| FDA APPROVES NEW ORAL CHELATOR |
Picture Courtesy of www.thalassemia.org |
Food and Drug Administration approve the oral iron chelator Exjade for the treatment of transfusional iron overload.
"This is a tremendous step forward for people with thalassemia," said Gina Cioffi, Esq., National Executive Director of the Cooley's Anemia Foundation, the non-profit organization
that is the voice of the thalassemia community in the United States.
Individuals with thalassemia, often called Cooley's anemia, require lifelong blood transfusions as often as every two weeks. As a result
of these transfusions, iron accumulates in and causes severe damage to their organs.
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Cioffi explained that "at the moment, the only FDA-approved method of removing that dangerous excess iron is through subcutaneous infusion of the iron chelator deferoxamine, commonly known by the brand name Desferal.
Patients are required to stick a needle in themselves and pump deferoxamine into their bodies for up to 12 hours, almost every night. The process is burdensome for many patients and has proven to have a pronounced negative
impact on the effectiveness of the drug resulting in increased life-altering complications. "Desferal is a good drug, but its method of delivery creates obstacles that are insurmountable for a large portion of our patient population," Cioffi continued. "We think that the availability of an oral chelator
can make a meaningful difference in both the quality and quantity of life for those patients."
Cioffi testified in favor of Exjade at the Blood Products Advisory Committee meeting, along with Gargi Pahuja, JD, MPH, the President of the Thalassemia Action Group (TAG), CAF's patient- run support group. They were joined in their testimony by several other CAF representatives, including patients and family members of patients.
CAF representatives detailed the effects of transfusional iron overload in the thalassemia community, emphasizing how it overwhelms the heart and liver, leading to premature and painful death.
"For decades, the thalassemia community has been told that an oral chelator was coming," said Pahuja, who was born with thalassemia. "The Committee's decision today means that an oral chelator is at last within reach for US patients. On behalf of all thalassemia patients, I encourage the FDA to follow the Committee's lead and make Exjade available
to patients whose lives depend upon an alternative treatment."
"We hope that approval of Exjade is a beginning, not an end," Pahuja added. "Each thalassemia patient has his or her own specific needs. To meet those needs, doctors should have available several chelating options, so that treatment can be tailored in the most effective way possible."
The FDA is expected to respond to the Committee's recommendation within a short period of time. In most instances, the FDA agrees with the findings of such committees, which are comprised of individuals with expertise in the subject at hand.
Thalassemia is a fatal genetic blood disorder that disproportionately affects people of Mediterranean, Northern African, Middle Eastern, South Asian, Chinese and Southeast Asian descent. There is at this time no cure for thalassemia, and the life expectancy for patients, even with treatment, is significantly below the national average. Individuals with thalassemia are susceptible to a wide range of complications, including heart and liver failure, diabetes, osteoporosis and blood borne infections such as hepatitis and West Nile Virus.
Founded in 1954, the Cooley's Anemia Foundation is the only national non-profit dedicated to fighting the genetic blood disorder thalassemia, also known as Cooley's anemia. The Foundation provides import patient services, funds crucial medical research and educates health care professionals, government officials and the general public about thalassemia.
For more information, contact CAF at 800-522-7222 or info@cooleysanemia.org, or visit www.cooleysanemia.org
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