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2 FDA Breakthrough Therapy Approvals: Vertex Pharmaceuticals’ Kalydeco And Regeneron Pharmaceuticals’ Eylea

March 25, 2015



This Blog post reviews 2 FDA Breakthrough Therapy Designations (BTDs) that are approved this past week:

•   Vertex Pharmaceuticals’ Kalydeco (Ivacaftor)

•   Regeneron Pharmaceuticals’ Eylea (Aflibercept) Injection.

I – Vertex Pharmaceuticals’ Kalydeco

On March 18th, Vertex Pharmaceuticals receives FDA approval for Kalydeco for children ages 2 – 5 with Cystic Fibrosis (CF), who have 1 of the following 10 mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene:

1) G551D                           6) S1251N

2) G1244E                         7) S1255P

3) G1349D                         8) S549N

4) G178R                           9) S549R

5) G551S                          10) R117H.

Before last week’s approval, Kalydeco is approved in the US for patients ages 6+, with these 10 mutations. An additional 300 US children can now take Kalydeco. To meet the needs of this young age group, a “new weight-based oral granule formulation of Kalydeco (50 mg & 75 mg) that can be mixed in soft foods or liquids was created … ”. This allows children who may not be able to swallow a tablet to take Kalydeco.

II – Regeneron Pharmaceuticals’ Eylea

On March 25th, Regeneron Pharmaceuticals announces that the FDA approves Elyea injection for the treatment of Diabetic Retinopathy (DR) in patients with Diabetic Macular Edema (DME). Eylea is injected into the eye every month for 5 months, and then every 2 months.

In September 2014, Eylea receives the FDA BTD and Priority Review for this indication. Eylea is currently approved in the US, European Union (EU), and other countries, for the following indications:

•   Wet Age-Related Macular Degeneration (AMD)

•   Macular Edema following Central Retinal Vein Occlusion (CRVO)

•   Diabetic Macular Edema (DME)

•   Diabetic Retinopathy (DR) in patients with Diabetic Macular Edema (DME).

Eylea is a VEGF (Vascular Endothelial Growth Factor) inhibitor formulated as an injection for the eye. It blocks the growth of new blood vessels and also decreases the ability of fluid to pass through blood vessels in the eye.

Diabetic Retinopathy is a complication of diabetes that results in damaging the eye’s retina, which can lead to poor vision, vision loss, and other vision problems, such as:

“… DME, which refers to the swelling of the macula (the part of the retina responsible for central, fine vision) and progression to proliferative diabetic retinopathy, which often results in profound visual loss due to associated complications … DME is the most frequent cause of vision loss in patients with diabetes and eventually can lead to blindness”.

In February 2015, the FDA approved Genentech’s Lucentis (Ranibizumab), once-monthly injection, for the same indication – DR in patients with DME.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “White Pocket Reflection” by John Fowler (Flickr: White Pocket Reflection) [CC BY 2.0] | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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