The US Food and Drug Administration (FDA) has granted Lucentis (ranibizumab), a vascular endothelial growth factor (VEGF) inhibitor, its Breakthrough Therapy (BT) designation for the treatment of diabetic retinopathy.
Diabetic retinopathy is a vision-threatening complication of type 1 and type 2 diabetes. It is the most common diabetic eye disease and a leading cause of vision loss in working adults, affecting over 93 million people worldwide. Diabetic macular edema is a form of diabetic retinopathy.
The FDA based the BT designation on results of the RISE and RIDE phase 3 trials. In these trials, meaningful improvements in disease were observed in a clinically significant proportion of diabetic retinopathy patients treated with ranibizumab at 2 years compared with patients treated with sham (control group). Benefits of ranibizumab were maintained during year 3 of treatment, and the safety in the RISE and RIDE phase 3 trials was consistent with previous studies.
The BT designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or placebo if there is no available therapy. The designation includes all of the FDA’s fast-track program features, as well as more intensive guidance and discussion. The BT designation is distinct from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.
Lucentis is currently indicated for diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion. If approved, Lucentis will be the first ocular medication approved for the treatment of diabetic retinopathy.