2012 Report     Medicines in Development     Children     presented by america’s biopharmaceutical     research companies ...
Medicines in Development for ChildrenMedicines in Development for Children*       Arthritis/Musculoskeletal               ...
Medicines in Development for Children Arthritis/Musculoskeletal Diseases Product Name           Sponsor                Ind...
Medicines in Development for ChildrenCancerProduct Name              Sponsor                     Indication               ...
Medicines in Development for Children Cancer Product Name                    Sponsor                            Indication...
Medicines in Development for ChildrenCancerProduct Name                  Sponsor                     Indication           ...
Medicines in Development for Children Cancer Product Name                     Sponsor                             Indicati...
Medicines in Development for ChildrenCancerProduct Name                 Sponsor                          Indication       ...
Medicines in Development for Children Cardiovascular Disease Product Name           Sponsor                       Indicati...
Medicines in Development for Children Cardiovascular Disease Product Name            Sponsor                            In...
Medicines in Development for Children Diabetes Product Name           Sponsor                     Indication              ...
Medicines in Development for Children Gastrointestinal Disorders Product Name                 Sponsor                     ...
Medicines in Development for Children Gastrointestinal Disorders Product Name           Sponsor                        Ind...
Medicines in Development for Children Genetic Disorders Product Name                    Sponsor                         In...
Medicines in Development for Children Genetic Disorders Product Name                     Sponsor                     Indic...
Medicines in Development for Children Genetic Disorders Product Name                  Sponsor                            I...
Medicines in Development for Children Genetic Disorders Product Name                 Sponsor                         Indic...
Medicines in Development for Children Genetic Disorders Product Name                      Sponsor                       In...
Medicines in Development for Children Infectious Diseases Product Name                         Sponsor                    ...
Medicines in Development for Children Infectious Diseases Product Name                   Sponsor                          ...
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
PhRMA Report 2012: Medicines in Development for Children
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PhRMA Report 2012: Medicines in Development for Children

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America’s biopharmaceutical companies are researching 316 medicines to help meet the unique health care needs of children and adolescents. Considerable progress in the fight against diseases that impact children has been achieved in recent decades, with medicines playing a key role.

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PhRMA Report 2012: Medicines in Development for Children

  1. 1. 2012 Report Medicines in Development Children presented by america’s biopharmaceutical research companies More Than 300 Medicines are Being Researched in Clinical Trials to Meet the Unique Needs of Children ay rn tod ive A child bo ect to l r Medicines in Development can exp longe for Children 30 years rn ild bo o than a ch y ag a c e n t ur 55 53 52 Biopharmaceutical research companies are and the “Best Pharmaceuticals for Children Act” testing 316 medicines to meet the special (BPCA), highly successful programs that gener- health needs of infants, children and adoles- ate new knowledge about medicines for use in cents. These medicines offer hope that the children. PREA and BPCA are set to expire on significant improvements achieved in children’s October 1, 2012 unless reauthorized or made 29 health over the past few decades will continue permanent by Congress. 26 and even accelerate. BPCA and PREA have led to hundreds of Infant mortality has sunk to record lows. New pediatric studies covering more than 16 broad vaccines protect children against many child- categories of diseases that affect children. Sig- 18 hood diseases. Antibiotics prevent deaths from nificant progress has been made in our ability pneumonia and other infectious diseases that to treat pediatric patients thanks to the research used to claim the lives of children. Thanks in conducted as a result of BPCA and PREA. part to major treatment advances, 82 percent of Today, pediatricians have more information than children diagnosed with cancer in recent years ever about which medicines are safe and effec- will survive five years or longer, compared