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Orphan Drugs: The Rising Stars

March 20, 2013

Thomson Reuters publishes a report, “The Ones to Watch”, that identifies and analyzes drugs, from October – December 2012, that are the “most promising drugs” or what the Orphan Druganaut Blog calls “the rising stars”. The review of the 4th Quarter 2013 drugs are organized by the following 4 categories, with the identification of 5 drugs/category :

  1. Launched or receiving approval
  2. Entering Phase III trials
  3. Entering Phase  II trials
  4. Entering Phase   I trials.

The Report identifies the following four orphan drugs under two of the categories :

Launched or Receiving Approval

The first orphan drug identified in this category is UniQure’s lead product Glybera (adeno-associated vector expressing the human lipoprotein lipase protein), for the treatment of LipoProtein Lipase Deficiency (LPLD). LPLD is caused by a mutation in the gene which codes lipoprotein lipase. This results in the loss of the ability to produce lipoprotein lipase enzymes necessary for effective breakdown of fatty acids. According to Reuters, it is estimated that Glybera will cost close to $1 million/patient when it launches in the Summer of 2013 in Europe. UniQure is a European company based in The Netherlands. Glybera is the first gene therapy to receive approval in the EU (November 2012). Glybera has orphan drug status in both the EU and the US, with regulatory filings planned for the US and Canada.

The second orphan drug identified in this category is Ariad Pharmaceuticals’ Iclusig (Ponatinib). In December 2012, the FDA following priority review, gives Accelerated Approval to Iclusig for the treatment of Chronic Myeloid Leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic Leukemia (Ph+ALL). This 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. In the EU, a Marketing Authorization Application (MAA) is filed in August 2012, where it is being evaluated under an accelerated review program. EU approval is expected in the third quarter 2013.

Entering Phase III Trials

The first orphan drug identified in this category is the Italian biopharmaceutical company Dompe Group’s Repertaxin (Reparixin), a selective interleukin-8 (IL-8) antagonist, under development to improve the efficacy of pancreatic islet transplantation in patients with T1D (Type 1 Diabetes). The drug has orphan drug status in both the US (September 2012) and the EU for this indication. A Phase III clinical trial is initiated in the 4th quarter 2012. Pancreatic islet cell transplantation is an experimental procedure where islets, or clusters of cells, are “taken from the pancreas of an organ donor, processed, and infused into the liver of the T1D recipient. Once transferred, the islets begin to produce insulin.” This  drug would help with the patient’s requirement for frequent insulin injections.    

The second orphan drug identified in this category is Lexicon Pharmaceuticals’  Telotristat Etiprate for the treatment of Carcinoid Syndrome – carcinoid tumors that commonly occur in the gastrointestinal tract. A Phase III clinical trial, TELESTAR, is initiated in October 2012. Both the US FDA and the EU EMA grants the drug orphan drug status, with Fast Track status in the US.

Please Note : “Umbrella with 12 Spokes & Stars” by StromBer (Own work) [CC-BY-3.0] | Wikimedia Commons.

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

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