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Orphan Drugs: The Rising Stars Of 3Q 2013

November 28, 2013

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 Q3 2013, July – September 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 Q3 2013 :

Launched or Receiving Approval

There are two orphan products in this category:

•   Gilotrif for Non-Small Cell Lung Cancer (NSCLC – Boehringer Ingelheim)
•   Adempas for Chronic Thromboembolic Pulmonary Hypertension (Bayer).

The first orphan product in this category is Gilotrif (Afatinib). Gilotrif is the first FDA-approved oncology product from Boehringer Ingelheim (BI) and provides a personalized treatment approach for patients. The drug receives approval as a new first-line treatment for patients with metastatic NSCLC with common Epidermal Growth Factor Receptor (EGFR) mutations. The EGFR mutation is detected by an FDA-approved test. Patients must have biomarker testing to determine if a common EGFR mutation is present. BI collaborates with Qiagen, a Netherlands holding company specializing in sample and assay technologies, to develop a companion diagnostic for Gilotrif.

The following are the regulatory actions for Gilotrif:

•   Receives FDA Orphan Drug Designation (ODD) in December 2012
•   Receives FDA approval in July 2013 & is evaluated under its Priority Review program
•   Receives European approval in September 2013.

The second orphan product in this category is Adempas (Riociguat). The FDA approves Adempas in October 2013 to treat adults for two forms of Pulmonary Hypertension (PH):

•   Pulmonary Arterial Hypertension (PAH)
•   Chronic Thromboembolic Pulmonary Hypertension (CTEPH).

Adempas is the first drug to receive approval for the treatment of CTEPH and is the 10th treatment for PH that the FDA approves. Also, in October 2013, the FDA approves Actelion Pharmaceuticals’ orphan drug Opsumit (Macitentan) for PAH. Opsumit is the 11th PAH treatment approved by the FDA. Both drugs are the first in 5 years to be approved by the FDA for PAH.

A patient with PAH has high blood pressure in arteries that supply the lungs; the muscles in the walls of the arteries thicken and the arteries in the lungs get narrower. This results in difficulty of the blood flowing to the lungs. PAH is debilitating and life-threatening, shortening the life of a patient due to breathing problems and possible heart failure. There is currently no cure for PAH. PH is a rare and life-threatening lung disease affecting approximately 20,000 – 30,000 patients.

Entering Phase II Trials

There are two orphan products in this category:

•   Sel-G1 for vaso-occlusive in sickle cell anemia (Selexys Pharmaceuticals)
•   NSI-566 for Amyotrophic Lateral Sclerosis (ALS – Neuralstem).

The first orphan product in this category is Selexys Pharmaceuticals’ Sel-G1, which is being developed for the intravenous treatment of vaso-occlusive in patients with sickle cell anemia. There is currently no cure for sickle cell anemia. Sickle cell anemia is an inherited blood disorder that mostly affects people of African ancestry. Per the company’s website :

“Patients with sickle cell disease suffer anemia as well as vaso-occlusive complications in which sickled red cells, white blood cells, and platelets adhere to one another and to small blood vessels blocking blood flow to downstream organs … results in intense pain crises and repeated hospitalizations.”

Sel-G1 prevents various cells in the bloodstream from sticking together. Selexys Pharmaceuticals is currently conducting a Phase II clinical trial (SUSTAIN study) to assess safety and impact of Sel-G1 in sickle cell disease patients with pain crises. Sel-G1 has orphan drug designation in both the United States and Europe.

The second orphan product in this category is Neuralstem’s NSI-566 for the treatment of ALS. NSI-566, a human spinal cord stem cell (HSSC) therapy, receives FDA orphan drug designation in February 2011. Neuralstem developed the “Spinal Cord Delivery Platform and Floating Cannula” for the purpose of delivering “stem cells intraspinally for the first time worldwide and has licensed the novel delivery device to industry and academia.” To treat ALS, Neuralstem isolates:

“and expands human neural stem cells from the spine and delivers them directly to the grey matter of the spinal cord, where they are expected to integrate into the host tissue and help establish new signaling pathways and express neuroprotective growth facctors, thus protecting existing neurons from further damage.”

A Phase II US trial is initiated in September 2013 in patients with ALS. An excellent overview of Neuralstem’s ALS Treatment and the Phase II trial is published in a recent Forbes online article.

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

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

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