to tive for children and at what doses. Since 1998, 58 percent 30 years ago. Biopharmaceutical BPCA and PREA have resulted in 424 pediatric research companies are working to continue labeling changes, according the U.S. Food and this progress and to meet new challenges to Drug Administration. er rs s s e es er er as de nc as rd rd children’s health. se Ca r se so so iso New medicines, and new knowledge about pe- Di Di Di Di yD lar In addition to creating new medicines specifi- diatric use of existing medicines, can offer hope tic gic us tor cu ne olo tio cally for children, biopharmaceutical research that children today will recover from illnesses as ira Ge ec ur iov sp companies are testing many existing medicines and grow into healthy adults. Inf Ne RerdCa to determine safe and effective dosage levels for children. In 2007, Congress reauthorized both the “Pediatric Research Equity Act” (PREA)
  2. 2. Medicines in Development for ChildrenMedicines in Development for Children* Arthritis/Musculoskeletal 7 Cancer 53 Cardiovascular Disease 18 Diabetes 11 Eye Disorders 5 Gastrointestinal Disorders 11 Genetic Disorders 52 Infectious Diseases 55 Neurologic Disorders 29 Psychiatric Disorders 14 Respiratory Disorders 26 Skin Disorders 15 Other 31* Some medicines are listed in more than one category. Arthritis/Musculoskeletal Diseases Product Name Sponsor Indication Development Status†** Actemra® Roche active systemic juvenile idiopathic Phase I tocilizumab Nutley, NJ arthritis up to 24 months (973) 235-5000 Cymbalta® Eli Lilly juvenile primary fibromyalgia Phase III duloxetine Indianapolis, IN syndrome 13 years – 17 years (see also psychiatric) (800) 545-5979 Humira® Abbott Laboratories juvenile idiopathic arthritis Phase III adalimumab Abbott Park, IL (see also gastrointestinal) 2 years – 4 years (847) 937-6100 Ilaris® Novartis Pharmaceuticals systemic juvenile idiopathic Phase III canakinumab East Hanover, NJ arthritis 2 years and older (Orphan Drug) (888) 669-6682† ll of the products listed in this report are being tested in children. In most cases, we have identified in the “Development Status” column the ages for which the A trials are being conducted.**For more information about a specific medicine in this report, please call the telephone number listed.2 Medicines in Development Children 2012
  3. 3. Medicines in Development for Children Arthritis/Musculoskeletal Diseases Product Name Sponsor Indication Development Status Orencia® Bristol-Myers Squibb juvenile rheumatoid arthritis in clinical trials abatacept Princeton, NJ pediatric patients (800) 332-2056 -------------------------------------------------- ------------------------------------------- juvenile idiopathic arthritis in clinical trials 2 years – 17 years (800) 332-2056 -------------------------------------------------- ------------------------------------------- juvenile rheumatoid arthritis in clinical trials (subcutaneous formulation) pediatric patients (800) 332-2056 Savella® Forest Laboratories primary fibromyalgia syndrome Phase II milnacipran New York, NY 13 years – 17 years (800) 678-1605 Simponi® Janssen Biotech juvenile idiopathic arthritis Phase III golimumab Titusville, NJ 2 years – 18 years (800) 526-7736 Cancer Product Name Sponsor Indication Development Status AC220 Ambit Biosciences acute lymphoblastic leukemia (ALL), Phase I San Diego, CA acute myelogenous leukemia (AML) 1 month – 21 years (858) 334-2100 Adcetris™ Millennium Oncology Hodgkin’s lymphoma Phase II completed brentuximab vedotin Cambridge, MA (monotherapy) 12 years and older (Orphan Drug) Seattle Genetics (Fast Track) (425) 527-4000 Bothell, WA -------------------------------------------------- ------------------------------------------- refractory or relapsed anaplastic Phase II large cell lymphoma 12 years and older (425) 527-4000 -------------------------------------------------- ------------------------------------------- CD30-positive non-Hodgkin’s Phase II lymphoma, CD30-positive 6 years and older nonlymphomatosus malignancies (425) 527-4000Medicines in Development Children 2012 3
  4. 4. Medicines in Development for ChildrenCancerProduct Name Sponsor Indication Development StatusAPN301 Apeiron Biologics neuroblastoma Phase II completed(hu14.18-IL2) Vienna, Austria pediatric patients National Cancer Institute www.apeiron-biologics.com Bethesda, MDARC-100 Archer Biosciences medulloblastoma, neuroblastoma Phase I/II New York, NY 12 months – 21 years (646) 747-9090ARQ-197 ArQule microphthalmia transcription (MiT) Phase II completed Woburn, MA factor tumors 13 years and older (781) 994-0300AT-9283 Astex Pharmaceuticals solid tumors Phase I Dublin, CA adolescents and children (925) 560-0100blinatumomab Micromet relapsed/refractory acute ALL Phase I/II(Orphan Drug) Rockville, MD up to 18 years (240) 