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.

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
Re
rd
Ca
to determine safe and effective dosage levels for
children. In 2007, Congress reauthorized both
the “Pediatric Research Equity Act” (PREA)

2. Medicines in Development for Children
Medicines 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. 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) 527-4000
Medicines in Development Children 2012 3

4. Medicines in Development for Children
Cancer
Product Name Sponsor Indication Development Status
APN301 Apeiron Biologics neuroblastoma Phase II completed
(hu14.18-IL2) Vienna, Austria pediatric patients
National Cancer Institute www.apeiron-biologics.com
Bethesda, MD
ARC-100 Archer Biosciences medulloblastoma, neuroblastoma Phase I/II
New York, NY 12 months – 21 years
(646) 747-9090
ARQ-197 ArQule microphthalmia transcription (MiT) Phase II completed
Woburn, MA factor tumors 13 years and older
(781) 994-0300
AT-9283 Astex Pharmaceuticals solid tumors Phase I
Dublin, CA adolescents and children
(925) 560-0100
blinatumomab Micromet relapsed/refractory acute ALL Phase I/II
(Orphan Drug) Rockville, MD up to 18 years
(240) 752-1420
CAT-8015 MedImmune ALL, non-Hodgkin’s lymphoma Phase I
(moxetumomab pasudotox) Gaithersburg, MD 6 months – 25 years
(301) 398-0000
cintredekin besodotox INSYS Pharma glioma Phase I
(Orphan Drug) Phoenix, AZ (Fast Track) adolescents and children
(602) 910-2617
cixutumumab 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) 545-5979
Clolar® Genzyme AML Phase II
clofarabine Cambridge, MA pediatric patients
(617) 252-7500
CLT-008 Cellerant Therapeutics hematological malignancies Phase I
San Carlos, CA 12 years and older
(650) 232-2122
Dacogen® Eisai AML Phase II
decitabine Woodcliff Lake, NJ 1 year – 16 years
(888) 442-4743
EZN-2208 Enzon Pharmaceuticals solid tumors Phase I/II
Piscataway, NJ 1 year – 21 years
(732) 980-4500
4 Medicines in Development Children 2012

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) 842-2937
Medicines in Development Children 2012 5

6. Medicines in Development for Children
Cancer
Product Name Sponsor Indication Development Status
NiCord™ Gamida Cell hematological malignancies Phase I/II
umbilical cord blood Jerusalem, Israel 8 years and older
stem cell therapy www.gamida-cell.com
nimotuzumab YM Biosciences USA recurrent glioma Phase II
(Orphan Drug) Wayne, PA 3 years – 18 years
(610) 560-0600
PF-03084014 Pfizer cancer Phase I
New York, NY 16 years and older
(860) 732-5156
PM00104 PharmaMar Ewing’s sarcoma Phase II
Madrid, Spain 16 years and older
www.pharmamar.com
propranolol Pierre Fabre infantile hemangioma Phase II/III
Parsippany, NJ 35 days – 150 days
(973) 898-1042
PTC299 PTC Therapeutics central nervous system (CNS) cancer Phase I
South Plainfield, NJ pediatric patients
National Cancer Institute www.ptcbio.com
Bethesda, MD
Rexin-G™ Epeius Biotechnologies sarcoma Phase I/II completed
tumor-targeted gene therapy San Marino, CA 10 years and older
(Orphan Drug) (626) 441-6695
ridaforolimus Ariad Pharmaceuticals sarcoma Phase III
(Orphan Drug) Cambridge, MA 13 years and older
Merck (800) 672-6372
Whitehouse Station, NJ
SAR3419 sanofi-aventis ALL Phase II
Bridgewater, NJ 16 years and older
(800) 633-1810
SL-701 Stemline Therapeutics glioma Phase I/II
(brain cancer vaccine) New York, NY pediatric patients
(212) 831-1111
Sprycel® Bristol-Myers Squibb chronic myeloid leukemia (CML), in clinical trials
dasatinib Princeton, NJ Philadelphia-positive ALL pediatric patients
(Orphan Drug) (800) 332-2056
Tarceva® Genentech recurrent/refractory ependymoma Phase II
erlotinib South San Francisco, CA 1 year – 21 years
OSI Pharmaceuticals (631) 962-0600
Farmingdale, NY
6 Medicines in Development Children 2012

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) 616-4000
Medicines in Development Children 2012 7

