid nanoparticle technology for developing base-editing therapies in rare genetic diseases.

As Novo Nordisk continues to build its rare disease portfolio, the pharmaceutical industry watches closely to see if this longstanding diabetes powerhouse can successfully diversify and establish itself as a leader in the competitive and complex world of rare disease therapeutics.