752-1420CAT-8015 MedImmune ALL, non-Hodgkin’s lymphoma Phase I(moxetumomab pasudotox) Gaithersburg, MD 6 months – 25 years (301) 398-0000cintredekin besodotox INSYS Pharma glioma Phase I(Orphan Drug) Phoenix, AZ (Fast Track) adolescents and children (602) 910-2617cixutumumab ImClone Systems advanced or metastatic sarcoma Phase II(IMC-A12) New York, NY 12 years and older Eli Lilly (212) 645-1405 Indianapolis, IN (800) 545-5979Clolar® Genzyme AML Phase IIclofarabine Cambridge, MA pediatric patients (617) 252-7500CLT-008 Cellerant Therapeutics hematological malignancies Phase I San Carlos, CA 12 years and older (650) 232-2122Dacogen® Eisai AML Phase IIdecitabine Woodcliff Lake, NJ 1 year – 16 years (888) 442-4743EZN-2208 Enzon Pharmaceuticals solid tumors Phase I/II Piscataway, NJ 1 year – 21 years (732) 980-45004 Medicines in Development Children 2012
  5. 5. Medicines in Development for Children Cancer Product Name Sponsor Indication Development Status EZN-3042 Enzon Pharmaceuticals ALL Phase I Piscataway, NJ pediatric patients Therapeutic Advances in (732) 980-4500 Childhood Leukemia Lymphoma Los Angeles, CA FANG™ Gradalis solid tumors Phase I autologous tumor cell vaccine Carrollton, TX 12 years and older (214) 442-8100 GliAtak™ Advantagene malignant brain tumors Phase I cancer gene therapy Auburndale, MA 3 years – 22 years (Orphan Drug) (617) 916-5445 GS 1101 Gilead Sciences Hodgkin’s lymphoma Phase II Foster City, CA 12 years and older (800) 445-3235 GSK2110183 GlaxoSmithKline Langerhans cell histiocytosis Phase II Rsch. Triangle Park, NC 12 years and older (888) 825-5249 JX-594 Jennerex Biotherapeutics refractory solid tumors Phase I (recombinant vaccinia virus) San Francisco, CA 2 years – 21 years (415) 281-8886 KRX-0401 Keryx Biopharmaceuticals solid tumors Phase I (perifosine) New York, NY pediatric patients (212) 531-5965 LDE225 Novartis Pharmaceuticals solid tumors Phase I (erismodegib) East Hanover, NJ 12 months – 17 years (888) 669-6682 Marqibo® Talon Therapeutics refractory cancer Phase I/II vincristine sulfate liposomes San Mateo, CA 2 years – 20 years injection (650) 588-6404 midostaurin Novartis Pharmaceuticals relapsed/refractory AML and ALL Phase I/II (PKC412) East Hanover, NJ 3 months – 18 years (Orphan Drug) (888) 669-6682 monoclonal antibody United Therapeutics neuroblastoma Phase III ch14.18 Silver Spring, MD pediatric patients National Cancer Institute (301) 608-9292 Bethesda, MD Nexavar® Bayer HealthCare Pharmaceuticals glioma, neurofibromatoses Phase II sorafenib Wayne, NJ pediatric patients (888) 842-2937Medicines in Development Children 2012 5
  6. 6. Medicines in Development for ChildrenCancerProduct Name Sponsor Indication Development StatusNiCord™ Gamida Cell hematological malignancies Phase I/IIumbilical cord blood Jerusalem, Israel 8 years and olderstem cell therapy www.gamida-cell.comnimotuzumab YM Biosciences USA recurrent glioma Phase II(Orphan Drug) Wayne, PA 3 years – 18 years (610) 560-0600PF-03084014 Pfizer cancer Phase I New York, NY 16 years and older (860) 732-5156PM00104 PharmaMar Ewing’s sarcoma Phase II Madrid, Spain 16 years and older www.pharmamar.compropranolol Pierre Fabre infantile hemangioma Phase II/III Parsippany, NJ 35 days – 150 days (973) 898-1042PTC299 PTC Therapeutics central nervous system (CNS) cancer Phase I South Plainfield, NJ pediatric patients National Cancer Institute www.ptcbio.com Bethesda, MDRexin-G™ Epeius Biotechnologies sarcoma Phase I/II completedtumor-targeted gene therapy San Marino, CA 10 years and older(Orphan Drug) (626) 441-6695ridaforolimus Ariad Pharmaceuticals sarcoma Phase III(Orphan Drug) Cambridge, MA 13 years and older Merck (800) 672-6372 Whitehouse Station, NJSAR3419 sanofi-aventis ALL Phase II Bridgewater, NJ 16 years and older (800) 633-1810SL-701 Stemline Therapeutics glioma Phase I/II(brain cancer vaccine) New York, NY pediatric patients (212) 831-1111Sprycel® Bristol-Myers Squibb chronic myeloid leukemia (CML), in clinical trialsdasatinib Princeton, NJ Philadelphia-positive ALL pediatric patients(Orphan Drug) (800) 332-2056Tarceva® Genentech recurrent/refractory ependymoma Phase IIerlotinib South San Francisco, CA 1 year – 21 years OSI Pharmaceuticals (631) 962-0600 Farmingdale, NY6 Medicines in Development Children 2012
