These slides studies the 2018 report by CDER on New Drug Therapy Approvals. Novel drugs were approved at an all time high of 59 (in contrast to the annual average of 33). Among notable approvals include monoclonal for HIV infection, non-opioid drugs for withdrawal symptoms, marijuana based drugs for seizures, and even contraceptive for women that can be used up to a year.

1. New Drug Therapies
Advancing Health Through Innovation
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2. 2018: Year Of Innovation for Drug Therapies
The Center for Drug Evaluation
and Research (CDER) under the
United States Food & Drug
Administration (FDA) approved a
wide variety of drugs.
This has helped a lot of patients
that are suffering from different
medical conditions. New drugs are
giving patients improved chances
of surviving life-threatening
illnesses.
Record approvals
over the past 2
years with 2018 at
an all-time high.
Source: CDER Report on 2018 New Drug Approvals
CDER drug
approvals in
2018 will
address
many unmet
needs.

3. Drug Classes Approved
Rare
Infectious
Neurological
Heart, Lungs
&
Circulatory
Women’s
Health
Cancer &
Blood
Source: CDER Report on 2018 New Drug Approvals

4. 2018 Highlights
Source: CDER Report on 2018 New Drug Approvals
UTI
Once-daily
antibiotic for the
treatment of
complicated
urinary tract
infections.
New Class of RNA-based
Gene-Silencing Drugs
First of a promising and entirely
new class of drugs called a small
interfering ribonucleic acid (siRNA).
Marijuana
First drug
derived from
marijuana for
treatment of
seizures.
Non-Opioid
First non-opioid
drugs to treat
opioid withdrawal
symptoms.
HIV Infection
First monoclonal
antibody approved
to treat HIV-1
infection.

5. Novel Drugs
Many of last year’s new
drugs therapies were novel
drugs. These drugs were
never before approved or
marketed in the US.
Novel drugs are often
innovative products that
serve previously unmet
medical needs, in addition
to significantly advancing
patient treatments
59 Novel Drugs were
approved by CDER
in 2018.
*Annual
average
is 33.
Source: CDER Report on 2018 New Drug Approvals

6. Novel Drugs TYPES
CDER considers
these new drugs
are notable
advances.
The active
ingredients or
ingredients in a
novel drug has
never been
approved in the US.
New Drug
Applications
(NDAs)
New
Molecular
Entities (NMEs)
Biologics License
Applications
(BLAs)
New
Therapeutic
Biologics
Source: CDER Report on 2018 New Drug Approvals

7. First-in-Class
19 of 59 novel
drugs approved
were 1st-in-class 32%
Source: CDER Report on 2018 New Drug Approvals
Galafold by Amicus
Therapeutics
Treat Fabry disease
(fat buildup in body cells)
Lucemyra by US Worldmeds
First non-opioid to reduce
opioid withdrawal symptoms
Trogarzo by Thera
Technologies
Monoclonal antibody for
adults living with HIV-1
These drugs are indicated to have strong positive impact on healthcare.
They often have mechanisms of actions different from existing therapies.

8. DRUGS FOR RARE Diseases
34 of 59 novel
drugs approved
to treat rare
diseases
58%
As at 2018, around 200,000 Americans are affected by rare or “orphan”
diseases. They often have few or no drugs available for treatment.
Source: CDER Report on 2018 New Drug Approvals
Crysvita by Kyowa Kirin
To treat rare, inherited form of
rickets (impaired bone growth)
Epidiolex by Greenwich
Biosciences
Treatment of seizures w/
epilepsy: Lennox-Gastaut &
Davet syndrome
Palinziq by BioMarin
Pharmaceutical
For phenylketonuria (PKU):
Inability to break down amino
acid phenylalanine

9. DRUGS FOR RARE Diseases: 2017 vs 2018
2018:
34 of 59 novel
drugs approved
to treat rare
diseases.
58%
In 2018, there was a major increase in the number of orphan drugs approved.
Source: US FDA 2018 New Drug Approvals
2017:
18 of 46 novel
drugs approved
to treat rare
diseases.
39%

10. Notable Infectious
Tpoxx by Siga
Technologies
Smallpox, under
USAMRIID as
biodefense
Krintafel by
GlaxoSmithKline
One-dose
treatment for
vivax malaria
Source: CDER Report on 2018 New Drug Approvals

11. Notable FOR Neurological
Almovig by Novartis,
Ajovy by Teva
Pharmaceuticals, &
Emgality by Lilly USA
Treat migraine for adults
Diacomit by BioCodex
France
Treat seizures in patients
with Dravet syndrome
*In the US, more than
38 million people
suffer from migraines.
Source: https://migraine.com/migraine-statistics/

