AIDS Awareness

2012 Report
Medicines in Development
HIV/AIDS
presented by americas biopharmaceutical
research companies
Biopharmaceutical Researchers Are Testing

More Than 70 Medicines and Vaccines For
HIV Infection
for HIV/AIDS
40
1.2 million Americans

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are living with HIV,

50,000 are newly diagnosed
each year
* Some medicines are listed in more than one

phase of development.
Biopharmaceutical research companies

are developing 73 medicines and vaccines,
focusing on improved treatment regimens,
more effective therapies and promising new
preventative vaccines.
Although HIV/AIDS is one of the most
devastating diseases affecting people around
the world, the number of new infections has
been steadily declining. In the United States,
the AIDS-related death rate has fallen by 79
percent due to antiretroviral therapy.
Over the past 30 years, nearly 40 medicines
have been approved to treat HIV/AIDS.
Testing for the disease also has advanced
dramatically, enabling earlier treatment. While
these medicines have helped to prolong the
lives of HIV-infected patients, making HIV a
manageable chronic disease, opportunities for
even greater progress remain.
For example, biopharmaceutical companies
are intensifying their efforts to develop vaccines that would help prevent HIV. Current
estimates show that a 50 percent effective
vaccine given to only a third of the population

could reduce new HIV infections by 24 percent
over 15 years.
The medicines in the development pipeline
include:
A
gene therapy that uses genetic material to
remove disease-causing aspects of the virus.
A
transdermal vaccine that helps suppress virus replication and destroys HIV-infected cells.
A first-in-class medicine intended to prevent the
HIV virus from breaking through the cell membrane.
Despite the incredible progress to date, the
HIV/AIDS epidemic remains a complex problem. Americas biopharmaceutical research
companies are continuing their efforts to develop novel and more effective therapies, vaccines to prevent the disease, and potentially a
cure, so the millions of patients suffering today
have hope for a better tomorrow.
Medicines in Development for HIV/AIDS
Antivirals
Product Name
Sponsor
Indication
Development Status*
abacavir/dolutegravir/lamivudine
fixed-dose combination
(integrase inhibitor/reverse
transcriptase inhibitor)
ViiV Healthcare
Rsch. Triangle Park, NC
HIV infection therapy in

treatment-naive patients
Phase III
(877) 844-8872
amdoxovir (DAPD)
RFS Pharma
Tucker, GA
HIV-1 infection treatment
Phase II
(404) 601-1430
BI-224436
(integrase inhibitor)
Gilead Sciences
Foster City, CA
HIV infection treatment
Phase I completed
(800) 445-3235
CB1922
(synthetic steroidal lactone)
Canopus BioPharma
Studio City, CA
Phase II
www.canopusbiopharma.com
cenicriviroc
(CCR5 receptor antagonist)
Tobira Therapeutics
South San Francisco, CA
Phase II
(650) 741-6625
CMX157
(tenofovir PIM conjugate)
Merck
Whitehouse Station, NJ
Phase I completed
(800) 672-6372
cobicistat
(PK enhancer)
Gilead Sciences
Foster City, CA
application submitted
(800) 445-3235
cobicistat/darunavir
(PK enhancer/protease
inhibitor)
Gilead Sciences
Foster City, CA
Janssen Therapeutics
Titusville, NJ
HIV infection
Phase I
(800) 445-3235
(800) 526-7736
cobicistat/darunavir/
emtricitabine/GS-7340
Gilead Sciences
Foster City, CA
Titusville, NJ
HIV-1 infection
Phase II
(800) 445-3235
(800) 526-7736
cobicistat/elvitegravir/
emtricitabine/GS-7340
Gilead Sciences
Foster City, CA
HIV-1 infection
Phase II
(800) 445-3235
dapivirine
(NNRTI)
International Partnership for

Microbicides
Silver Spring, MD
HIV infection prevention

(vaginal ring)
-------------------------------------------------HIV infection prevention
(vaginal gel)
Phase I/II completed