  7. 7. Medicines in Development for Children Cancer Product Name Sponsor Indication Development Status Tasigna® Novartis Pharmaceuticals ALL, CML Phase I nilotinib East Hanover, NJ up to 18 years (Orphan Drug) (888) 669-6682 TH-302 Threshold Pharmaceuticals soft tissue sarcoma Phase III South San Francisco, CA 15 years and older (650) 474-8200 TheraCIM® YM Biosciences recurrent diffuse intrinsic Phase II completed nimotuzumab Lehigh Valley, PA pontine glioma 3 years – 18 years (Orphan Drug) (610) 560-0600 Torisel® Pfizer solid tumors Phase I/II temsirolimus New York, NY 1 year – 21 years (860) 732-5156 Treanda® Cephalon relapsed or refractory acute leukemia Phase I/II bendamustine Frazer, PA (ALL, AML) 1 year – 20 years (610) 344-0200 trivalent ganglioside MabVax Therapeutics sarcoma Phase II cancer vaccine San Diego, CA 16 years and older (858) 259-9405 Ultratrace™ MIBG Molecular Insight Pharmaceuticals neuroendocrine tumors Phase II Iobenguane I-131 Cambridge, MA (Fast Track) 12 years and older (Orphan Drug) (617) 492-5554 Vectibix® Amgen solid tumors Phase I panitumumab Thousand Oaks, CA 1 year – 17 years (800) 772-6436 vismodegib Genentech medulloblastoma Phase II (GDC-0049) South San Francisco, CA 3 years – 21 years National Cancer Institute (800) 626-3553 Bethesda, MD Xalkori® Pfizer ALK-positive tumors Phase I crizotinib New York, NY 15 years and older (860) 732-5156 Xeloda® Roche gliomas Phase I capecitabine Nutley, NJ 3 years – 21 years (973) 235-5000 Xerecept® Celtic Pharma brain edema associated with Phase I/II corticorelin acetate injection Hamilton, Bermuda brain tumors 1 year – 18 years (Orphan Drug) (212) 616-4000Medicines in Development Children 2012 7
  8. 8. Medicines in Development for ChildrenCancerProduct Name Sponsor Indication Development StatusXgeva® Amgen recurrent or unresectable Phase IIdenosumab Thousand Oaks, CA giant cell tumor of bone 12 years and older (800) 772-6436Yervoy® Bristol-Myers Squibb advanced/metastatic melanoma in clinical trialsipilimumab Princeton, NJ 12 years – 17 years(Orphan Drug) (800) 332-2056Yondelis® Janssen Research Development soft tissue sarcoma Phase IIItrabdectedin Raritan, NJ 15 years and older (800) 526-7736Cardiovascular DiseaseProduct Name Sponsor Indication Development StatusCardiolite® Lantheus Medical Imaging Kawasaki disease Phase I/II completedkit for the preparation of N. Billerica, MA 4 years – 16 yearstechnetium Tc99m sestamibi (800) 362-2668for injectionCER-001 Cerenis Therapeutics acute coronary syndrome Phase II Ann Arbor, MI (see also genetic) pediatric patients included (734) 769-1110cicletanine Gilead Sciences pulmonary arterial hypertension Phase II(Orphan Drug) Foster City, CA 16 years and older (800) 445-3235Cozaar® Merck hypertension Phase IIIlosartan Whitehouse Station, NJ 6 months – 6 years (800) 672-6372Edarbi® Takeda Pharmaceuticals hypertension Phase Iazilsartan medoxomil North America 1 year and older Deerfield, IL (877) 825-3327Eliquis™ Bristol-Myers Squibb venous thromboembolism in clinical trialsapixaban Princeton, NJ pediatric patients Pfizer (800) 332-2056 New York, NY (860) 732-51568 Medicines in Development Children 2012
  9. 9. Medicines in Development for Children Cardiovascular Disease Product Name Sponsor Indication Development Status Fragmin® Eisai venous thromboembolism Phase II dalteparin Woodcliff Lake, NJ up to 18 years (888) 442-4743 L-citrulline Asklepion Pharmaceuticals pulmonary hypertension in children Phase I/II (intravenous) Brentwood, TN undergoing heart bypass surgery up to 6 years (615) 377-4617 Letairis™ GlaxoSmithKline pulmonary arterial hypertension Phase II ambrisentan Rsch. Triangle Park, NC 8 years – 18 years (Orphan Drug) (888) 825-5249 macitentan Actelion Pharmaceuticals US pulmonary arterial hypertension Phase III (ACT-064992) South San Francisco, CA 12 years and older (Orphan Drug) (650) 624-6900 mipomersen Genzyme severe hypercholesterolemia Phase III Cambridge, MA (see also genetic) 12 years and older Isis Pharmaceuticals (617) 252-7500 Carlsbad, CA (760) 931-9200 oral treprostinil United Therapeutics pulmonary arterial hypertension Phase III (Orphan Drug) Silver Spring, MD 12 years and older (301) 608-9292 Pradaxa® Boehringer Ingelheim venous thromboembolism Phase II dabigatran Pharmaceuticals pediatric patients Ridgefield, CT (800) 243-0127 sildenafil citrate Pfizer pulmonary arterial hypertension Phase III New York, NY 1 year – 17 years (860) 732-5156 Tekturna® Novartis Pharmaceuticals hypertension Phase III aliskiren East Hanover, NJ 6 years – 17 years (888) 669-6682 Tracleer® Actelion Pharmaceuticals US pulmonary arterial hypertension Phase III bosentan South San Francisco, CA 3 months – 12 years (Orphan Drug) (650) 624-6900 -------------------------------------------------- ------------------------------------------- pulmonary arterial hypertension Phase III up to 7 days (650) 624-6900 -------------------------------------------------- ------------------------------------------- persistent pulmonary hypertension Phase III in newborns up to 7 days (650) 624-6900Medicines in Development Children 2012 9
