Orphan Drugs: The Power Of Multiple Indications
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|
|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|
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:
|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|
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)|
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