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FDA Breakthrough Therapy Designation: Ariad Pharmaceticals Denied

August 8, 2013

A FDA Breakthrough Therapy Designation (BTD) that is denied, is publicly disclosed by Ariad Pharmaceuticals at their August 7, 2013, Q2 2013 Earnings conference call.  Ariad Pharmaceuticals is a global biotechnology company headquartered in Massachusetts and Switzerland, focusing on the development of treatments for cancer.

Ariad Pharmaceuticals discloses that the FDA denied BTD for the company’s AP26113 for the treatment of Anaplastic Lymphoma Kinase-positive (ALK+) Non-Small Cell Lung Cancer (NSCLC). According to the company, the BTD is denied because of the “relatively short follow-up” and the small number of ALK+ patients. Ariad Pharmaceuticals submitted to the FDA, data from only 24 evaluable patients in the Phase I portion of a Phase I/II trial, which resulted in 14 partial responses and 1 complete response in a Xalkori-naïve patient. Timonthy P. Clackson, Chief Scientific Officer and President of Research & Development at Ariad Pharmaceuticals says that:

“ … We continue to move expeditiously in the development of 113 (AP26113) with a pivotal trial in ALK-positive lung cancer patients to begin as planned this quarter, and we believe this decision by the agency (FDA) will have no impact on our ability to pursue our ambitious development plans and timeline. ALK+ Non-Small Cell Lung Cancer, including in brain metastasis in these patients, and we are pursuing this opportunity with great focus …”.

Ariad Pharmaceuticals did say that they can refile for the FDA BTD, but the company is not saying whether or not it will refile, and when it would refile. A clinical update on the Phase I/II trial data of AP26113 will be presented at the September 2013 annual meeting of the European Society of Medical Oncology (ESMO).

The FDA  grants Novartis in March 2013, BTD for their compound LDK378 for the treatment of  patients with ALK+ metastatic NSCLC, who have progressed during treatment with, or were intolerant to Pfizer’s orphan drug Xalkori (Crizotinib). The FDA designation is based on positive early data in patients with ALK+ NSCLC who have previously been treated with Crizotinib. Patients with ALK+ NSCLC have few treatment options.

The FDA has a “Frequently Asked Questions: Breakthrough Therapies” section on their website where the latest statistics on the FDA CDER Breakthrough Therapy Designations are published and updated as of July 26, 2013 :

CDER Breakthrough     Therapy (BT) Designation Category

# of   CDER BT   Designations (10/01/12-07/26/13)

Total       # of BT Requests Received


Total       # of BTs Granted


Total #     of   BTs Denied


Total       # of BTs Pending


Seventeen (17) Breakthrough Therapy Designations have been identified by the sponsor companies as of July 26, 2013.  With 25 total Breakthrough Therapy Designations granted, there are 8 that have not been identified by the sponsor company.

The FDA doesn’t disclose information regarding sponsors who submitted requests for or who have been granted or denied breakthrough therapy designations. Per the FDA website,  “Breakthrough therapy designation requests are typically submitted to an IND, and the FDA cannot disclose the existence of an IND, or any submissions that have been submitted to the IND, unless it has previously been publicly disclosed or acknowledged per 21 CFR 312.130(a).”

References Ariad Pharmaceuticals Management Discusses Q2 2013 Results – Earnings Call Transcript

Please Note: FDA Official Logo from FDA website.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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