On February 5th it was press released that Vertex Pharmaceuticals received a CRL from the FDA. A Food and Drug Administration CRL is issued when the FDA communicates to a company that an application to market a drug (NDA, new drug application) will not be approved in it's present form, and that the review by the FDA has been complete.
Vertex submitted a supplemental new drug application (sNDA) for the already approved Kalydeco. The sNDA was filed for Cystic Fibrosis people 2 years and older, who have one of 23 residual function mutations in the cystic fibrosis gene. Eight of the 23 were represented in a phase 2A study. It is estimated that over 1,500 people in the U.S. have one of the 23 gene mutations. The numbers do not seem all that large. But if you factor in that Kalydeco is priced around $300k for annual therapy, then the numbers become quite large, up to around $450mn in potential annual revenue for the company if the sNDA was approved.
The 23 residual function mutations included in the sNDA were: 2789+5G- > A, 3849+10kbC- > T, 3272-26A- > G, 711+3A- > G, E56K, P67L, R74W, D110E, D110H, R117C, L206W, R347H, R352Q, A455E, D579G, E831X, S945L, S977F, F1052V, R1070W, F1074L, D1152H, and D1270N. The press release is here Vertex Complete Response Letter. More than likely the CRL was issued to Vertex because only eight of the 23 residual function mutations were represented in the phase 2A study. Thank you for reading.
Vertex submitted a supplemental new drug application (sNDA) for the already approved Kalydeco. The sNDA was filed for Cystic Fibrosis people 2 years and older, who have one of 23 residual function mutations in the cystic fibrosis gene. Eight of the 23 were represented in a phase 2A study. It is estimated that over 1,500 people in the U.S. have one of the 23 gene mutations. The numbers do not seem all that large. But if you factor in that Kalydeco is priced around $300k for annual therapy, then the numbers become quite large, up to around $450mn in potential annual revenue for the company if the sNDA was approved.
The 23 residual function mutations included in the sNDA were: 2789+5G- > A, 3849+10kbC- > T, 3272-26A- > G, 711+3A- > G, E56K, P67L, R74W, D110E, D110H, R117C, L206W, R347H, R352Q, A455E, D579G, E831X, S945L, S977F, F1052V, R1070W, F1074L, D1152H, and D1270N. The press release is here Vertex Complete Response Letter. More than likely the CRL was issued to Vertex because only eight of the 23 residual function mutations were represented in the phase 2A study. Thank you for reading.
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