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FDA Breakthrough Therapy Designation: Recent Developments

March 31, 2014

This Blog Post reviews recent developments for the FDA Breakthrough Therapy Designation (BTD).

I – Syndax Pharmaceuticals

Syndax Pharmaceuticals, a small Massachusetts-based biopharmaceutical company, is developing and commercializing its lead candidate, Etinostat, an epigenetic therapy for treatment-resistant cancer. In September 2013, Etinostat, an oral, highly selective Histone Deacetylase (HDAC) inhibitor, receives the FDA BTD for the treatment of “.. locally recurrent or metastatic Estrogen Receptor-Positive (ER+) breast cancer when added to Exemestane in postmenopausal women whose disease has progressed following non-steroidal aromatase inhibitor therapy”.

Just last week, Syndax Pharmaceuticals files a registration statement S-1 with the US Securities and Exchange Commission (SEC) related to a proposed Initial Public Offering (IPO) of shares of its common stock. The company plans to trade under “SNDX” on the NASDAQ.  The number of shares to be offered or the price range of the shares have not been identified yet.

II – Novartis

Novartis’ Acute Heart Failure (AHF) drug, Serelaxin (RLX030), receives the FDA BTD in June 2013. In late January of this year, the European Medicines Agency’s CHMP (Committee for Medicinal Products for Human Use) adopts a negative opinion for the marketing authorization for Serelaxin. Novartis responds by announcing that the company will submit a revised filing package, including new data analyses, for the re-examination for a conditional marketing authorization in the EU.

Novartis’ Serelaxin receives another blow last week, when the FDA’s Cardiovascular and Renal Drugs Advisory Committee votes 11-0 against approval of the Biologics License Application (BLA). The Advisory Committee questions the design of the company’s 1,161-patient pivotal trial and Serelaxin’s efficacy. The FDA is not required to follow the Advisory Committee’s recommendations (usually the agency does). The FDA’s final decision is expected by May 17th. Please reference Novartis’ response.

Another Novartis drug in the news is LDK3768 (Ceritinib), which receives the BTD in March 2013 for ALK+ Non-Small Cell Lung Cancer (NSCLC). The Phase I study results published in the New England Journal of Medicine shows that Ceritinib achieves :

•   Overall Response Rate (ORR) of 58%

•   Median Progression-Free survival of 7 months.

III – Provectus Biopharmaceuticals

Provectus Biopharmaceuticals, a Knoxville, Tennessee company focusing on oncology and dermatology, last week announces that the company submits an application to the FDA for BTD for PV-10, for the treatment of Melanoma.

IV – New England Journal of Medicine Article

On March 27th, an article titled, “New FDA Breakthrough-Drug Category – Implications for Patients” is published in the NEJM. The Orphan Druganaut Blog will leave it to the reader to draw their own conclusions about the article.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

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

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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