hbm new drug approval report...hbm new drug approval report 2019 4 approvals for 2004 and after...

1HBM New Drug Approval Report 2019

HBM New Drug Approval ReportAnalysis of FDA New Drug Approvals in 2018

(and Multi-Year Trends)

For questions, comments or corrections please contact the authors of this report

Dr Ulrich Geilinger, [email protected] and Dr Chandra Leo, [email protected]

January 2019

The use of data and charts is permitted with reference to “HBM New Drug Approval Report”

Also available online with data of all new drugs approved by FDA since 2003

http://www.hbmpartners.com/en/industry-reports

Further Reading:

FDA 2018 New Drug Approval Report:

https://www.fda.gov/drugs/developmentapprovalprocess/druginnovation/ucm592464.htm

Fierce Biotech – List of new drugs approved in 2018:

https://www.fiercebiotech.com/special-report/two-years-after-sluggish-2016-new-drug-approvals-hit-their-stride-2018

mailto:[email protected]

mailto:[email protected]

http://www.hbmpartners.com/en/news-research/#tab_d858312ec47cb6a64ac33daec0044371_3




2018 New US Drug Approvals - Summary

▪ Record number of 59 (or 57*) new drugs (NMEs = New

Molecular Entities) approved by FDA in 2018

▪ Peak sales potential of new drugs approved in 2018

lower than in some previous years

▪ Lower number of new potential blockbusters approved

(13 in 2018 vs. 23 in 2017)

▪ Most new drugs approved in 2018 target orphan or

niche indications, 33 (or 68%) of new drugs with

Orphan Designation

▪ Record number of 11 new drugs approved target rare

genetic diseases

▪ Close to 50% of all new drugs approved in 2018 are

owned by smaller, innovative biopharma companies

(outside of the largest 50 pharma companies).

▪ Accessing novel drug candidates by licensing and

acquisition has become more important. And, on

average, less than 50% of approvals stem from

inhouse development efforts (true for company of all

sizes).

* HBM counts only 57 NMEs vs. FDA’s 59, see slide 3

FDA Drug Approval Numbers

2018 (and 2017)

59* NMEs

approved (46)

13 potential

blockbusters (23)

$40 billion peak sales

potential

($50 billion)

21 “First-in-

class” drugs (14)

16 cancer

drugs (13)

33 “Orphan”

drugs (17)

7 Priority Review

Vouchers awarded (6)

11 rare

genetic

disease

drugs (5)

5 anti-virals (3)

5 immunology

drugs (4)

4 antibiotics (4)

4 CNS/pain

drugs (2)


Record Number of New Drugs Approved by FDA in 2018

FDA’s CDER approved 59 NMEs

(New Molecular Entities) in 2018.

In the HBM analysis, we counted

only with 57 NMEs:

Braftovi/Mektovi, a combination

drug, is only counted as one NME

in our analysis

Injectable Akynzeo was omitted as

the combination drug was already

approved as an oral version in

2014.

There were also new biologics

approved by CBER in 2018 that

could be considered NMEs such as

Jivi (Hemophilia A bleeding control)

and Andexxa (aniticoagulant

reversal). These are not included in

the HBM analysis.

In previous years, new drugs

counted by FDA as NMEs also

included certain diagnostic or other

non-therapeutic agents. Thus, our

count of NMEs in some years is

slightly lower than the official FDA

NME count.

Source: FDA, HBM analysis

NME = new molecular entity, NCE = new chemcial entity, i.e. small molecule

26 6 6 6

211 12

612

1716

2015

2433

25

3033

16

34

42

18

39

27

41

22

46

59

17

26

21

28

35

25

40

45

19

45

57

0

10

20

30

40

50

60

2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

s A

pp

rove

db

yF

DA

NCEs approved (FDA count)

New biologics approved (FDA count)

Therapeutic NMEs approved (HBM count)

New Drugs (NMEs) Approved by FDA 2008-2019 (CDER Approvals)

▪ Highest number of new drug approvals ever (topping the 53

approvals in 1996)

▪ Record number NCEs (42) and biologics (17) approved

▪ However, lower average peak sales expected from new drugs

approved in 2018


Approvals for 2004 and after include Biologic

License Applications (BLAs) for therapeutic

biologic products transferred from the Center for

Biological Evaluation and Research (CBER) to the

Center for Drug Evaluation and Research (CDER).

