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Week of 02/25/13 Orphan Drug Events

February 25, 2013

I – Ariad Pharmaceuticals’ 4th Quarter & Year-End 2012 Financial Results

Ariad Pharmaceuticals will host a live webcast of its 4th Quarter and Year-End 2012 Financial Results conference call, on Monday, February 25, 2013, 8:30 AM EST. Investors will be watching to see what is reported for orphan drug Iclusig’s sales.

In December 2012, the FDA following priority review, gives Accelerated Approval of Ariad Pharmaceuticals’ orphan drug Iclusig (Ponatinib) for the treatment of Chronic Myeloid Leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic Leukemia (Ph+ALL). This approval came 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. The approval provides CML patients with another option if they are not responding to other drugs.

Iclusig’s Orphan Drug Competitors Approved Of By The FDA In 2012 :

  1. Pfizer’s orphan drug Bosulif (Bosutinib) for CML
  2. Teva Pharmaceutical’s orphan drug Synribo (Omacetaxine Mepesuccinate) for CML
  3. Talon Therapeutics’ orphan drug Marqibo for Ph+ALL.

II – 6th Annual International Rare Disease Day 2013

Rare Disease Day is scheduled for February 28, 2013. This is the 6th year that it has been celebrated. This year’s slogan is “Rare Disorders Without Borders”, which supports cross-border cooperation for rare diseases affecting small populations in each country.

Social Media tools are being used to promote and create buzz about Rare Disease Day 2013 :

Twitter page

Facebook page

YouTube channel

Flickr gallery.

Rare Disease Day logo, banner, and an information pack are available for download.

Additional information on Rare Disease Day can be found through these organizations’ links :

RARE Project

III – Stivarga (Regorafenib)

The FDA PDUFA date for Bayer and Onyx Pharmaceuticals’ orphan drug Stivarga (Regorafenib), for the treatment of Gastrointestinal Stromal Tumors (GIST), is February 28, 2013. The drug in Phase III clinical trials, produces a Progression-Free Survival (PFS) of 4.8 months, compared with 0.9 months for the control arm. Stivarga receives FDA Orphan Drug Designation (ODD) in January 2011, for GIST.

If Stivarga receives FDA approval this week, it will be the second indication for the drug. Approval is given in September 2012 as a secondary treatment for metastatic colorectal cancer.

Generic    Name Trade    Name Sponsor Indication PDUFA    Date
Regorafenib Stivarga Bayer/Onyx Pharma. GastroIntestinal   Stromal Tumors (GIST) 2/28/13

Please note the links for the following 2 chart columns above :

1) “Generic Name” Column Link = Is a source for the FDA PDUFA Date

2) “Indication” Column Link = Is the FDA Orphan Drug Product Designation Database Link.

Rare Disease Day JPG from Rare Disease Day Organization.

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

  1. teresascruton permalink

    We are now seeing the first round of insurance blowback. Insurance companies are severely limiting the amount of orphan drugs that the patient can access in a calendar year, or are refusing to authorize payment for drugs previously approved and utilized in the prior calendar year.
    This action is catastrophic in the orphan disease community, for people that rely on these drugs to stay alive, and to maintain some semblance of a quality-of-life.

    • Hi Teresa – Thanks for visiting the Orphan Druganaut Blog. Yes, I agree with you 100% that the insurance companies & government medical programs are cutting back for orphan drugs because of the high price. Some companies are limiting the coverage as you pointed out per calendar year or calendar month or limited the drug to # of doses or cycles. That is why it is important for patients & families and advocacy groups to get the word out there !! Thanks again for stopping by.

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