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Families of Spinal Muscular Atrophy Receives FDA Orphan Drug Designation For Quinazoline495 For The Treatment of Spinal Muscular Atrophy
Date:8/28/2009

cki, Ph.D. "Orphan Designation will allow us to utilize all the opportunities provided by the Orphan Drug Act, including working closely with the FDA Office of Orphan Products Development throughout clinical development. In preclinical studies, the drug has been shown to efficiently cross the blood brain barrier - a critical feature for a SMA drug - and prolong survival significantly in two different mouse models of SMA."

Families of SMA has now requested a pre-IND meeting with the FDA to review plans to begin clinical trials for this drug. This meeting will occur within the next two months.

For information on how to support FSMA's drug discovery programs please visit www.curesma.org

About SMA:

Spinal Muscular Atrophy (SMA) is an inherited motor neuron disease, which is often fatal in the most severe form. It results from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord that control the muscles used in crawling, walking, head and neck control, breathing, and swallowing. It is a relatively common "rare genetic disorder" inherited in autosomal recessive manner. Approximately 1 in 6000 babies born are affected, making it the leading genetic killer of infants. 1 in 40 people are genetic carriers, indicating approximately 7.5 million carriers in the United States.

About the Orphan Drug Act:

The Orphan Drug Act (P.L. 97-414) was signed into law on January 4, 1983. The Act provides incentives for pharmaceutical manufacturers to develop drugs, biotechnology products, and medical devices for the treatment of rare diseases and conditions. These products are commonly referred to as orphan products. Importantly, th
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SOURCE Families of Spinal Muscular Atrophy
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