`Body
`Oct 14, 2011, 15:10 ET from US. Food and Drug Administration
`
`FDA Logo. (PRNewsFoto/US. Food and Drug Administration (FDAli
`" Eaggfiook aTwitter @ Pinterest
`
`SILVER SPRING, Md., Oct. 14, 2011 /PRNewswire-USNewswire/ -— The US. Food and Drug Administration today approved Ferriprox (deferiprone) to
`treat patients with iron overload due to blood transfusions in patients with thalassemia, a genetic blood disorder that causes anemia, who had
`an inadequate response to prior chelation therapy.
`
`(Logo: http://photos.prnewswirecom/prn h/20090824/FDALOCO)
`
`Patients with thalassemia have excess iron in the body from the frequent blood transfusions (transfusionai iron overload), a condition that is
`
`serious and can be fatal. These patients also have a risk of developing liver disease, diabetes, arthritis, heart failure or an abnormal heart rhythm.
`
`The standard of care to treat transfusional iron overload is chelation therapy~ chemical agents that are used to remove heavy metals from the
`body. Ferriprox is intended for use when chelation therapy is inadequate.
`
`“Ferriprox represents the first new FDA-approved treatment for this disorder since 2005," said Richard Pazdur. MD, director of the Office of
`Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
`
`The safety and effectiveness of Ferriprox is based on an analysis of data from twelve clinical studies in 236 patients. Patients participating in the
`study did not respond to prior iron chelation therapy. Ferriprox was considered a successful treatment for patients who experienced at least a 20
`percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the patients in the study experienced at least a 20
`percent decrease in ferritin levels.
`
`The most common side effects seen in patients who received Ferriprox included nausea, vomiting, abdominal and joint pain, urine discoloration
`(chromaturia), a decrease in the number of white blood cells (neutropenia), and an increase in the level of a liver enzyme that may be indicative
`of tissue or liver damage at unsafe amounts,
`
`The most serious side effect seen in about two percent of patients treated with Ferriprox was the development of agranulocytosis, a serious and
`potentially life-threatening reduction in the number of granulocytes (a type of white blood cell that fights infection).
`
`The therapy is being approved under the FDA‘s accelerated approval program, designed to provide patients with earlier access to promising new
`drugs followed by further studies to confirm the drug‘s Clinical benefit.
`
`Apotex Tech.
`Ex. 2008
`
`Apotex Tech.
`Ex. 2008
`
`
`
`The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug
`
`has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than
`
`survival or irreversible morbidity (illness).
`
`ApoPharma has agreed to several post-marketing requirement and commitments. One commitment includes further study of the use of
`Ferriprox in patients with sickle cell disease who have transfusional iron overload.
`
`Earlier this year, the US. Department of Health and Human Services (HHS) launched the Sickle Cell Disease (SCD) Initiative bringing together
`
`HHS agencies to enhance the quality and quantity of SCD data, develop best practice guidelines and quality of care metrics, improve health care
`
`delivery and coordination of care for patients with SCD, facilitate approval of new medical products, and expand research on SCD. The post-
`
`marketing requirement for further study of Ferriprox aligns with the goals of the SCD Initiative.
`
`V
`
`Ferriprox is marketed by ApoPharma Inc. of Toronto
`
`For more information:
`
`FDA: Office of Hematology and Oncology Products
`
`http://www.fda,gov/AboutFDA/CentersOffices/CDER/ucm091745htm
`
`FDA: Spotlight on Drug Innovation — Update of FDA's novel drug approvals in 2011
`
`http://www.fda.gov/Drugs/lnformationOnDrugs/ApprovedDrugs/ucm254242.htm
`
`The FDA, an agency within the US. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness,
`and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is
`responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and
`for regulating tobacco products.
`
`Media Inquiries: Erica Jefferson, 301-796—4988, erica.jefferson@fda.hhs.gov
`Consumer Inquiries: 888-lNFO-FDA
`
`SOURCE US. Food and Drug Administration
`
`RELATED LINKS
`
`http://wwwfdagov/
`
`
`
`FDA Approves Ferriprox to Treat Patients with Excess Iron in the Body
`
`Page 1 of 4
`
`FDA Approves Ferriprox to Treat
`Patients with Excess Iron in the Body
`
`Oct 14, 2011, 15:10 ET from US. Food and Drug Administration
`
`
`
`FDA Logo. (.PRNewsFoto/U5. Food and Drug Administration (FDA))
`
`‘} Facebook OT__w_itter amPinterest
`
`SILVER SPRING, Md., Oct. 14, 2011 /PRNewswire—USNewswire/-— The US. Food and Drug
`
`Administration today approved Ferriprox (deferiprone) to treat patients with iron
`
`overload due to blood transfusions in patients with thalassemia, a genetic blood
`
`disorder that causes anemia, who had an inadequate response to prior chelation
`
`therapy.
