AstraZeneca has secured a win for its rare disease pipeline, with its Phase III trial of gefurulimab demonstrating the drug’s favourable efficacy and safety profile in patients with generalised myasthenia gravis (gMG).
During the study, a weekly dose of gefurulimab was found to offer clinically significant improvements to quality of life (QoL), as measured by each patient’s myasthenia gravis activities of daily living (MG-ADL) score after 26 weeks.
In a statement released on 24 July, AstraZeneca's rare disease business Alexion also noted that the therapy met all its secondary endpoints. However, the company is yet to disclose details.
Following the success of the PREVAIL trial (NCT05556096), the pharmaceutical giant is likely to apply for gefurulimab’s approval with the US Food and Drug Administration (FDA), which already granted orphan drug designation to the medication in 2023.
If approved, the subcutaneous complement C5 inhibitor will bolster AstraZeneca’s current gMG portfolio, which includes Soliris (eculizumab) and Ultomiris (ravulizimab), drugs that the company attained through its $39bn acquisition of Alexion Pharmaceuticals in 2021.
gMG market competition heats up
Positive results of the Phase III PREVAIL trial come at a key time in the gMG niche, as intravenous medications begin to feel the strain in this tumultuous market.
This phenomenon is mainly driven by approvals of more patient-centric formats such as UCB Pharma’s Rystiggo (rozanolixizumab) and Zilbrysq (zilucoplan), as well as Argenx’s Vyvgart (efgartigimod alfa), which are all approved for self-administration.
AstraZeneca is also facing tough competition from other key pharmaceutical players, with Johnson & Johnson’s FcRn therapy Imaavy (nipocalimab-aahu) forecasted to generate $5bn per year at its peak following its recent approval, according to the company.
By attaining market permissions for gefurulimab, the company will be able to maintain its footing in this lucrative and fast-growing industry, which is estimated to reach a value of $10.5bn by 2034 across the seven major markets (7MM: the US, France, Germany, Italy, Spain, the UK, and Japan), according to Clinical Trials Arena’s parent company, GlobalData.
If approved, GlobalData predicts sales of gefurulimab to reach $723m in 2031.
“The positive results from the PREVAIL Phase III trial demonstrate gefurulimab’s potential to offer rapid and sustained disease control for this patient population,” stated AstraZeneca rare disease CEO Marc Dunoyer.
The trial’s principal investigator, Kelly Gwanthmey, also noted that a C5 treatment option offering a once-weekly dosing pattern and self-administration “would offer patients greater convenience and independence in managing their condition, allowing users to gain more control over their therapy”.
