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Orphan Drugs: 2 New FDA Designations

October 24, 2014

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

•   Amyotrophic Lateral Sclerosis (ALS)

•   Retinitis Pigmentosa (RP).

I – Mayo Clinic And ALS

On October 22nd, the FDA grants the Mayo Clinic in Rochester, Minnesota, ODD for ALS.

ALS, also known as Lou Gehrig’s Disease, is a fatal progressive neurodegenerative disease. ALS destroys basic movements, such as breathing, and typically causes death within 2-5 years after diagnosis.

FDA ODD Database Record For Mayo Clinic’s ALS Indication

Generic Name: Autologous adipose derived mesenchymal stromal cells
Trade Name: n/a
Date Designated: 10-22-2014
Orphan Designation: Amyotrophic Lateral Sclerosis
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Mayo Clinic 200 First Street SW Rochester, MN 55905


II – Gene Therapy For RP

On October 20th, the FDA grants RetroSense Therapeutics, an Ann Arbor, Michigan, biotechnology company ODD for the company’s gene therapy for Retinitis Pipmentosa (RP). RetroSense Therapeutics is developing gene therapy to restore functional vision in patients suffering from blindness due to RP and advanced dry-AMD. There are currently no FDA approved therapies to improve or restore vision in patients with these retinal conditions.

Retinitis pigmentosa (RP) is a group of inherited genetic disorders characterized by progressive peripheral vision loss and night vision difficulties followed by eventual central vision loss and blindness in many cases. RP is typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies from person to person. Approximately 100,000 people living in the US and between 0.03% and 0.04% of the global population suffer from RP.

RetroSense Therapeutics’ lead gene therapy product candidate, RST-001, is being developed initially for RP. RST-001 is a gene therapy that delivers a photosensitivity gene (channelrhodopsin-2) to retinal cells, to restore the ability of eyes to sense light. RST-001 is an injection into the eye, where channelrhodopsin-2 is encapsulated in a virus vector that delivers the gene. Once the gene is delivered to the retinal cell, it begins to produce light-sensitive protein.

FDA ODD Database Record For RetroSense Therapeutics’ RP Indication

Generic Name: Non-replicating recombinant adeno-associated virus vector containing a fragment of the gene encoding channelrhodopsin-2 protein
Trade Name: n/a
Date Designated: 10-20-2014
Orphan Designation: Treatment of Retinitis Pigmentosa
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: RetroSense Therapeutics, LLC 330 East Liberty Street, LL Ann Arbor, MI 48104


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

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