Sprifermin shows potential as first disease-modifying therapy for knee osteoarthritis

  • Hochberg MC & al.
  • JAMA
  • 08/10/2019

  • Miriam Davis, PhD
  • Clinical Essentials
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Takeaway

  • Intra-articular injections of sprifermin, a recombinant human fibroblast growth factor, increases femorotibial joint cartilage thickness in knee osteoarthritis (KOA) at the highest doses, but leaves pain, stiffness, and function unchanged.

Why this matters

  • If more evidence of benefit unfolds, sprifermin could become the first disease-modifying therapy for OA.

Study design

  • 5-year, dose-finding, multicenter phase 2 trial of 549 patients aged 40-85 years with symptomatic, radiographic KOA of Kellgren-Lawrence grade 2/3.
  • Participants were randomly assigned to 1 of 5 treatment groups:
    • 100 μg sprifermin every 6 or 12 months.
    • 30 μg sprifermin every 6 or 12 months.
    • Placebo every 6 months.
  • Each treatment episode consisted of weekly intra-articular injections over 3 weeks.
  • Primary outcome: change in total femorotibial joint cartilage thickness assessed by quantitative MRI at 2 years.
  • Funding: Merck KGaA.

Key results

  • Groups 1 and 2 were the only ones to show significant improvement in femorotibial joint cartilage thickness over placebo (0.05 mm [95% CI, 0.03-0.07 mm] and 0.04 mm [95% CI, 0.02-0.06 mm], respectively).
  • None of the groups showed improvement over placebo in Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) total scores, as well as WOMAC subscores for pain, stiffness, and function.

Limitations

  • Durability of results unknown.