Families of Spinal Muscular Atrophy (Libertyville, IL) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted Orphan Drug Designation to Quinazoline495 for the treatment of Spinal Muscular Atrophy.
This is the first time a new therapy specifically designed for Spinal Muscular Atrophy has ever reached the important stage of being awarded orphan drug status by the FDA. Reaching this key milestone for the first time is a significant step forward for the entire SMA community and signifies the rapid progress being made to develop an effective treatment for this terrible disease.
This is a demonstration of the resolve and power of an orphan disease community to come together to raise funds to advance high-risk research programs. Families of SMA has invested over $13 million in this specific program during the last 9 years. The organization relies on its volunteer chapters and families to raise funds to support the research programs that the organization conducts.
SMA is the leading genetic killer of children under the age of two. SMA is typically marked by the degeneration of muscle movement including the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA.
The US Orphan Drug Act is intended to assist and encourage the development of safe and effective therapies for the treatment of rare diseases and disorders. In addition to providing a seven-year term of market exclusivity upon final FDA approval, orphan drug designation also provides advantages through a wide range of financial and regulatory benefits.
"We are extremely pleased that the FDA has awarded orphan drug status to this promising drug for the treatment of SMA," said FSMA Research Director Jill Jarecki, 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.