(301) 608-2221
------------------------------------------Phase I/II completed
(301) 608-2221
dolutegravir
(S/GSK1349572)
Shionogi
Florham Park, NJ
ViiV Healthcare
Phase III
(973) 966-6900
(877) 844-8872
*For more information about a specific medicine in this report, please call the telephone number listed.
Medicines in Development HIV/AIDS 2012
Antivirals
Product Name
Sponsor
Indication
Development Status
efavirenz/lamivudine/
tenofovir fumarate
Mylan Laboratories
Canonsburg, PA
(724) 514-1800
elvitegravir
Gilead Sciences
Foster City, CA
(800) 445-3235
elvucitabine
(NRTI)
Achillion Pharmaceuticals
New Haven, CT
Phase II
(203) 624-7000
GS-7340
(NtRTI)
Gilead Sciences
Foster City, CA
Phase II
(800) 445-3235
HIV attachment inhibitor
Bristol-Myers Squibb
Princeton, NJ
Phase II
(800) 332-2056
HIV maturation inhibitor
Princeton, NJ
in clinical trials
(800) 332-2056
ibalizumab
(TMB-355)
(fusion inhibitor)
TaiMed Biologics USA

Irvine, CA

(intravenous) (Fast Track)
-------------------------------------------------HIV-1 infection treatment
(subcutaneous)
Phase II
(949) 769-6543
------------------------------------------Phase I
(949) 769-6543
Intelence
etravirine
(NNRTI)
Titusville, NJ
HIV infection combination therapy in

treatment-naive patients
(Fast Track)
Phase II
(800) 526-7736
KD-247
(monoclonal antibody)
Kaketsuken
Kumamoto, Japan
Phase I
www.kaketsuken.or.jp
KP-1461
(replication inhibitor)
Koronis Pharmaceuticals
Redmond, WA
Phase II
(425) 825-0240
lamivudine (3TC)/lopinavir/
ritonavir fixed-dose
combination
Abbott Laboratories
Abbott Park, IL
in clinical trials
(847) 937-6100
lamivudine (3TC)/maraviroc/
zidovudine fixed-dose
combination
GlaxoSmithKline
HIV infection
Phase I completed
(888) 825-5249
lersivirine (UK-453061)
(NNRTI)
ViiV Healthcare
Phase II
(877) 844-8872
Lexiva
fosamprenavir
(PI)
Vertex Pharmaceuticals
Cambridge, MA
ViiV Healthcare
HIV infection treatment in

adolescents, children and infants
Phase II
(617) 444-6100
(877) 844-8872
Antivirals
Product Name
Sponsor
Indication
Development Status
MK-1439
(NNRTI)
Merck
Whitehouse Station, NJ
Phase I
(800) 672-6273
Norvir
ritonavir
powdered formulation
(PI)
Abbott Laboratories
Abbott Park, IL
in clinical trials
(847) 937-6100
NRT inhibitor
Princeton, NJ
Phase II
(800) 332-2056
Prezista
darunavir
(once-daily 800 mg)
Titusville, NJ
HIV infection
(800) 526-7736
PRO 140
CytoDyn
Lake Oswego, OR
HIV-1 infection prevention and

treatment
Phase II completed
(971) 204-0382
RAP101
RAPID Pharmaceuticals
Huenenberg, Switzerland
Phase II
www.rapidpharma.com
RPI-MN
ReceptoPharm
Plantation, FL
Phase I
(954) 321-8988
S/GSK1265744
Shionogi
Florham Park, NJ
ViiV Healthcare
Phase II
(973) 966-6900
(877) 844-8872
SPL-7013
(vaginal gel)
Starpharma
Melbourne, Australia

(Fast Track)
Phase I completed
www.starpharma.com
TBR-220
Tobira Therapeutics
South San Francisco, CA
Phase I
(650) 741-6625
tenofovir vaginal gel

(NtRTI)
CONRAD
Arlington, VA
International Partnership for
Microbicides
Silver Spring, MD
Phase I
(703) 524-4744
TMC310911
(PI)
Titusville, NJ
Phase II completed
(800) 526-7736
Antivirals
Product Name
Sponsor
Indication
Development Status
UB-421
(FI)
United Biomedical
Hauppauge, NY
Phase II
(631) 273-2828
VRX806
(NNRTI)
Valeant Pharmaceuticals
Mississauga, Canada
Phase II
(905) 286-3000
Cell Therapy/Gene Therapy

