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Orphan Drugs And The Myelodysplastic Syndrome Market

February 7, 2013

Orphan drugs are key players in the Myelodysplastic Syndromes (MDS) drug market. Drug therapy is just one of many methods physicians use to treat MDS. Three single-drug approaches are approved by the US FDA for treatment of MDS.

MDS has recently been in the news when the US Good Morning America anchor, Robin Roberts, announces in June 2012 that she is fighting MDS. Ms. Roberts believes that her breast cancer treatments several years ago may have led to the development of MDS.

MDS is a group of blood disorders associated with low blood counts.  In patients with MDS, the cells in the bone marrow don’t function normally and not enough normal blood cells are produced. This rare disease  usually occurs in older people, typically starting after age 50 and is rarely inherited. MDS increases the risk of infection, bleeding, and problems related to anemia. It can occur on its own or 1 – 15 years after being exposed to certain forms of chemotherapy or radiation.  For many people, it develops without any known cause.

There is currently only one curative treatment for MDS – allogeneic hematopoietic stem cell transplant (HSCT). 75% of patients are not eligible for HSCT due to poor health and age.

According to  a Decision Resources Report, Vidaza is the leading therapeutic option for MDS in both the US and the EU. Between 2004 – 2006, the US MDS market grows when the first drug therapy options receive approval :

  1. Vidaza (Azacitidine) Injection from Celgene
  2. Dacogen (Decitabine) IV from Eisai/MGI Pharma
  3. Revlimid (Lenalidomide) from Celgene.

Here are the FDA Orphan Drug Product Designation Database records for these 3 MDS orphan drugs currently on the market in the US :

Row Num Generic Name/Brand Name Marketing Approval Date Indication
1                                                                          Decitabine/Dacogen 05.02.06 MDS
2 Azacitidine/Vidaza 05.19.04 MDS
3 Lenalidomide/Revlimid 12.27.05 MDS

According to a article, Celgene’s Revlimid, which has 2 FDA-approved indications (MDS and Multiple Myeloma), accounts for 66% of the company’s sales and is forecast to grow between 9% – 12% per year.  The total sales for 2012 is $3.767 billion, an increase of 17%.  Also, Revlimid has 3 FDA Orphan Drug Designations (ODDs) for :

  1. Chronic Lymphocytic Leukemia
  2. Diffuse Large B-Cell Lymphoma
  3. Mantle Cell Lymphoma.

Celgene’s Vidaza, which has FDA approval for MDS and FDA ODD for Acute Myeloid Leukemia, has 2012 total sales of $823 million, an increase of 17%.

Eisai’s Dacogen has FDA approval for MDS and 3 FDA ODDs for :

  1. Chronic Myelogenous Leukemia
  2. Acute Myeloid Leukemia
  3. Sickle Cell Anemia.

Since 2006 in the US, no new drugs have received FDA approval for MDS. Only Vidaza is approved for MDS in Europe. The Report believes that EU approval for Revlimid is just a matter of time. The Report goes on to say that it expects the MDS therapeutic options to improve with the approval of 5 new drugs between 2014 – 2018.


Celgene Webcast Presentation at BIO CEO & Investor Conference – Monday, February 11, 2013 8:00 AM ET

Myelodysplastic Syndromes (MDS) Foundation

Aplastic Anemia & MDS (AAMDS) International Foundation

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

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