  10. 10. Medicines in Development for Children Cardiovascular Disease Product Name Sponsor Indication Development Status Xarelto® Bayer HealthCare Pharmaceuticals venous thromboembolism Phase I rivaroxaban Wayne, NJ 6 months – 18 years Janssen Research Development (888) 842-2937 Raritan, NJ (800) 526-7736 Zetia® Merck primary hypercholesterolemia Phase III ezetimibe Whitehouse Station, NJ 6 years – 10 years (800) 672-6372 Diabetes Product Name Sponsor Indication Development Status alogliptin Takeda Pharmaceuticals type 2 diabetes Phase I North America 10 years and older Deerfield, IL (877) 825-3327 autoimmune diabetes Diamyd Therapeutics prevention of type 1 diabetes Phase III vaccine (rhGAD65) Stockholm, Sweden 10 years and older (Orphan Drug) Pittsburgh, PA (412) 770-1310 Byetta® Amylin Pharmaceuticals type 2 diabetes Phase III exenatide San Diego, CA 10 years – 17 years (858) 552-2200 dapagliflozin AstraZeneca type 2 diabetes in clinical trials Wilmington, DE 10 years – 17 years Bristol-Myers Squibb (800) 236-9933 Princeton, NJ (800) 332-2056 Janumet® Merck type 2 diabetes Phase III sitagliptin/metformin Whitehouse Station, NJ 10 years – 17 years (800) 672-6372 Januvia® Merck type 2 diabetes Phase I completed sitagliptin Whitehouse Station, NJ adolescents (800) 672-6372 Onglyza™ AstraZeneca type 2 diabetes in clinical trials saxagliptin Wilmington, DE 10 years – 17 years Bristol-Myers Squibb (800) 236-9933 Princeton, NJ (800) 332-2056 otelixizumab Tolerx type 1 diabetes Phase II (Orphan Drug) Cambridge, MA 12 years and older (617) 354-810010 Medicines in Development Children 2012
  11. 11. Medicines in Development for Children Diabetes Product Name Sponsor Indication Development Status Prochymal® Osiris Therapeutics type 1 diabetes Phase II remestemcel-L Columbia, MD (see also other) 12 years and older (Orphan Drug) (443) 545-1800 teplizumab MacroGenics type 1 diabetes Phase III (MGA031) Rockville, MD 8 years and older (Orphan Drug) (301) 251-5172 Victoza® Novo Nordisk type 2 diabetes Phase III completed liraglutide Princeton, NJ 10 years and older (609) 987-5800 Eye Disorders Product Name Sponsor Indication Development Status AC-150 Aciex Therapeutics allergic conjunctivitis Phase II Boston, MA 10 years and older (508) 439-4946 AC-170 Aciex Therapeutics allergic conjunctivitis Phase II/III Boston, MA 10 years and older (508) 439-4946 Cystaran™ Sigma-Tau Pharmaceuticals corneal cysteine crystal application submitted cysteamine HCl Gaithersburg, MD accumulation in cystinosis 1 year and older (Orphan Drug) (800) 447-0169 Durezol™ Alcon Research inflammation following Phase III difluprednate Fort Worth, TX cataract surgery up to 3 years (800) 862-5266 ocriplasmin ThromboGenics vitrectomy Phase II (recombinant human New York, NY up to 16 years microplasmin) (212) 201-0920 (Orphan Drug)Medicines in Development Children 2012 11
  12. 12. Medicines in Development for Children Gastrointestinal Disorders Product Name Sponsor Indication Development Status Aciphex® Eisai gastroesophageal reflux disease Phase III rabeprazole Woodcliff Lake, NJ (GERD) 1 year – 11 years Janssen Research Development (888) 442-4743 Raritan, NJ (800) 526-7736 -------------------------------------------------- ------------------------------------------- GERD Phase III 1 month – 11 months (888) 442-4743 (800) 526-7736 -------------------------------------------------- ------------------------------------------- GERD Phase I less than 44 weeks (888) 442-4743 (800) 526-7736 Asacol® Warner-Chilcott ulcerative colitis Phase III completed mesalamine delayed-release Rockaway, NJ 5 years – 17 years tablets (400mg) (800) 521-8813 Asacol® HD Warner-Chilcott ulcerative colitis Phase III mesalamine delayed-release Rockaway, NJ 5 years – 17 years tablets (800mg) (800) 521-8813 Cimzia® UCB Crohn’s disease Phase II certolizumab pegol Smyrna, GA 6 years – 17 years (770) 970-7500 crofelemer Napo Pharmaceuticals diarrhea Phase I San Francisco, CA pediatric patients (415) 371-8300 dexlansoprazole Takeda Pharmaceuticals GERD Phase I North America 1 year – 11 years Deerfield, IL (877) 825-3327 Humira® Abbott Laboratories Crohn’s disease Phase III adalimumab Abbott Park, IL (see also arthritis) 7 years – 18 years (847) 937-6100 MMX mesalamine Shire Pharmaceutical ulcerative colitis Phase I Cambridge, MA 5 years – 17 years www.shire.com nepadutant Menarini infant colic Phase II oral solution Florence, Italy 1 month – 4 months www.menarini.com oral viscous budesonide Meritage Pharma eosinophilic esophagitis Phase II completed (Orphan Drug) San Diego, CA 2 years – 18 years (858) 436-166012 Medicines in Development Children 2012