Product Name
Sponsor
Indication
Development Status
HIV gene therapy
Adaptimmune
Philadelphia, PA
Cardiff University
Cardiff, Wales
University of Pennsylvania
Philadelphia, PA
HIV infection
Phase I
(267) 499-2066
lexgenleucel-T
(replication inhibitor)
VIRxSYS
Gaithersburg, MD
HIV infection therapy in

treatment-experienced patients
Phase II
(301) 987-0480
SB-728-T
Sangamo BioSciences
Richmond, CA
Phase II
(510) 970-6000
Stealth Vector
HGTV-43
antisense gene medicine
Enzo Therapeutics
New York, NY
Phase I/II
(212) 583-0100
Immunomodulators
Product Name
Sponsor
Indication
Development Status
AMZ0026
Amazon Biotech
New York, NY
Phase I/II
(212) 444-1019
CYT107
(recombinant human
interleukin-7)
Cytheris
Rockville, MD
Phase II
(301) 231-0450
Cytolin
anti-CD8 mAb
CytoDyn
Lake Oswego, OR
Phase I
(971) 204-0382
IRT-103
(low-dose naltrexone)
TNI BioTech
New York, NY
Phase II
www.tnibiotech.com
Vaccines
Product Name
Sponsor
Indication
Development Status
ADVAX
(DNA vaccine)
Aaron Diamond AIDS Research

Center
New York, NY
International AIDS Vaccine Initiative
New York, NY
Ichor Medical Systems
San Diego, CA
Phase I completed
(212) 448-5000
(212) 847-1111
------------------------------------------Phase I completed
(212) 448-5000
(212) 847-1111
AGS-004
(autologous dendritic cell
vaccine-intradermal injection)
Argos Therapeutics
Durham, NC
Phase II
(919) 287-6300
AVX101
(single gene HIV vaccine)
AlphaVax
HIV-1 infection prevention
Phase I
(919) 595-0400
DCVax-001
(recombinant protein vaccine)
Celldex Therapeutics
Needham, MA
Rockefeller University
New York, NY
HIV infection prevention and

treatment
Phase I
(781) 433-0771
DermaVir Patch
DNA topical patch vaccine
Genetic Immunity
McLean, VA
Phase II
(703) 879-6803
HIV gp41 vaccine
Mymetics
Epalinges, Switzerland
Phase I
www.mymetics.com
HIV recombinant vaccine
GlaxoSmithKline
Phase I
(888) 825-5249
HIV vaccine
Crucell
Leiden, The Netherlands
Beth Israel Deaconess Medical
Center
Boston, MA
International AIDS Vaccine Initiative
New York, NY
Phase I
(212) 847-1111
HIV vaccine
GeoVax Labs
Smyrna, GA
Phase II
(678) 384-7220
HIV vaccine
GeoVax Labs
Smyrna, GA
Phase I/II
(678) 384-7220
-------------------------------------------------HIV infection prevention

(new delivery system)
Vaccines
Product Name
Sponsor
Indication
Development Status
HIV vaccine
Massachusetts General Hospital

Boston, MA
Opal Therapeutics
Parkville, Australia
HIV infection
Phase I
(617) 726-2000
HIV vaccine
Novartis Vaccines & Diagnostics

Cambridge, MA
National Institutes of Health
Bethesda, MD
HIV infection
Phase I
(617) 871-7000
HIV vaccine
PaxVax
San Diego, CA
in clinical trials
(858) 450-9595
HIV vaccine
(MAG pDNA)
Profectus Biosciences
Baltimore, MD
Phase I
(866) 938-8559
HIV vaccine
(rVSV)
Profectus Biosciences
Baltimore, MD
Phase I
(866) 938-8559
HIV vaccine
(SAV001)
Sumagen
Seoul, South Korea
HIV-1 infection
Phase I
www.sumagen.co.kr
HIVAX
replication-defective
HIV-1 vaccine
GeneCure Biotechnologies
Norcross, GA
HIV-1 infection
Phase I
(770) 263-7508
ITV-1
immune therapeutic vaccine
Immunotech Laboratories
Pasadena, CA
in clinical trials
(818) 409-9091
Pennvax-B
DNA vaccine (clade B)
Inovio Pharmaceuticals
Blue Bell, PA
HIV infection prevention and

treatment
Phase I
(267) 440-4200
Pennvax-G
DNA vaccine (clades A, C, D)
Inovio Pharmaceuticals
Blue Bell, PA
Phase I
(267) 440-4200
TUTI-16
(lipoprotein vaccine)
Thymon
Short Hills, NJ
Phase I/II
(973) 467-9558
vacc-4x
(intradermal vaccine)
Bionor Pharma
Oslo, Norway
Phase II
www.bionorpharma.com
Vaccines
Product Name
Sponsor
Indication
Development Status
VRC-HIVADV014-00-VP
(HIV-1 recombinant adenovirus
vaccine)
GenVec
Gaithersburg, MD
Vaccine Research Center (NIAID)
Bethesda, MD
Phase II completed
www.vrc.nih.gov
VRC-HIVADV027-00-VP
(HIV adenovector Ad35 vaccine)
GenVec
Gaithersburg, MD
Bethesda, MD
Phase I
www.vrc.nih.gov
VRC-HIVDNA016-00-VP

