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Orphan Drugs: The Rising Stars

May 30, 2014

Thomson Reuters publishes a quarterly report, “The Ones to Watch”, that identifies and analyzes the “most promising drugs”, or what the Orphan Druganaut Blog calls “the rising stars”. The latest report, published May 2014, is for the Q1 2014, January – March 2014.

The report reviews the drugs by the following four categories or phases of development:

•   Launched or receiving approval

•   Entering Phase III trials

•   Entering Phase II trials

•   Entering Phase I trials.

The following orphan products from the report identify the most promising under these categories from the Q1 2014.

Launched Or Receiving Approval

There are two orphan Products in this category:

•   Alprolix (Coagulation factor IX (recombinant), Fc fusion protein) for Hemophilia B (Biogenic Idec)

•   Defitelio (Defibrotide) for Veno Occlusive Disease (VOD) (Gentium).

The first orphan product in this category is Biogen Idec’s Alprolix for use in both children and adults with Hemophilia B. It is the first approved long-acting recombinant coagulation Factor IX concentrate for Hemophilia B. Alprolix is made up of the Factor IX molecule linked to a protein fragment Fc (found in antibodies), which makes the product last longer in circulation. This results in less frequent injections for patients.

Alprolix is approved in March 2014 in the US and Canada. The therapy receives Orphan Drug Designation (ODD) designation in the US. Alprolix is currently under review by regulatory authorities in Australia and Japan.

Hemophilia B, which affects approximately 4,000 people in the United States and 28,000 worldwide, is an inherited sex-linked blood clotting disorder affecting mostly males. It is caused by defects in the Factor IX gene.

According to the report, worldwide sales of Alprolix are expected to reach:

•   $121 million in 2015

•   $178 million in 2016

•   $216 million in 2017.

The second orphan product in this category is Gentium’s (subsidiary of Jazz Pharmaceuticals) Defitelio for the treatment of Veno Occlusive Disease (VOD).  VOD is a vascular system disorder that can be a result of high levels of chemotherapy given before stem-cell transplantation. This results in the endothelial lining of the liver being damaged causing clotting, vessel blockage, and liver failure. About 80% of patients with VOD die within 100 days of stem-cell transplantation if no treatment is given.

Defitelio has ODD in the US and the EU. In October 2013, Defitelio is approved in the EU under “exceptional circumstances” – requiring the setup of a patient registry to investigate long-term safety and health outcomes. In March 2014, the drug is launched in Austria and Germany. From 2014-2015, plans are to launch Defitelio in an additional 27 countries.

Also, in this category (but not an orphan drug), is Novartis’ Bexsero (Meningococcal Group B Vaccine [rDNA, component, adsorbed]), which receives in April 2014, the coveted FDA Breathrough Therapy Designation (BTD). In the UK, the Joint Committee on Vaccination and Immunization (JCVI) just recommended in March 2014 including Bexsero in the country’s National Immunization Program (NIP) for “routine use in infants from 2 months of age.”

Entering Phase III Trials

The only orphan product moving into Phase III trials is:

•   BMN-701 for Pompe Disease (BioMarin Pharmaceutical).

BioMarin Pharmaceutical is developing BMN-701 as an Enzyme Replacement Therapy (ERT) for late-onset Pompe Disease. ODD is given from both the FDA and the EMA.  A Phase III trial is initiated in March 2014 in the US with patients who had previously received Genzyme’s Lumizyme (Alglucosidase Alfa), which has been on the market for several years. According to the report, the estimated “global annual sales of the drug will reach $229 million by 2019”.

Also, in this category (but not an orphan drug), is Portola Pharmaceuticals’ Andexanet Alfa, which receives in November 2013 the FDA BTD. Andexanet Alfa is a recombinant Factor Xa inhibitor that reverses anticoagulant activity in patients treated with a Factor Xa inhibitor who experience uncontrolled bleeding. Portola Pharmaceuticals plans on following the FDA’s Accelerated Approval route for Andexanet Alfa, with the start of registration-enabling studies in 2014.

Entering Phase II OR I Trials

There are no orphan products under these two categories.

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

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

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