  13. 13. Medicines in Development for Children Gastrointestinal Disorders Product Name Sponsor Indication Development Status reslizumab Cephalon eosinophilic esophagitis Phase III (Orphan Drug) Frazer, PA (see also respiratory) 5 years and older (610) 344-0200 Genetic Disorders Product Name Sponsor Indication Development Status A-001 Anthera Pharmaceuticals acute chest syndrome in patients Phase II (varespladib) Hayward, CA with sickle cell disease 5 years and older (Orphan Drug) (Fast Track) (510) 856-5600 AAV2-RPE65 Applied Genetic Technologies leber congenital amaurosis Phase I/II (Orphan Drug) Alachua, FL 6 years and older (386) 462-2204 AFQ056 Novartis Pharmaceuticals fragile X syndrome Phase II/III East Hanover, NJ 12 years – 17 years (888) 669-6682 -------------------------------------------------- ------------------------------------------- fragile X syndrome Phase I 3 years – 11 years (888) 669-6682 arbaclofen Seaside Therapeutics treatment of social withdrawal in Phase III (STX209) Cambridge, MA patients with fragile X syndrome 12 years and older (see also psychiatric) (617) 374-9009 -------------------------------------------------- ------------------------------------------- treatment of social withdrawal Phase II in patients with fragile X syndrome 5 years – 11 years (617) 374-9009 Arimidex® AstraZeneca McCune-Albright syndrome Phase II anastrozole Wilmington, DE up to 10 years (800) 236-9933 AT1001 Amicus Therapeutics Fabry disease Phase III (migalastat) Cranbury, NJ 16 years and older (Orphan Drug) GlaxoSmithKline (800) 825-5249 Rsch. Triangle Park, NCMedicines in Development Children 2012 13
  14. 14. Medicines in Development for Children Genetic Disorders Product Name Sponsor Indication Development Status ataluren PTC Therapeutics cystic fibrosis Phase III (PTC124) South Plainfield, NJ (Fast Track) 6 years and older (Orphan Drug) (908) 222-7000 -------------------------------------------------- ------------------------------------------- Duchenne/Becker muscular Phase III dystrophy (boys) 7 years and older (Fast Track) (908) 222-7000 AVI-4658 AVI BioPharma Duchenne muscular dystrophy Phase II (eteplirsen) Bothell, WA (Fast Track) 7 years – 13 years (Orphan Drug) (425) 354-5038 Berinert® CSL Behring hereditary angioedema Phase II/III completed C1 esterase inhibitor, human King of Prussia, PA 6 years and older (Orphan Drug) (610) 878-4000 BMN-110 BioMarin Pharmaceuticals mucopolysaccharidosis IV A Phase III (Orphan Drug) Novato, CA (Morquio A syndrome) 5 years and older (415) 506-6700 BMN-701 BioMarin Pharmaceuticals late-onset Pompe disease Phase I/II (Orphan Drug) Novato, CA 13 years and older (415) 506-6700 CER-001 Cerenis Therapeutics homozygous familial Phase II Ann Arbor, MI hypercholesterolemia 12 years and older (see also cardiovascular) (734) 769-1110 Cinryze™ ViroPharma hereditary angioedema Phase II C1 esterase inhibitor (human) Exton, PA 2 years – 11 years (Orphan Drug) (888) 651-0201 -------------------------------------------------- ------------------------------------------- hereditary angioedema Phase II 12 years and older (888) 651-0201 conestat alfa Pharming hereditary angioedema Phase III (recombinant human C1 Leiden, The Netherlands (Fast Track) 13 years and older inhibitor) www.pharming.com ecopipam Psyadon Pharmaceuticals Lesch-Nyhan disease Phase I (Orphan Drug) Germantown, MD 6 years and older (301) 919-2020 Elaprase® Shire Human Genetic Therapies central nervous system Phase I/II idursulfase-IT Lexington, MA involvement in Hunter syndrome 3 years – 18 years (Orphan Drug) (mucopolysaccharidosis II) (866) 888-066014 Medicines in Development Children 2012
  15. 15. Medicines in Development for Children Genetic Disorders Product Name Sponsor Indication Development Status ENB-0040 Enobia Pharma hypophosphatasia Phase II/III (Orphan Drug) Cambridge, MA (Fast Track) up to 5 years (617) 674-5720 -------------------------------------------------- ------------------------------------------- hypophosphatasia Phase II (Fast Track) 5 years – 13 years (617) 674-5720 ------------------------------------------- -------------------------------------------------- Phase II hypophosphatasia 6 months – 42 months (617) 674-5720 EPI-743 Edison Pharmaceuticals inherited mitochondrial diseases Phase II (Orphan Drug) Mountain View, CA 1 year and older www.edisonpharma.com Faslodex® AstraZeneca McCune-Albright syndrome Phase II fulvestrant Wilmington, DE (precocious puberty) up to 10 years (800) 236-9933 Genz-112638 Genzyme Gaucher disease type 1 Phase III (eliglustat) Cambridge, MA 16 years and older (617) 252-7500 GMI-1070/PF-06460031 GlycoMimetics treatment of sickle cell pain crisis Phase II (Orphan Drug) Gaithersburg, MD (Fast Track) 12 years and older Pfizer (240) 243-1201 New York, NY (860) 732-5156 GSK656933 GlaxoSmithKline cystic fibrosis Phase II completed Rsch. Triangle Park, NC 16 years and older (888) 825-5249 GSK2402968 GlaxoSmithKline Duchenne muscular dystrophy Phase II (Orphan Drug) Rsch. Triangle Park, NC 5 years and older (888) 825-5249 HQK-1001 HemaQuest Pharmaceuticals sickle cell anemia Phase II (Orphan Drug) San Diego, CA 12 years and older (858) 356-5590 HuCNS-SC® StemCells Pelizaeus-Merzbacher disease Phase I adult neural stem cell therapy Newark, CA 6 months – 5 years (510) 456-4000 human-cl rhFVIII Octapharma severe hemophilia A Phase III Hoboken, NJ 12 years and older (866) 766-4860Medicines in Development Children 2012 15