Bethesda, MD
Phase II
www.vrc.nih.gov
The content of this report has been obtained through public, government and industry sources, and the Adis R&D Insight database based on the
latest information. Report current as of November 16, 2012. The information in this report may not be comprehensive. For more specific information about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development
is available on PhRMAs web site.
A publication of PhRMAs Communications & Public Affairs Department. (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info
Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright 2012 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.
Pharmaceutical Research and Manufacturers of America 950 F Street, NW, Washington, DC 20004
Approved Medicines for HIV Infection/AIDS
Entry Inhibitors

Selzentry (maraviroc) ViiV Healthcare

Fuzeon (enfuvirtide) Genentech, Trimeris
Integrase Inhibitor

Isentress (raltegravir) Merck
Nucleoside Reverse Transcriptase Inhibitors (NRTI)

Combivir (lamivudine/zidovudine) ViiV Healthcare
Emtriva (emtricitabine) Gilead Sciences
Epivir (lamivudine) ViiV Healthcare
Epzicom (abacavir/lamivudine) ViiV Healthcare
Hivid (zalcitabine) Roche, marketing discontinued
Retrovir (zidovudine) ViiV Healthcare
Trizivir (abacavir/lamivudine/zidovudine) ViiV Healthcare
Videx (didanosine) Bristol-Myers Squibb
Videx EC (didanosine delayed release) Bristol-Myers Squibb
Zerit (stavudine) Bristol-Myers Squibb
Zerit XR (stavudine extended-release) Bristol-Myers Squibb, marketing discontinued
Ziagen (abacavir) ViiV Healthcare
Non-Nucleoside Reverse Transcriptase Inhibitors (NNRTI)

Edurant (rilpivirine) Janssen Therapeutics
Intelence (etravirine) Janssen Therapeutics
Rescriptor (delvaridine) ViiV Healthcare
Sustiva (efavirenz) Bristol-Myers Squibb
Viramune (nevirapine) Boehringer Ingelheim Pharmaceuticals
ViramuneXR (nevirapine extended-release) Boehringer Ingelheim Pharmaceuticals
Approved Medicines for HIV Infection/AIDS
Nucleotide Reverse Transcriptase Inhibitor (NtRTI)

Viread (tenofovir disoproxil fumarate) Gilead Sciences
Protease Inhibitors

Agenerase (amprenavir) GlaxoSmithKline, Vertex Pharmaceuticals
Aptivus (tipranavir) Boehringer Ingelheim Pharmaceuticals
Crixivan (indinavir) Merck
Fortovase (saquinavir soft-gel) Roche, marketing discontinued
Invirase (saquinavir) Genentech
Kaletra (lopinavir/ritonavir) Abbott Laboratories
Lexiva (fosamprenavir) ViiV Healthcare, Vertex Pharmaceuticals
Norvir (ritonavir) Abbott Laboratories
Prezista (darunavir) Janssen Therapeutics
Reyataz (atazanavir) Bristol-Myers Squibb
Viracept (nelfinavir) ViiV Healthcare
Combination Medicines
NNRTI/NRTI/NtRTI

Atripla (efavirenz/emtricitabine/tenofovir disoproxil fumarate) Bristol-Myers Squibb, Gilead Sciences
NRTI/NNRTI/NtRTI

Complera (emtricitabine/rilpivirine/tenofovir, disoproxil fumarate) Gilead Sciences
Integrase Inhibitor/PK Enhancer/NRTI/NtRTI
Stribild (elvitegravor/cobicistat/emtricitabine/tenofvoir disoproxil fumarate) Gilead Sciences
NRTI/NtRTI

10
Truvada (emtricitabine/tenofovir disoproxil fumarate) Gilead Sciences
Glossary
application submittedAn application for

marketing has been submitted by the company
to the Food and Drug Administration (FDA).
entry inhibitorUnlike other HIV drugs that
work after HIV has entered the human immune
cell, entry inhibitors work outside the CD4
cell, blocking the virus from entering the cell.
The process of HIV entry into a cell requires a
series of steps in sequence involving several
key proteins. Different entry inhibitors target
separate proteins in the process. One type of
entry inhibitor blocks the attachment of the HIV
protein gp120 to CD4 cell receptors on the cell
surface. Another inhibitor targets the binding
of the virus to CCR5 or CXCR4 co-receptors
involved in the virus entering the cell. And a
third entry inhibitor interferes with the fusion of
the HIV virus with T-cells at the cell membrane.
in the virus replication, blocking it can halt

further spread of the virus.
PK enhancerPharmacokinetic (PK) enhancer increases the effectiveness of pharmaceutical treatment.
reverse transcriptase inhibitor (RTI)When
HIV infects a cell, reverse transcriptase
changes the single-stranded RNA into a
double-stranded viral DNA. The new viral DNA
is then integrated into the human DNA cells, allowing reproduction of the virus. RTIs block this
action and prevent completion of synthesis of
the double-stranded viral DNA, preventing HIV
from multiplying. RTIs are a class of antiretroviral drugs.
NRTINucleoside reverse transcriptase
inhibitor.
HIV infectionThe presence of antibodies in

the blood to the human immunodeficiency virus
(the virus that causes AIDS). HIV-1 refers to
the most common strain of the virus found in
U.S. AIDS patients.
NNRTINon-nucleoside reverse transcriptase

inhibitor.
integrase inhibitor A class of antiretroviral

drugs designed to block the action of integrase,
an enzyme that inserts the virus into the DNA
of human cells. Since integration is a vital step
Phase 0First-in-human trials conducted

in accordance with FDAs 2006 guidance on
exploratory Investigational New Drug (IND)
studies designed to speed up development of
NtRTINucleotide reverse transcriptase