8. Medicines in Development for Children
Cancer
Product Name Sponsor Indication Development Status
Xgeva® Amgen recurrent or unresectable Phase II
denosumab Thousand Oaks, CA giant cell tumor of bone 12 years and older
(800) 772-6436
Yervoy® Bristol-Myers Squibb advanced/metastatic melanoma in clinical trials
ipilimumab Princeton, NJ 12 years – 17 years
(Orphan Drug) (800) 332-2056
Yondelis® Janssen Research Development soft tissue sarcoma Phase III
trabdectedin Raritan, NJ 15 years and older
(800) 526-7736
Cardiovascular Disease
Product Name Sponsor Indication Development Status
Cardiolite® Lantheus Medical Imaging Kawasaki disease Phase I/II completed
kit for the preparation of N. Billerica, MA 4 years – 16 years
technetium Tc99m sestamibi (800) 362-2668
for injection
CER-001 Cerenis Therapeutics acute coronary syndrome Phase II
Ann Arbor, MI (see also genetic) pediatric patients included
(734) 769-1110
cicletanine Gilead Sciences pulmonary arterial hypertension Phase II
(Orphan Drug) Foster City, CA 16 years and older
(800) 445-3235
Cozaar® Merck hypertension Phase III
losartan Whitehouse Station, NJ 6 months – 6 years
(800) 672-6372
Edarbi® Takeda Pharmaceuticals hypertension Phase I
azilsartan medoxomil North America 1 year and older
Deerfield, IL (877) 825-3327
Eliquis™ Bristol-Myers Squibb venous thromboembolism in clinical trials
apixaban Princeton, NJ pediatric patients
Pfizer (800) 332-2056
New York, NY (860) 732-5156
8 Medicines in Development Children 2012

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) 624-6900
Medicines in Development Children 2012 9

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) 354-8100
10 Medicines in Development Children 2012

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. 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) 436-1660
12 Medicines in Development Children 2012

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, NC
Medicines in Development Children 2012 13

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) 888-0660
14 Medicines in Development Children 2012

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) 766-4860
Medicines in Development Children 2012 15

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) 252-7500
16 Medicines in Development Children 2012

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) 357-9900
Medicines in Development Children 2012 17

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) 450-3500
18 Medicines in Development Children 2012

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) 806-1074
Medicines in Development Children 2012 19

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) 871-5500
20 Medicines in Development Children 2012

21. Medicines in Development for Children
Infectious Diseases
Product Name Sponsor Indication Development Status
GSK134612 GlaxoSmithKline meningococcal infections Phase III
(Men ACWY) Rsch. Triangle Park, NC 15 years and older
(888) 825-5249
------------------------------------------------- -------------------------------------------
meningococcal infections Phase III
4 years – 16 years
(888) 825-5249
-------------------------------------------------- -------------------------------------------
meningococcal infections Phase II
2 years – 6 years
(888) 825-5249
GSK1557484A GlaxoSmithKline pandemic H5N1 influenza Phase III
(influenza virus vaccine) Rsch. Triangle Park, NC 6 months – 17 years
(888) 825-5249
GSK2202083A GlaxoSmithKline meningococcal group C infections, Phase II
(heptavalent combination Rsch. Triangle Park, NC Haemophilus influenzae type b infants
vaccine) infection, diphtheria, hepatitis B, (888) 825-5249
tetanus, pertussis and poliomyelitis
Intelence® Tibotec Pharmaceuticals HIV infection Phase III
etravirine County Cork, Ireland 6 years – 18 years
Janssen Research Development (800) 526-7736
Raritan, NJ
Isentress® Merck HIV infection Phase I/II
raltegravir Whitehouse Station, NJ 2 years – 18 years
(800) 672-6372
ivermectin topical Topaz Pharmaceuticals pediculosis (head lice) application submitted
Horsham, PA pediatric patients
(215) 672-7140
Ixiaro® Intercell Japanese encephalitis Phase III
Japanese encephalitis Gaithersburg, MD 2 months – 17 years
vaccine (301) 556-4500
Lexiva® Vertex Pharmaceuticals HIV infection Phase II
fosamprenavir Cambridge, MA 2 years – 18 years
ViiV Healthcare (617) 444-6100
Rsch. Triangle Park, NC (877) 844-8872
-------------------------------------------------- -------------------------------------------
HIV infection Phase II
up to 2 years
(617) 444-6100
(877) 844-8872
Medicines in Development Children 2012 21