12. Notable FOR Cancer & Blood Disorders
CANCER
Breast
Talzenna by
Pfizer
Prostate
Erleada by
Janssen Biotech
LEUKEMIA
(cancer of blood-
forming tissues)
Lymphocytic
Copiktra by
Verastem
Myeloid
Daurismo by
Pfizer
Tibsovo by Agios
Pharmaceuticals
Xospata by
Astellas Pharma
*In addition:
Droptelet by Dova
Pharmaceuticals is first
new therapy to treat
thrombocytopenia (low
blood platelet count) in
adults with chronic
liver disease.
Source: CDER Report on 2018 New Drug Approvals

13. Notable FOR Women’s Health
Annovera by Population
Council & TherapeuticsMD
Vaginal ring combined w/
hormonal contraceptive for
women (1st to use for entire
year)
Orilissa by AbbVie
Treat endometriosis (tissue
that lines the uterus grows in
other parts of the body)
* Endometriosis
affects 1 in 10 women
(ages 15-49), which is
approx. 176 million
women worldwide.
Source: http://endometriosis.org/resources/articles/facts-about-endometriosis/

14. Other Notable Drugs
Xofluza by
Genentech USA
Uncomplicated
influenza (flu)
Zemdri by Achaogen
Complicated urinary
tract infections (cUTI)
Firdapse by Catalyst
Pharmaceuticals
Treat symptoms of LEMS
Gamifant by Swedish Orphan
Biovitrum
Treat hemophagocytic
lymphohistiocytosis
Vitrakvi by Bayer & Loxo
Oncology
Locally advanced or metastatic
solid tumors
Source: CDER Report on 2018 New Drug Approvals

15. Bio-Similars
Biosimilars are highly similar to
already FDA-approved biological
product (reference).
Biological products are highly
complex, and often used to treat
patients with serious life-
threatening conditions.
Bio-similars create competition,
increase patient access and
potentially reduce cost of
important therapies.CDER has now approved a total of 16 biosimilars
for 9 different reference products since 2015.
Source: CDER Report on 2018 New Drug Approvals

16. Biologicals: 2014-2018
Source: US FDA 2018 New Drug Approvals
20
13
15
22
21
2014 2015 2016 2017 2018
First ever
approvals for gene
therapies in the US
Mainly vaccines,
clotting factors and
immunoglobulins

17. Notable Approved Bio-Similars
Retacrit by Celltrion & Teva
Pharmaceutical Industries
Breast or metastatic
stomach cancer (gastric or
gastroesophageal junction
adenocarcinoma)
Nivestym by Pfizer
Decreases chance of
infection during anti-cancer
drug treatment.
Retacrit by Pfizer
Anemia caused by
chronic kidney disease,
chemotherapy, or use of
zidovudine in patients
with HIV-1 infection
Hyrimoz by Sandoz
(Novartis)
Various arthritis,
Crohn's disease,
ulcerative colitis and
plaque psoriasis.

18. Key Designations / pathways
CDER used several regulatory pathways to expedite development and
approval of novel drugs in 2018.
24 of 59 novel drugs
designated for
Fast Track
41%
14 of 59 novel drugs
designated as
Breakthrough Therapies
24%
*For serious life-threatening
diseases for which there is
unmet medical need
*Potential to address unmet
medical needs
43 of 59 novel drugs
designated for
Priority Review
73%
*Potentially provide a
significant advance in
medical care
4 of 59 novel drugs
designated as
Accelerated Approval
7%
*Drug that offers a benefit over
current treatments for serious or
life-threatening illness
Source: CDER Report on 2018 New Drug Approvals

19. Worldwide revenue
Source: https://www.statista.com/statistics/263102/pharmaceutical-market-worldwide-revenue-since-2001/
In 2001, worldwide
revenue was around
390.2 billion U.S. dollars.
Ten years later, this
figure stood at some 963
billion U.S. dollars.
In 2014, global
pharmaceutical
revenues for the first
time increased to over
one trillion U.S. dollars.

20. Key Drivers
Source: EvaluatePharma World Preview 2018
+6.4% CAGR*
Worldwide
Prescription
Drug Sales
2018-2024.
*Compound Annual
Growth Rate
124 billion USD
Est. additional sales in 2024 from
‘orphan drugs’ - Synthetic
pharmaceutical which remains
commercially undeveloped.
5 billion USD
Additional sales
in 2024 from
advanced
therapies.
59 New
Approvals
For Novel Drugs
in 2018,
rebounding from
22 in 2016.
1.2 trillion
USD in 2024
For prescription
drug sales due to
novel therapies
addressing unmet
needs, and
increasing access
to medicines
globally.

21. By xeraya capital
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