inhibitor.
promising drugs by establishing very early on

whether the agent behaves in human subjects
as was anticipated from preclinical studies.
Phase IResearchers test the drug in a small
group of people, usually between 20 and 80
healthy adult volunteers, to evaluate its initial
safety and tolerability profile, determine a
safe dosage range, and identify potential side
effects.
Phase IIThe drug is given to volunteer
patients, usually between 100 and 300, to see
if it is effective, identify an optimal dose, and to
further evaluate its short-term safety.
Phase IIIThe drug is given to a larger, more
diverse patient population, often involving between 1,000 and 3,000 patients (but sometime
many more thousands), to generate statistically
significant evidence to confirm its safety and
effectiveness. They are the longest studies,
and usually take place in multiple sites around
the world.
PIProtease inhibitors are a class of antiretroviral drugs used to treat HIV infection. They
prevent the HIV virus from replicating by inhibiting the activity of proteases, such as HIV-1.
11
Selected Facts about HIV/AIDS
Overview
U.S. AIDS Diagnoses through 20101
Adults/Adolescents
Pediatric (under age 13)
TOTAL
U.S. AIDS Deaths through 20091
1,119,651
614,394
9,475
4,986
1,163,575*
641,976*
* Because totals for the estimated numbers were calculated independently of the values for the subpopulations, the subpopulation values may not
equal these totals.
HIV/AIDS Worldwide 2
In 2010, 2.7 million people became newly infected with HIV infection (including 390,000 children younger than age 15), down from
3.1 million in 2001. Although the annual number of people newly infected with HIV has dropped since its peak in the late 1990s, it is still occurring at an unacceptably high rate: between 2.5 million and 3 million people annually for the past five years, adding to the global number of people
living with HIV that reached 34 million (including 3.4 million children younger than age 15) by the end of 2010.
Globally, the annual number of people newly infected with HIV continues to decline, although there is stark regional variation. In sub-Saharan
Africa, where most of the people newly infected with HIV live, an estimated 1.9 million people became infected in 2010. That was 16 percent
fewer than the estimated 2.2 million people newly infected with HIV in 2001, and 27 percent fewer than the annual number of people newly
infected between 1996 and 1998, when the incidence of HIV in sub-Saharan Africa peaked overall.
Reductions in the number of people acquiring HIV infection, especially people ages 1524 in the countries in sub-Saharan Africa that have a
high burden of HIV, have been offset by increases in new infections in Eastern Europe and Central Asia. In those areas, where the primary
mode of transmission of HIV is among people who inject drugs and their sexual networks, the number of people dying from AIDS-related
causes increased 1,100 percent during the past decade: from an estimated 7,800 in 2001 to 89,500 in 2010.
The annual number of people dying from AIDS-related causes worldwide is steadily decreasing from a peak of 2.2 million in 2005 to an
estimated 1.8 million in 2010. That year, an estimated 250,000 children younger than age 15 died from AIDS-related causes, 20 percent fewer
than in 2005. The number of people dying from AIDS-related causes began to decline in 20052006 in sub-Saharan Africa, South and Southeast
Asia and the Caribbean and has continued subsequently.
Introducing antiretroviral therapy has averted 2.5 million AIDS deaths in low- and middle-income countries globally since 1995. SubSaharan Africa accounts for the vast majority of the averted deaths: about 1.8 million.
Providing antiretroviral prophylaxis to pregnant women living with HIV has prevented more than 350,000 children from acquiring HIV
infection since 1995. Eighty-six percent of the children who avoided infection live in sub-Saharan Africa, the region with the highest prevalence
of HIV infection among women of reproductive age.
12
Selected Facts about HIV/AIDS
HIV/AIDS in the United States 1
In 2010, an estimated 48,298 people were newly diagnosed with HIV infection in the 46 states and 5 U.S. dependent areas with confidential
name-based HIV infection reporting. In the 46 states, 46,912 adults and adolescents were newly diagnosed with HIV infection, with 37,045 diagnoses in males and 9,868 diagnoses in females. Among children younger than age 13, there were an estimated 217 diagnoses of HIV infection.
At the end of 2009, an estimated 1,148,200 people age 13 and older were living with HIV infection in the United States, including 207,600
people whose infections had not been diagnosed.
In 2009, the estimated number of deaths of people with a diagnosis of HIV infection in the 46 states and 5 U.S. dependent areas with
confidential name-based HIV infection reporting was 21,601. In the 46 states only, that included 21,007 adults and adolescents and 8 children
younger than age 13.
In 2010, the estimated number of people diagnosed with AIDS in the United States and 6 U.S. dependent areas was 33,630. In the 50 states and
the District of Columbia, 24,749 AIDS diagnoses were among adult and adolescent males, 8,242 were among adult and adolescent females, and 23
diagnoses were among children younger than age 13.
In 2009, the estimated number of deaths of people with an AIDS diagnosis in the United States and 6 U.S. dependent areas was
18,234. In the 50 states and the District of Columbia, that included 17,770 adults and adolescents and 4 children younger than age 13.
HIV/AIDS Economic Impact
The lifetime treatment cost of an HIV infection can be used as a conservative threshold value for the cost of averting one infection. Currently,
the lifetime treatment cost of an HIV infection is estimated at $379,668 (in 2010 dollars); therefore, a prevention intervention is deemed
cost-saving if its cost-effectiveness (CE) ratio is less than $379,668 per infection averted. The average annual cost of HIV care in the
antiretroviral (ART) era is estimated to be $19,912 (in 2006 dollars; $23,000 in 2010 dollars). One study has estimated the medical savings
from infections averted by United States prevention programs from 1991-2006 to be $129.9 billion with 361,878 HIV infections averted.1
Nearly 30 years into the HIV epidemic, HIV continues to take a heavy toll in the United States. More than 1.1 million people are currently
living with HIV, nearly 18,000 people with AIDS still die each year, and lifetime medical care for those who become infected with HIV
each year is estimated to cost $20 billion.1
Without intervention, a perinatal HIV transmission rate of 25 percent would result in 1,750 HIV-infected infants born annually in the
United States with lifetime medical costs estimated to be $282 million. The cost of intervention (HIV counseling, testing, and zidovudine treatment) was estimated to be $67.6 million. That intervention would prevent 656 pediatric HIV infections, saving $105.6 million in
medical care costsa net cost-savings of $38.1 million annually.3
Sources:
1. U.S. Centers for Disease Control and Prevention, HIV Surveillance Report: Diagnoses of HIV Infection and AIDS in the United States and
Dependent Areas, 2010; Vol. 22., www.cdc.gov
2. World Health Organization (WHO), www.who.int/en
3. KidSource OnLine, Inc., www.kidsource.com
13
The Drug Discovery, Development and Approval Process
Developing a new medicine takes an average of 10-15 years;