  16. 16. Medicines in Development for Children Genetic Disorders Product Name Sponsor Indication Development Status IB1001 Inspiration Biopharmaceuticals hemophilia B Phase III (recombinant factor IX) Laguna Niguel, CA up to 12 years www.inspirationbio.com idebenone Santhera Pharmaceuticals Friedreich’s ataxia Phase III (Orphan Drug) Charlestown, MA (Fast Track) 9 years and older (617) 886-5161 -------------------------------------------------- ------------------------------------------- Duchenne muscular dystrophy Phase III 10 years – 18 years (617) 886-5161 -------------------------------------------------- ------------------------------------------- MELAS syndrome Phase II 8 years and older (617) 886-5161 immune globulin infusion Baxter Healthcare primary immunodeficiency Phase II/III (human)/recombinant human Deerfield, IL disorders 2 years and older hyaluronidase (rHuPH20) (800) 422-9837 inhaled mannitol Pharmaxis cystic fibrosis Phase III (Orphan Drug) Exton, PA (Fast Track) 6 years and older (610) 363-5120 Kogenate-PF® Bayer HealthCare Pharmaceuticals hemophilia A Phase III octocog alfa, Wayne, NJ up to 12 years plasma protein-free (888) 842-2937 (recombinant factor VIII) L-glutamine powder for oral Emmaus Medical sickle cell anemia, thalassemia Phase III solution Torrance, CA 5 years and older (Orphan Drug) (877) 420-6493 mipomersen Genzyme familial hypercholesterolemia Phase III (Orphan Drug) Cambridge, MA (hyperlipoproteinemia type IIa) 12 years and older Isis Pharmaceuticals (see also cardiovascular) (617) 252-7500 Carlsbad, CA (760) 931-9200 Myozyme® Genzyme infantile-onset Pompe disease Phase I/II completed alglucosidase alfa Cambridge, MA (glycogen storage disease-II) 6 months – 36 months (Orphan Drug) (617) 252-750016 Medicines in Development Children 2012
  17. 17. Medicines in Development for Children Genetic Disorders Product Name Sponsor Indication Development Status NN1841 Novo Nordisk congenital FXIII deficiency application submitted (recombinant factor XIII) Princeton, NJ 6 years and older (609) 987-5800 -------------------------------------------------- ------------------------------------------- congenital FXIII deficiency Phase III 1 year – 6 years (609) 987-5800 NN7999 Novo Nordisk hemophilia B Phase III (glycopegylated rFIX) Princeton, NJ 13 years and older (609) 987-5800 oxalobacter formigenes OxThera primary hyperoxaluria Phase II/III (OC3) Fort White, FL 2 years and older www.oxthera.com plasma-derived AT-III Grifols Biologicals antithrombin III deficiency Phase II/III concentrate Los Angeles, CA 12 years and older (888) 474-3657 rAvPAL-PEG BioMarin Pharmaceuticals phenylketonuria Phase II (Orphan Drug) Novato, CA 16 years and older (415) 506-6700 recombinant factor VIII-Fc Biogen Idec severe hemophilia A Phase II/III fusion protein (rFVIIIFc) Weston, MA 12 years and older (Orphan Drug) (781) 464-2000 recombinant factor IX-Fc Biogen Idec hemophilia B Phase II/III fusion protein (rFIXFc) Weston, MA 12 years and older (Orphan Drug) (781) 464-2000 Replagal® Shire Human Genetic Therapies Fabry disease Phase II/III agalsidase alfa Lexington, MA (Fast Track) 7 years – 17 years (Orphan Drug) (866) 888-0660 RP103 Raptor Pharmaceutical cystinosis Phase III (cysteamine bitartrate Novato, CA 6 years and older delayed-release) (877) 727-8679 (Orphan Drug) SBC-102 Synageva BioPharma Wolman disease Phase I/II (Orphan Drug) Lexington, MA (lysosomal acid lipase deficiency) infants and children (Fast Track) (781) 357-9900Medicines in Development Children 2012 17
  18. 18. Medicines in Development for Children Genetic Disorders Product Name Sponsor Indication Development Status Spiriva® HandiHaler® Boehringer Ingelheim cystic fibrosis Phase III tiotropium bromide Pharmaceuticals (see also respiratory) pediatric patients Ridgefield, CT (800) 243-0127 taliglucerase alfa Pfizer Gaucher’s disease Phase III (Orphan Drug) New York, NY 2 years and older Protalix Biotherapeutics (860) 732-5156 Carmiel, Israel thymosin beta 4 RegeneRx Biopharmaceuticals epidermolysis bullosa Phase II (Orphan Drug) Rockville, MD 2 years and older (301) 208-9191 turoctocog alfa Novo Nordisk hemophilia A Phase III (recombinant factor VIII) Princeton, NJ 6 months and older (609) 987-5800 vatreptacog alfa Novo Nordisk hemophilia A or B with inhibitors Phase III Princeton, NJ 12 years and older (609) 987-5800 VX-770 Vertex Pharmaceuticals cystic fibrosis in patients with the application submitted (ivacaftor) Cambridge, MA G551D gene mutation 6 years and older (Orphan Drug) (Fast Track) (617) 444-6100 Wilate® Octapharma USA prevent bleeding during surgery Phase III von Willebrand factor/ Hoboken, NJ in patients with von Willebrand 6 years and older coagulation factor VIII complex disease (866) 766-4860 (human) Zyntha® Pfizer hemophilia A Phase III antihemophilic factor New York, NY up to 6 years (recombinant), (860) 732-5156 plasma/albumin-free (AF-CC) (Orphan Drug) Infectious Diseases Product Name Sponsor Indication Development Status 33525 Tinea Pharmaceuticals tinea corporis, tinea curis, tinea pedis Phase III (antifungal topical cream) Palo Alto, CA 12 years and older (650) 473-3811 ADS-8902 Adamas Pharmaceuticals influenza A infections Phase II Emeryville, CA 1 year and older (510) 450-350018 Medicines in Development Children 2012
