Skip to content

Orphan Drugs: The Power Of Multiple Indications

February 18, 2015

Orphan drug companies have used with tremendous success as part of their drug life cycle management, the strategy to extend the life of their product by devising a succession of multiple discrete indications for the orphan drug. This strategy is critical to the continued financial success for orphan drug companies, especially when facing the challenges of generic competition and the demise of the traditional blockbuster drug strategy. The advantage of using multiple indications is that it allows a company to progressively expand the drug’s market.

This Blog Post presents several biotechnology companies that have used the multiple indication strategy for orphan drugs successfully:

•   Alexion Pharmaceuticals’ Soliris

•   Celgene’s Revlimid

•   Novartis’ Gleevec.

I – Alexion Pharmaceuticals And Soliris

The best known example of an orphan drug having multiple indications and generating blockbuster profits is Alexion Pharmaceuticals’ Soliris (Eculizumab). Soliris is Alexion Pharmaceuticals only marketed product and the drug has multiple indications for several ultra-rare diseases. The company has shown a slow, steady, continuous, successful growth from Soliris, by expanding the Soliris franchise :

•   Adding other indications

•   Expanding its geographic presence and penetration.

Soliris is approved for treatment of the following ultra-rare diseases:

•   Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare genetic blood disorder

•   Atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder.

Since 2003, Soliris receives FDA Orphan Drug Designation (ODD) for the following indications:

Num FDA ODD Date/ Approval Date Indication
1 08-20-2003/ 03.16.2007 PNH
2 04-29-2009/ 09.23.2011 aHUS
3 10-18-2011 Shiga-Toxin producing Escherichia Coli Hemolytic Uremic   Syndrome (STEC-HUS)
4 06-24-2013 NeuroMyelitis Optica (NMO)
5 01-10-2014 Prevention of Delayed Graft Function (DGF)  after Renal Transplantation
6 06-13-2014 Myasthenia Gravis


Alexion Pharmaceuticals is not just relying on multiple indications, the company is also developing other rare disease drug candidates to lessen the dependence on Soliris for revenue and growth. Two of these rare disease drug candidates receive the FDA Breakthrough Therapy Designation (BTD) in 2013:

Num Drug Name Indication
1 Asfotase Alfa Hypophosphatasia (HPP)
2 Cyclic Pyranopterin Monophosphate (cPMP) Molybdenum CofactorDeficiency(MoCD) Type A


II – Celgene And Revlimid

Oncology is another indication where companies have used the strategy of multiple indications to expand the orphan drug revenue. Celgene has used this strategy successfully with Revlimid (Lenalidomide) to continue and expand the company’s oncology franchise. Revlimid receives FDA ODD for the following indications since 2004:

Num FDA ODD Date/Approval Date Indication
1 01-29-2004/ 12.27.2005 Myelodysplastic syndromes associated with a deletion 5 q cytogenetic abnormality with or without additional cytogenetic abnormalities
2 01-17-2007 Chronic lymphocytic leukemia (CLL)
3 04-27-2009/ 06-05-2013 Mantle cell lymphoma where disease has relapsed/progressed after 2 prior therapies, 1 of which included bortezomib
4 03-28-2011 Diffuse large B-cell lymphoma
5 09-13-2013 Follicular lymphoma


Celgene announces February 18th that the FDA expands the indication for Revlimid in combination with Dexamethasone to include patients with newly diagnosed Multiple Myeloma.

III – Novartis And Gleevec

Novartis is another example of a company making successful use of multiple indications for oncology indications, for the orphan drug Gleevec (Imatinib). The chart below shows Gleevec’s (Imatinib) FDA ODDs that receive FDA approval:

Row Num Generic Name FDA ODD Date/ Marketing Approval Date Approved Labeled Indication
1 Imatinib 01.31.01/05.10.01 Chronic Myeloid Leukemia (CML)
2a Imatinib Mesylate 11.01.01/02.01.02 Kit-positive Unresectable and/or Metastatic Malignant Gastrointestinal Stromal Tumors (GIST)
2b Imatinib Mesylate 11.01.01/12.19.08 Adjuvant Treatment Following Complete Resection Kit-Positive Gastronintestinal Tumor (GIST)
3 Imatinib Mesylate 08.25.05/10.19.06 Hypereosinophilic Syndrome and/or Chronic Eosinophilic Leukemia
4 Imatinib Mesylate 09.09.05/10.19.06 Aggressive Mastocytosis   without D816V c-kit mutation
5 Imatinib Mesylate 10.05.05/10.19.06 Myelodysplastic   /Myeloproliferative Diseases
6a Imatinib 10.11.05/10.19.06 Relapsed or   Refractory Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL)
6b Imatinib 10.11.05/01.25.13 Newly Diagnosed Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) with   Chemotherapy
7 Imatinib Mesylate 12.19.05/10.19.06 Dermatofibrosarcoma Protuberans (DFSP)


Please Note: “Marty Schmidt in the Himalayas” by Sdiangelo (Own work) [CC-BY-SA-4.0]  | Wikimedia Commons.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

  1. Claude permalink

    Very interesting article.

    My next question is how do you identify the new indications for an existing drug on the market?

Trackbacks & Pingbacks

  1. Orphan Drugs: The Power Of Multiple Indications | urddad-foundation-blogroll

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: