FDA ApprovesFerriprox to Treat Patients with ExcessIron in the
`Body
`Oct 14, 2071, 15:10 ET from U.S. Food and Drug Administration
`
`FDALogo.(PRNewsFoto/U.S. Food and Drug Administration (FDA)}
`
`8 Facebook @plwitter } Pinterest
`
`SILVER SPRING,Md., Oct. 14, 2011 /PRNewswire-USNewswire/-- The U.S. 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 responseto prior chelation therapy.
`
`(Logo: http://photos.prnewswire.com/prnh/20090824/FDALOGO)
`
`Patients with thalassemia have excess iron in the body from the frequent blood transfusions(transfusional iron overload), a condition thatis
`serious and can befatal. These patients also have a risk of developingliver 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 thefirst new FDA-approved treatmentfor this disorder since 2005," said Richard Pazdur, M.D., director of the Office of
`Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
`
`The safety and effectivenessof Ferriprox is based on an analysis of data from twelve clinical studies in 236 patients. Patients participating in the
`study did not respondto prior iron chelation therapy. Ferriprox was considered a successful treatmentfor 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
`percentdecreasein 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 numberof white blood cells (neutropenia), and an increasein the levelof a liver enzyme that maybeindicative
`oftissue or liver damage at unsafe amounts.
`
`The mostserious side effect seen in about two percent of patients treated with Ferriprox was the developmentof 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 endpointthat 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).
`
`ApoPharmahas 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 U.S. 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 requirementfor further study of Ferriprox aligns with the goals of the SCD Initiative.
`
`Ferriprox is marketed by ApoPharmaInc. of Toronto.
`
`For more information:
`
`FDA:Office of Hematology and Oncology Products
`http:/Awww.fda.gov/AboutFDA/CentersOffices/CDER/ucm091745.htm
`
`FDA: Spotlight on Drug Innovation - Update of FDA's novel drug approvals in 2011
`http:/Awww.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm254242.htm
`
`The FDA, an agency within the U.S. 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 agencyalso 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,ericajefferson@fda.hhs.gov
`ConsumerInquiries: 888-INFO-FDA
`
`SOURCE USS. Food and Drug Administration
`
`RELATED LINKS
`
`http:/Awww.fda.gov/
`
`

`

`FDA ApprovesFerriprox 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 U.S. Food and Drug Administration
`
`
`
`FDA Logo. (PRNewsFoto/U.S. Food and Drug Administration (FDA))
`@ Facebook @plwitter ©} Pinterest
`
`SILVER SPRING, Md., Oct. 14, 2011 /PRNewswire-USNewswire/ -- The U.S. 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 responseto prior chelation
`
`therapy.
`
`(Logo: http://ohotos.prnewswire.com/prnh/20090824/FDALOGO)
`
`http://www.prnewswire.com/news-releases/fda-approves-ferriprox-to-treat-patients-with-excess-iron-in-the-body- 131876763 .html
`
`8/23/2017
`
`

`

`FDAApproves Ferriprox to Treat Patients with Excess Iron in the Body
`
`Page 2 of 4
`
`Patients with thalassemia have excessiron in the body from the frequent blood
`transfusions (transfusional iron overload), a condition that is serious and can befatal.
`
`These patients also have a risk of developing liver disease, diabetes, arthritis, heart
`failure or an abnormal heart rhythm.
`
`The standardof 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 whenchelation therapyis inadequate.
`
`"Ferriprox represents the first new FDA-approved treatment for this disorder since
`2005," said Richard Pazdur, M.D., 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 treatmentfor
`patients who experienced at least a 20 percent decrease in serum ferritin, a protein
`that stores iron in the bodyfor later use. Half of the patients in the study experienced at
`least a 20 percent decreasein ferritin levels.
`
`The most commonside effects seen in patients who received Ferriprox included
`nausea, vomiting, abdominal and joint pain, urine discoloration (chromaturia), a
`decrease in the numberof white blood cells (neutropenia), and an increase in the level
`of a liver enzyme that may beindicative of tissue orliver damage at unsafe amounts.
`
`The mostseriousside effect seen in about two percentof patients treated with
`Ferriprox was the developmentof agranulocytosis, a serious and potentially life-
`threatening reduction in the numberof granulocytes(a type of white blood cell that
`fights infection).
`
`o8
`
`http://www.Prnewswire.com/news-releases/fda-approves-ferriprox-to-treat-patients-with-excess-iron-in-the-body-131876763 html
`
`8/23/2017
`
`

`

`FDA ApprovesFerriprox to Treat Patients with Excess Iron in the Body
`
`Page 3 of 4
`
`The therapy is being approved under the FDA's accelerated approval program,
`
`designed to provide patients with earlier access to promising new drugsfollowed 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).
`
`ApoPharmahas 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 havetransfusional iron overload.
`
`Earlier this year, the U.S. 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:/Awww.fda.gov/AboutFDA/CentersOffices/CDER/ucm091745.htm
`
`FDA: Spotlight on Drug Innovation - Update of FDA's novel drug approvals in 2011]
`http:/Avww.fda.gov/Drugs/InformationOnDrugs/Approved Drugs/ucm254242.htm
`
`oS
`
`http://www.prnewswire.com/news-releases/fda-approves-ferriprox-to-treat-patients-with-excess-iron-in-the-body-131876763.html
`
`8/23/2017
`
`

`

`FDA ApprovesFerriprox to Treat Patients with Excess Iron in the Body
`
`Page 4 of 4
`
`The FDA, an agencywithin the U.S. Department of Health and Human Services,
`protects the public health by assuring the safety, effectiveness, and security of human
`and veterinary drugs, vaccines and otherbiological products for human use, and
`medical devices. The agencyalso 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, ericajefferson@fda.hhs.gov
`ConsumerInquiries: 888-INFO-FDA
`
`SOURCE U.S. Food and Drug Administration
`
`RELATED LINKS
`
`http:/Awww.fda.gov/
`
`http://www.prhewswire.com/news-releases/fda-approves-ferriprox-to-treat-patients-with-excess-iron-in-the-body-131876763.html
`
`8/23/2017
`
`