`
`(Logo: http://photos.prnewswire.com/prnh/20090824/FDALOCO)
`
`http://www.prnewswire.com/news—releases/fda—approves—ferriprox-to-treat~patients-with-excess-iron-in-the-body- 1 3 1 876763 .html
`
`8/23/20] 7
`
`
`
`FDA Approves Ferriprox to Treat Patients with Excess Iron in the Body
`
`Page 2 of 4
`
`Patients with thalassemia have excess iron in the body from the frequent blood
`
`transfusions (transfusional iron overload), a condition that is serious and can be fatal.
`
`These patients also have a risk of developing liver disease, diabetes, arthritis, heart
`
`failure or an abnormal heart rhythm.
`
`The standard of care to treat transfusional iron overload is chelation therapy — chemical
`
`agents that are used to remove heavy metals from the body. Ferriprox is intended for
`
`use when chelation therapy is inadequate.
`
`"Ferriprox represents the first new FDA-approved treatment for this disorder since
`
`2005," said Richard Pazdur, MD, director of the Office of Hematology and Oncology
`Products in the FDA‘s Center for Drug Evaluation and Research.
`
`The safety and effectiveness of Ferriprox is based on an analysis of data from twelve
`
`clinical studies in 236 patients. Patients participating in the study did not respond to
`
`prior iron chelation therapy. Ferriprox was considered a successful treatment for
`
`patients who experienced at least a 20 percent decrease in serum ferritin, a protein
`
`that stores iron in the body for later use. Half of the patients in the study experienced at
`
`least a 20 percent decrease in ferritin levels.
`
`The most common side effects seen in patients who received Ferriprox included
`
`nausea, vomiting, abdominal and joint pain, urine discoloration (chromaturia), a
`
`decrease in the number of white blood cells (neutropenia), and an increase in the level
`
`of a liver enzyme that may be indicative of tissue or liver damage at unsafe amounts.
`
`The most serious side effect seen in about two percent of patients treated with
`
`Ferriprox was the development of agranulocytosis, a serious and potentially life—
`
`threatening reduction in the number of granulocytes (a type of white blood cell that
`
`fig hts infection).
`
`0:8
`
`http://www.prnewswire.conflnews-re1eases/fda-approves-ferriprox-to—treat-patients—with—excess-ir0n-in—the-body—131876763.htm1
`
`8/23/2017
`
`
`
`FDA Approves Ferriprox to Treat Patients with Excess Iron in the Body
`
`Page 3 0f4
`
`The therapy is being approved under the FDA‘s accelerated approval program,
`
`designed to provide patients with earlier access to promising new drugs followed by
`
`further studies to confirm the drug's clinical benefit.
`
`The accelerated approval program allows the agency to approve a drug to treat a
`
`serious disease based on clinical data showing that the drug has an effect on an
`
`endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect
`
`on a clinical endpoint other than survival or irreversible morbidity (illness).
`
`ApoPharma has agreed to several post-marketing requirement and commitments. One
`
`commitment includes further study of the use of Ferriprox in patients with sickle cell
`
`disease who have transfusional iron overload.
`
`Earlier this year, the US. Department of Health and Human Services (HHS) launched
`
`the Sickle Cell Disease (SCD) Initiative bringing together HHS agencies to enhance the
`
`quality and quantity of SCD data, develop best practice guidelines and quality of care
`
`metrics, improve health care delivery and coordination of care for patients with SCD,
`
`facilitate approval of new medical products, and expand research on SCD. The post—
`
`marketing requirement for further study of Ferriprox aligns with the goals of the SCD
`
`Initiative.
`
`Ferriprox is marketed by ApoPharma Inc. of Toronto.
`
`For more information:
`
`FDA: Office of Hematology and Oncology Products
`
`http://www.fda.gov/AboutFDA/CentersOffices/CDER/ucm09l745.htm
`
`FDA: Spotlight on Drug Innovation - Update of FDA's novel drug approvals in 2011
`
`http://wwwfdagov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm254242htm
`
`0%
`
`http://wwwprnewswire.com/news—releases/fda—approves-ferriprox-to—treat—patiants-with-excess—iron—in-the-body- 1 3 1 876763 .html
`
`8/23/2017
`
`
`
`FDA Approves Ferriprox to Treat Patients with Excess Iron in the Body
`
`Page 4 of 4
`
`The FDA, an agency within the US. Department of Health and Human Services,
`
`protects the public health by assuring the safety, effectiveness, and security of human
`
`and veterinary drugs, vaccines and other biological products for human use, and
`
`medical devices. The agency also is responsible for the safety and security of our
`
`nation's food supply, cosmetics, dietary supplements, products that give off electronic
`
`radiation, and for regulating tobacco products.
`
`Media Inquiries: Erica Jefferson, 301—796-4988, erica.jefferson@fda.hhs.gov
`
`Consumer Inquiries: 888—INFO—FDA
`
`SOURCE US. Food and Drug Administration
`
`RELATED LlNKS
`
`http://www.fda.gov/
`
`http://Www.pmewswire.com/news-releases/fda-approves-ferriprox~to-treat—patients-with-excess—iron-in-the-body- 1 3 1 876763 .html
`
`8/23/2017
`
`