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Orphan Drugs: Recent Cancer Regulatory Events

July 7, 2013

According to a Report “Orphan Drugs in Development For Rare Diseases”, from the Pharmaceutical Research and Manufacturers of America (, more than 33% of orphan drugs in development are rare cancer disease drugs in late stages of the pipeline. This statistic is reinforced by the Thomas Reuters study that came out in the summer of 2012, that says that about 40% of orphan drugs are used to treat cancer, the largest rare disease category.

Recent cancer regulatory events for orphan drugs are discussed in this Blog Post.

I – Genentech And Obinutuzumab

Genentech announces in May 2013, that orphan drug Obinutuzumab (GA101) receives FDA Breakthrough Therapy Designation (#13) for Chronic Lymphocytic Leukemia (CLL) based on a Phase III study (CLL11), that shows the drug significantly reduces the risk of disease progression or death. The Phase III data was presented at the annual meeting of the American Society of Clinical Oncology (ASCO), May 31 – June 4, 2013, in Chicago. The study compares the combination of either Obinutuzumab or Rituxan (Rituximab) and Chlorambucil (chemotherapy), to Chlorambucil alone in patients with CLL. Obinutuzumab + Chlorambucil shows a :

“ … significant 86% reduction in the risk of disease progression, relapse, or death. Additionally, the length of time during which people lived without their disease worsening was more than doubled when compared to Chlorambucil alone.”

The FDA Breakthrough Therapy Designation is followed by a July 2, 2013 announcement from Genentech. The company announces that the FDA accepts the Biologics License Application (BLA) for Obinutuzumab and grants Priority Review for the drug for CLL based on the final Stage 1 data from the CLL11 study. The FDA confirms the action date of December 20, 2013. In the US, Genentech has opened an Expanded Access Program (EAP) to provide Obinutuzumab to CLL patients under certain circumstances while the company seeks regulatory approval. It is estimated that there will be about 5,000 deaths from CLL in 2013, in the US.

A marketing application is submitted to the European Medicines Association (EMA)  in April 2013. In April 2013, Pharmacyclics and Janssen Research & Development’s cancer therapy orphan drug Ibrutinib receives FDA Breakthrough Therapy Designation for CLL.

II – Ariad Pharmaceuticals And Iclusig

On March 22, 2013, Ariad Pharmaceuticals announces that EMA’s Committee for Human Medicinal Products (CHMP) adopts a positive opinion on the Marketing Authorization Application (MAA) for Iclusig (Ponatinib) for two indications :

  1. Chronic Myeloid Leukemia (CML)
  2. Philadelphia chromosome-positive acute lymphoblastic Leukemia (Ph+ALL).

This is followed by Ariad Pharmaceuticals’ announcement on July 2, 2013, that the European Commission (EC) has granted a marketing authorization for Iclusig as an orphan medicinal product for the same two indications.

The FDA, in December 2012, gives Accelerated Approval (following Priority Review)  for Iclusig for the treatment of CML and Ph+ALL. The FDA approval comes with a black box warning about the side effects of blood clots and liver toxicity. The approval comes more than 3 months before the FDA PDUFA date of March 27, 2013. ARIAD Pharmaceuticals’ drug Iclusig is designated as an orphan drug on May 14, 2013 in Australia.

Please Note: “Globe and Books” by AlphaZeta (Own work) [CC0] | via Wikimedia Commons.

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

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