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Orphan Drugs: The Rising Stars

May 29, 2013

Thomson Reuters publishes a May 2013 report, “The Ones to Watch”, that identifies and analyzes drugs, from January – March 2013, that are the “most promising drugs” or what the Orphan Druganaut Blog calls “the rising stars”. “The Ones to Watch” Report is published quarterly. The review of the 1st Quarter 2013 drugs are organized by the following four categories :

•   Launched or receiving approval
•   Entering Phase III trials
•   Entering Phase II trials
•   Entering Phase I trials.

The Report identifies the most promising orphan drugs and drugs for rare diseases under these categories :

Launched or Receiving Approval

The orphan drug identified in this category is Exelixis’ Cometriq (Cabozantinib) for the treatment of Medullary Thyroid Cancer (MTC). Cometriq is approved by the FDA in November 2012, following a Fast-Tracked, rolling NDA filing under Orphan Drug and Priority Review Designations. The US prescribing information carries a boxed warning identifying the risks of gastrointestinal fistulas, and other side effects. Exelixis announces in January 2013 the commercial availability of Cometriq. US rights to Cometriq belong to Exelixis, with European commercialization rights belonging to Swedish Orphan Biovitrum (Sobi). The drug is under EU regulatory review for MTC. According to consensus data from Cortellis :

“ .. sales of Cometriq are expected to reach $9 million in 2013, rising to $150 million in 2017.”

Several online articles identify Exelixis as the BioTech to watch in 2013 because of the company’s plans on expanding Cometriq for future treatment of additional cancer indications :

•   Metastatic Castration-Resistant Prostate Cancer (mCRPC)
•   RCC
•   HCC.

According to a January 2013 SeekingAlpha.com article, the author believes that Cometriq will “ …. find limited success in MTC …. investors are most interested in its potential in the prostate cancer market ….”. COMET-1, is the Phase III trial for Cabozantinib for men with previously treated metastatic castration-resistant prostate cancer with bone-dominant disease. It is the data from this clinical trial according to the article, that is the catalyst the investors are waiting for to determine if the drug will make it in the prostate cancer drug market. The clinical trial is not to be completed until 2014.

Cometriq is to be featured in nine presentations at the 2013 American Society of Clinical Oncology (ASCO) Annual Meeting, May 31 – June 4, 2013.

Entering Phase III Trials

The orphan drug identified in this category is Vertex Pharmaceuticals‘ Kalydeco (Ivacaftor) in combination with Lumacaftor (VX-809) for the treatment of Cystic Fibrosis (CF). Kalydeco monotherapy is the first personalized medicine targeting a specific CF group – patients carrying the G551D mutation. In January 2013, the FDA grants this CF combination with a Breakthrough Therapy Designation.

In an April 2013 conference call, Vertex Pharmaceuticals discusses the Phase III study for VX-809 in combination with Kalydeco. The study consists of two 24-week Phase III studies in patients ages 12 and older with 2 copies of the most common mutation in the CFTR gene – F508del. Enrollment is currently underway. Nearly half (50%) of patients with CF have 2 copies of the F508del mutation. A February 26, 2013 press release provides additional information.

Entering Phase I Trials

In this category, three drugs for rare diseases are identified as the most promising drugs entering Phase I trials

•   Alnylam Pharmaceuticals’ drug ALN-TTRsc for Transthyretin Amyloidosis
•   Protalix Therapeutics’ drug PRX-112 for Gaucher’s Disease
•   BioCryst Pharmaceuticals’s drug BCX-4161 for Hereditary Angioedema (HAE).

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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