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Orphan Drugs: Two New FDA Designations

October 4, 2014

This Blog Post discusses two new FDA Orphan Drug Designations (ODDs) for the following two indications:

•   Glioma

•   Friedriech’s Ataxia.

Glioma

DNAtrix, Inc. is a biotech company that focuses on developing modified viruses for the treatment of aggressive forms of cancer. The company is working to modify a common cold virus so that it targets and kills cancer cells selectively. DNAtrix’s main product, DNX-2401, according to the company’s website is “the most potent and effective oncolytic virus delivered to human brain tumors to date.” DNX-2401 is injected directly into a patient’s brain tumor, selectively and efficiently invades tumor cells, and as tumor cells rupture, more DNX-2401, tumor antigens, and immune factors are released – with no harm to normal brain cells.

Gliomas are tumors that arise from glial cells and can occur in the spinal cord or brain (most common location). Gliomas are the most common type of brain tumor. Glioblastoma Multiforme (GBM) is the most common glioma to occur in adults, diagnosed at an average age of 55. Gliomas usually have a poor prognosis, with survival rates of about 30% at 1 year and 14% at 2 years. There is currently no approved drug to improve survival.

On October 1st, the FDA grants DNAtrix Inc., an ODD for Glioma:

Generic Name: Adenovirus delta 24-RGD, an oncolytic adenovirus genetically modified for treatment of glioma
Trade Name: n/a
Date Designated: 10-01-2014
Orphan Designation: Treatment of glioma
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: DNAtrix, Inc. 3210 Merryfield Row San Diego, CA 92121 The sponsor address listed is the last reported by the sponsor to OOPD.

.

Friedriech’s Ataxia

Horizon Pharma, a global specialty biopharmaceutical company, announces that their product Actimmune (Interferon Gamma-1b) receives FDA ODD for the rare, genetic disease, Friedriech’s Ataxia (FA):

Generic Name: Interferon gamma-1b
Trade Name: Actimmune
Date Designated: 10-01-2014
Orphan Designation: Treatment of Friedriech’s Ataxia
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Vidara Therapeutics Research Ltd. Adelaide Chambers Dublin 8 IRELAND ** The sponsor address listed is the last reported by the sponsor to OOPD.

** Horizon Pharma completed acquisition of Vidara Therapeutics in September 2014.

There are currently no approved treatments for FA. It is a degenerative neuro-muscular disorder that affects approximately 1/50,000 people in the United States. FA can start anywhere from childhood to adulthood. There is loss of muscle strength and coordination. Those patients diagnosed with FA at a young age require mobility aids, such as a wheelchair, walker, or cane by their teens or early 20’s.

Orphan drug Actimmune is currently approved in the United States for 2 indications:

•   Chronic Granulomatous Disease (GD) – genetic disorder that affects a type of white blood cell of the immune system

•   Delaying time to disease progression for Severe , Malignant Osteopetrosis (SMO) – genetic disorder that affects normal bone formation.

Please Note: “Chemicals in Flasks by Joe Sullivan (Flickr) [CC-BY-2.0] | Wikimedia Commons.

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

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