Families Of Spinal Muscular Atrophy Receives FDA Orphan Drug Designation For Quinazoline495 For The Treatment Of Spinal Muscular Atrophy

Main Category: Muscular Dystrophy / ALS
Also Included In: Regulatory Affairs / Drug Approvals;  Clinical Trials / Drug Trials
Article Date: 29 Aug 2009 - 1:00 PDT

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Families of Spinal Muscular Atrophy (Libertyville, IL) announced 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.

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, the Orphan Drug Act facilitates a close working relationship between regulatory agencies and companies aimed at accelerating the drug development and approval processes for treatment of rare diseases. Since the Act was passed in 1983, the Food and Drug Administration (FDA) has approved more than 200 new orphan products.

About Families of Spinal Muscular Atrophy:

Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:

-- Funding and advancing a comprehensive research program;

-- Supporting SMA families through networking, information and services;

-- Improving care for all SMA patients;

-- Educating health professionals and the public about SMA;

-- Enlisting government support for SMA;

-- Embracing all touched by SMA in a caring community.

Our vision is a world where Spinal Muscular Atrophy is treatable and curable.

A small group of parents started Families of SMA in 1984. They wanted to raise funds for SMA research to cure the disease, and support all affected families. Back then, very little was known about Spinal Muscular Atrophy. Very little research was being conducted. No one knew the cause of the disease let alone how to find a treatment and a cure. There were no family support services and no clinical trials. Patients and families affected by SMA were on their own and had little hope.

Today, FSMA has a different story to tell. Families of SMA has created hope for the SMA community that did not exist in 1984. FSMA has raised and funded over $50 million for SMA research. Support comes from generous individual donations and numerous fundraising events held by volunteer families and our Chapters.

Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients:

-- Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA.

-- FSMA has directed and funded the leading new drug development program for a therapy specially designed to treat SMA.

-- Families of SMA is building a pipeline of drug discovery programs based on our investments in basic research.

-- FSMA has invested significant resources into alternative approaches that show promise to cure SMA.

-- In 2009 FSMA had over 900 Attendees at the Annual SMA Family and Professionals Conference.

Families of SMA is a non-profit, 501(c)3 tax exempt organization with 26 Chapters throughout the United States and over 65,000 members and supporters.

Source: Families of Spinal Muscular Atrophy