  19. 19. Medicines in Development for Children Infectious Diseases Product Name Sponsor Indication Development Status Aeroquin™ Mpex Pharmaceuticals cystic fibrosis-associated respiratory Phase III levofloxacin for inhalation (Aptalis) tract infections 12 years and older (Orphan Drug) Birmingham, AL (800) 950-8085 -------------------------------------------------- ------------------------------------------- cystic fibrosis-associated respiratory Phase I tract infections 6 years – 16 years (800) 950-8085 Agriflu® Novartis Vaccines Diagnostics influenza A B virus infections Phase III influenza virus vaccine Cambridge, MA pediatric patients (617) 871-5500 Aptivus® Boehringer Ingelheim HIV-1 infection treatment Phase I completed tipranivir with low-dose ritonavir Pharmaceuticals 2 years – 18 years Ridgefield, CT (800) 243-0127 Avelox™ Bayer HealthCare Pharmaceuticals infections Phase III moxifloxacin Wayne, NJ 3 months – 17 years Merck (888) 842-2937 Whitehouse Station, NJ (800) 672-6372 Baraclude® Bristol-Myers Squibb chronic hepatitis B in clinical trials entecavir Princeton, NJ pediatric patients (800) 332-2056 Besivance® Bausch Lomb bacterial conjunctivitis Phase III besifloxacin ophthalmic solution Madison, NJ up to 31 days (877) 442-6925 BSYX-A110 Biosynexus Staphylococcal sepsis Phase II/III (pagibaximab) Gaithersburg, MD premature neonates (Orphan Drug) (301) 330-5800 Cayston® Gilead Sciences chronic Pseudomonas aeruginosa Phase II aztreonam lysine for inhalation Foster City, CA infections in cystic fibrosis 3 months and older (Orphan Drug) (800) 445-3235 ciprofloxacin Bayer HealthCare Pharmaceuticals cystic fibrosis-associated Phase II completed dry powder for inhalation Wayne, NJ Pseudomonas aeruginosa infections 12 years and older (Orphan Drug) Novartis Pharmaceuticals (888) 842-2937 East Hanover, NJ (800) 672-6372 CMX-001 Chimerix adenovirus infections Phase II Durham, NC (Fast Track) 3 months and older (919) 806-1074Medicines in Development Children 2012 19
  20. 20. Medicines in Development for Children Infectious Diseases Product Name Sponsor Indication Development Status Cubicin® Cubist Pharmaceuticals skin and skin structure infections Phase IV daptomycin Lexington, MA 2 years – 17 years (781) 860-8660 -------------------------------------------------- ------------------------------------------- Gram-positive bacterial infections Phase I 3 months – 24 months (781) 860-8660 Doribax™ Janssen Research Development complicated urinary tract Phase III doripenem Raritan, NJ infections, intra-abdominal 3 months – 18 years infections, pneumonia (800) 526-7736 -------------------------------------------------- ------------------------------------------- bacterial infections Phase I up to 12 weeks (800) 526-7736 DTP-HepB-polio-Hib Merck diphtheria, tetanus, pertussis, Phase III hexavalent vaccine Whitehouse Station, NJ hepatitis B, polio, Haemophilus infants sanofi-aventis influenzae type b (800) 672-6372 Bridgewater, NJ econazole nitrate foam 1% AmDerma Pharmaceuticals tinea pedis Phase III Bridgewater, NJ 12 years and older Quinnova Pharmaceuticals (877) 660-6263 Newton, PA Edurant™ Tibotec Pharmaceuticals HIV infection Phase II rilpivirine County Cork, Ireland 12 years – 18 years Janssen Research Development (800) 526-7736 Raritan, NJ Eraxis® Pfizer candidiasis Phase III anidulafungin New York, NY 1 month – 17 years (860) 732-5156 Eraxis® Pfizer invasive aspergillosis Phase III anidulafungin/ New York, NY 16 years and older Vfend® (860) 732-5156 voriconazole combination FluBlok® Protein Sciences prevention of influenza virus infection application submitted trivalent recombinant hemag- Meriden, CT children glutinin vaccine (800) 488-7099 FluLaval® GlaxoSmithKline prevention of influenza Phase III completed influenza virus vaccine Rsch Triangle Park, NC 3 years – 17 years (888) 825-5249 Fluvirin® Novartis Vaccines Diagnostics influenza A virus H1N1 subtype Phase II/III completed influenza virus vaccine Cambridge, MA pediatric patients (MF59-eH1N1) (617) 871-550020 Medicines in Development Children 2012

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