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Orphan Drugs: The Rising Stars

February 25, 2014

Thomson Reuters publishes a quarterly report, “The Ones to Watch”, that identifies and analyzes the “most promising drugs”, or what the Orphan Druganaut Blog calls “the rising stars”. The latest report published this month, is for the Q4 2013, October – December 2013.

The report reviews the drugs by the following four categories or phases of development :

•   Launched or receiving approval

•   Entering Phase III trials

•   Entering Phase II trials

•   Entering Phase I trials.

The following orphan products from the report identifies the most promising under these categories from the Q4 2013.

Launched or Receiving Approval

There are three orphan products in this category:

•    Gazyva for previously untreated Chronic Lymphocytic Leukemia (CLL) (Genentech)

•    Valchlor (Meclorethamine) for Stage IA/IB Mycosis Fungoides-Type Cutaneous T-Cell Lymphoma (Ceptaris Therapeutics)

•    Opsumit (Macitentan) for Pulmonary Arterial Hypertension (PAH) (Actelion Pharmaceuticals).

The first orphan product in this category is Genentech’s Gazyva, which is the first FDA Breakthrough Therapy Designation (BTD) to receive approval (November 2013).  Gazyva receives FDA approval for use in combination with Chlorambucil (chemotherapy), to treat patients with previously untreated Chronic Lymphocytic Leukemia (CLL). The approval comes with a boxed warning regarding the Hepatitis B virus and a rare disorder that damages the material that covers and protects nerves in the white matter of the brain (Progressive Multifocal Leukoencephalopathy). The approval of Gazyva comes  a month before the scheduled PDUFA (Prescription Drug User Fee Act) Date in December 2013, and is launched during the Q4 2013. Gazyva also has Orphan Drug Designation (ODD) in Europe and is being developed for other oncology indications. According to data from Cortellis Competitive Intelligence, Gazyva is “ … expected to generate $166 million in global revenue in 2014, rising to $1.1 billion by 2017.”

The second orphan product in this category is Ceptaris Therapeutics’ (subsidiary of Actelion Pharmaceuticals) topical gel formulation, Valchlor, for Stage IA/IB Mycosis Fungoides-Type Cutaneous T-Cell Lymphoma (CTCL). Valchlor also receives ODD in Europe. The drug is launched during the Q4 2013. According to data from Cortellis Competitive Intelligence: “ … global sales of Valchlor could reach $96 million a year by 2019.”

The third orphan product in this category is Actelion Pharmaceuticals’ oral, once daily Opsumit, approved for PAH, to delay disease progression. During Q4 2013, Opsumit is launched in the United States and receives EU approval for PAH. According to data from Cortellis Competitive Intelligence: “ .. global sales of Opsumit will reach approximately $1.1 billion a year by 2017 ..”.

Entering Phase III Trials

The only orphan product in this category is Infinity Pharmaceuticals’ Duvelisib which enters Phase III clinical trials in December 2013 for :

•   Chronic Lymphocytic Leukemia (CLL)

•   Small Lymphocytic Lymphoma (SLL).

Orphan drug designation is received in April 2013 for both indications in the EU and the US. The drug is also being investigated for inflammatory disorders – rheumatoid arthritis and asthma.

Entering Phase II or I Trials

There are no orphan products under these two categories.

Please Note : “Umbrella with 12 Spokes & Stars” by StromBer (Own work) [CC-BY-3.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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