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Vertex Pharmaceuticals: Another FDA Orphan Drug Designation For Cystic Fibrosis

April 26, 2014

On April 24th, Vertex Pharmaceuticals’ investigational drug VX-661 ((R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide) receives FDA Orphan Drug Designation (ODD) for the treatment of Cystic Fibrosis (CF) :

FDA Orphan Drug Designation Database Record
Generic Name: (R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide
Trade Name: n/a
Date Designated: 04-24-2014
Orphan Designation: Treatment of cystic fibrosis
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Vertex Pharmaceuticals Inc. 50 Northern Avenue Boston, MA 02210-1862 The sponsor address listed is the last reported by the sponsor to OOPD.


VX-661 is a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) corrector. VX-661 is being studied in combination with Kalydeco (Ivacaftor) for patients who have the F508del mutation. VX-661 is currently recruiting participants for a Phase II clinical trial to evaluate the safety and efficacy of VX-661 in combination with Kalydeco in subjects with CF who are homozygous (have 2 copies) for the F508del CFTR mutation. Vertex Pharmaceuticals has worldwide rights to develop and commercialize VX-661.

The new FDA ODD continues Vertex Pharmaceutials’ growth of the Kalydeco franchise. To treat the most common form of CF (F508del mutation), which accounts for approximately 40% of the total CF population, the company is investigating the use of the:

•   CFTR potentiator Kalydeco in combination with the CFTR corrector VX-809 (Lumacaftor)

•   CFTR potentiator Kalydeco in combination with the CFTR corrector VX-661.

Kalydeco receives 2 BTDs at the beginning of 2013, as a monotherapy and in combination with Lumacaftor (VX-809). In January 2014, a 3rd BTD is granted for Kalydeco in combination with VX-661 for CF patients who have two copies of the F508del mutation.

Three Kalydeco FDA BTDs

Row Num Drug Name
1 Kalydeco
2 Kalydeco + Lumacaftor (VX-809)
3 Kalydeco + VX-661




FDA BTD Approval Chart 

FDA BTD Statistics Chart.

Please Note: “Chemlab2” by Tahoenathan (Own work) [CC-BY-SA-3.0] | via Wikimedia Commons.

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