For every 5,000-10,000 compounds in the pipeline, only 1 is approved.
The Drug Development and Approval Process

The U.S. system of new drug approvals is
perhaps the most rigorous in the world.
It takes 10-15 years, on average, for an
experimental drug to travel from lab to U.S.
patients, according to the Tufts Center for the
Study of Drug Development. Only five in 5,000
compounds that enter preclinical testing make
it to human testing. And only one of those five
is approved for sale.
On average, it costs a company $1.2 billion,
including the cost of failures, to get one new
medicine from the laboratory to U.S. patients,
according to a 2007 study by the Tufts Center
for the Study of Drug Development.
in people. The IND shows results of previous

experiments; how, where and by whom the
new studies will be conducted; the chemical
structure of the compound; how it is thought
to work in the body; any toxic effects found in
the animal studies; and how the compound
is manufactured. All clinical trials must be
reviewed and approved by the Institutional
Review Board (IRB) where the trials will be
conducted. Progress reports on clinical trials
must be submitted at least annually to FDA and
the IRB.
Once a new compound has been identified in

the laboratory, medicines are usually developed as follows:
Clinical Trials, Phase IResearchers test

the drug in a small group of people, usually
between 20 and 80 healthy adult volunteers, to
evaluate its initial safety and tolerability profile,
determine a safe dosage range, and identify
potential side effects.
Preclinical Testing. A pharmaceutical company conducts laboratory and animal studies

to show biological activity of the compound
against the targeted disease, and the compound is evaluated for safety.
Clinical Trials, Phase IIThe drug is given

to volunteer patients, usually between 100 and
300, to see if it iseffective, identify an optimal
dose, and to further evaluate its short-term
safety.
Investigational New Drug Application (IND).

After completing preclinical testing, a company
files an IND with the U.S. Food and Drug
Administration (FDA) to begin to test the drug
Clinical Trials, Phase IIIThe drug is given to

a larger, more diverse patient population, often
involving between 1,000 and 3,000 patients (but
sometime many more thousands), to generate
statistically significant evidence to confirm its

safety and effectiveness. They are the longest
studies, and usually take place in multiple sites
around the world.
New Drug Application (NDA)/Biologic
License Application (BLA). Following the
completion of all three phases of clinical trials,
a company analyzes all of the data and files an
NDA or BLA with FDA if the data successfully
demonstrate both safety and effectiveness.
The applications contain all of the scientific
information that the company has gathered.
Applications typically run 100,000 pages or
more.
Approval. Once FDA approves an NDA or
BLA, the new medicine becomes available
for physicians to prescribe. A company must
continue to submit periodic reports to FDA,
including any cases of adverse reactions and
appropriate quality-control records. For some
medicines, FDA requires additional trials
(Phase IV) to evaluate long-term effects.
Discovering and developing safe and effective
new medicines is a long, difficult, and expensive
process. PhRMA member companies invested
an estimated $49.5 billion in research and
development in 2011.

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2012 Report

Biopharmaceutical Researchers Are Testing

1.2 million Americans

are living with HIV,

* Some medicines are listed in more than one

Biopharmaceutical research companies

vaccine given to only a third of the population

Medicines in Development for HIV/AIDS

HIV infection therapy in

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment

International Partnership for

HIV infection prevention

Phase I/II completed

HIV-1 infection treatment

Medicines in Development HIV/AIDS 2012

Medicines in Development for HIV/AIDS

HIV-1 infection treatment

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment

HIV attachment inhibitor

HIV infection treatment

HIV maturation inhibitor

HIV infection treatment

TaiMed Biologics USA

HIV-1 infection treatment

HIV infection combination therapy in

HIV-1 infection treatment

HIV infection treatment

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment in

Medicines in Development HIV/AIDS 2012

Medicines in Development for HIV/AIDS

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment

HIV-1 infection prevention and

HIV infection treatment

HIV infection treatment

HIV infection treatment

HIV infection prevention

HIV infection treatment

tenofovir vaginal gel

HIV infection prevention

HIV infection treatment

Medicines in Development HIV/AIDS 2012

Medicines in Development for HIV/AIDS

HIV-1 infection treatment

HIV infection treatment

Cell Therapy/Gene Therapy

HIV gene therapy

HIV infection therapy in

HIV infection treatment

HIV-1 infection treatment

HIV infection treatment

HIV infection treatment

HIV infection treatment

HIV infection treatment