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FDA Breakthrough Therapy: Pfizer’s Accelerated Approval And Merck’s Rescinding

February 5, 2015


This past week has been a busy week for the FDA’s Breakthrough Therapy Designation (BTD) with the granting of 3 new BTDs and the approval of a BTD for Waldenstrom’s Macroglobulinemia (WM).

This Blog Post reviews 2 additional FDA BTD activities:

•   Accelerated approval of Pfizer’s Ibrance (Palbociclib) for advanced breast cancer

•   Rescinding of Merck’s BTD for Hepatitis C.

I – Pfizer’s Accelerated Approval Of Ibrance (Palbociclib)

On February 3rd, Pfizer announces the FDA Accelerated Approval of Ibrance (Palbociclib), in combination with Letrozole. The BTD is for postmenopausal women with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) advanced breast cancer, as initial endocrine-based therapy for metastatic disease. Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial. The confirmatory Phase III clinical trial, PALOMA-2, is currently fully enrolled.

Ibrance is the first FDA-approved Cyclin-Dependent Kinase 4/5 (CDK 4/6) inhibitor. Per Pfizer’s news release, Ibrance is “available to order immediately through select specialty pharmacies.” Per an online article in Medical Marketing & Media (MM&M):

Ibrance is expected to cost around $9,850 per month, bringing the cost of a 17-month regimen to around $167,000. Leerink analyst Seamus Fernandez projected 2015 sales of around $150 million … and consensus estimates put peak sales at about $3.8 billion by 2020. Fernandez noted that his team sees a potential for an additional $10 billion market opportunity if Ibrance were to land indications for high-risk early-stage breast cancer and recurrent cancer.

Ibrance receives the FDA BTD in April 2013. The drug is reviewed and approved under the BTD and Priority Review programs. Pfizer’s 2 BTDs have received FDA Accelerated Approval:

Drug Name FDA Approval Date Indication
Trumenba (Meningococcal Group B Vaccine) 10.29.14 Meningococcal B Disease
Ibrance (Palbociclib) 02.03.15 ER+/HER2- Advanced Breast Cancer


II – FDA Rescinds Merck’s BTD For Hepatitis C

In an unusual move, Merck announces on February 4th, in the company’s 2014 earnings call, that the FDA rescinded its BTD for the Hepatitis C (HCV) drug combination of Grazoprevir/Elbasvir:

Merck recently announced that it expects to file a New Drug Application (NDA) with the FDA in the first half of 2015 for grazoprevir/elbasvir (MK-5172/MK-8742) … On Jan. 30, 2015, the company received notification from the FDA of its intent to rescind Breakthrough Therapy Designation status for this combination treatment regimen, citing the availability of other recently approved treatments for Genotype 1 patients. The company expects to discuss this matter with the FDA and does not expect that it will impact its ability to file an NDA for this combination regimen or the timing of that filing.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “IMG Carlingford Lough 0617” by Sarah777 at en.wikipedia [Public domain] | Wikimedia Commons.

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

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