FDA New Drug Approvals – Looking Back to 1990

23

3026 25

22

28

53

39

30

35

2724

1721

36

2022

18

2426

21

30

39

27

4145

22

46

59

0

10

20

30

40

50

60

1990 1995 2000 2005 2010 2015

# o

fN

ME

s A

pp

rove

db

yF

DA

Source: FDA, “Summary of NDA Approvals & Receipts, 1938 to the present”

https://www.fda.gov/ForConsumers/ConsumerUpdates/ucm2006085.htm

New Drug (NMEs) Approvals 1990-2018

1996-2003: + Average 4-5

new biologics approvals by

CBER p.a.

https://www.fda.gov/ForConsumers/ConsumerUpdates/ucm2006085.htm


Market Potential of 2018 Approvals Below Previous Highs

30.4

36.4

43.8

58.6

27.3

49.7

39.9

12.614.8

24.127.7

12.5

33.2

2012 2013 2014 2015 2016 2017 2018

Pea

k S

ales

Po

ten

tial

($

bill

ion

)

Worldwide

Peak Sales

Potential

Estimated

US Sales

5 Years

After Launch

(Evaluate-

pharma)

2018

Estimates

Not

Available

Yet

Worldwide Peak Sales Potential of New Drugs Approved 2012-2018(including expected US sales 5 years after approval)

Despite a record

number of FDA

approvals in 2018,

estimated peak sales

for all drugs* approved

in 2018 is lower than

in some previous

years.

Most new drugs

approved in 2018

target niche or orphan

markets and few (13)

have “blockbuster”

potential.

*NME approvals only

Estimated annual worldwide peak sales of NMEs approved were collected from analyst reports and other

sources. In case of several or differing estimates, we calculated the averages. Please note that in our analysis,

we use the worldwide peak sales of the new drugs as estimated at the time of approval. Thus, we do not

adjust the figures retrospectively when actual sales figures or new estimates become available.

Expected US peak sales 5 years after approval (a number frequently cited by some other sources such as

EvaluatePharma) are about 50% lower than our worldwide peak sales estimates.

Source: EvaluatePharma, HBM Analysis (from various sources)


Are Small Molecule Drugs Making a Comeback?

3.6 4.8

16.1 15.87.8

24.7

12.0

26.831.7

27.7

42.8

19.5

25.0

27.830.4

36.4

43.8

58.6

27.3

49.7

39.935

25

40

45

19

45

57

0

10

20

30

40

50

60

2012 2013 2014 2015 2016 2017 2018

Pea

k S

ales

Po

ten

tial

($

bill

ion

)

Number

of NMEs

Approved

(HBM count)

Worldwide

Peak Sales

Biologics

Worldwide

Peak Sales

NCEs

Total Worldwide

Peak Sales

Estimates

Estimated Peak-Sales Potential of NCEs & Biologics Approved 2012-2018

The 40 “traditional”

small molecule drugs

(NCEs) approved in

2018 are expected to

generate $27.8 billion

in worldwide sales.

Source: FDA, HBM Analysis / NME = new molecular entity / Biologics approved by CDER

Peak sales potential

of the 17 biologics

approved in 2018

dropped to $12

billion, even though

there was a record

number (17) of

biologics approvals.Some new biologics - that can be considered NMEs - were approved by CBER

(and not by CDER) and such products are not included in FDA’s list of novel

drugs and are also not included in our analysis.

For 2017, we estimated the market potential of such 6 products at $5.2 billion.

In 2018, there were only 2 such products (Jivi and Andexxa) with a combined

peak sales potential of $1.2 billion.


Fewer Potential Blockbusters Approved in 2018

Source: FDA, HBM Analysis

895

1'566

1'169

1'430 1'436

1'183

83110

1415

18

11

23

13

2012 2013 2014 2015 2016 2017 2018

Average WW Peak

Sales Potential

per New Drug

($ million)

Number of New Drugs

Approved With

Blockbuster Potential

(Peak Sales =>$1bn)

Average Peak Sales Potential of New Drugs Approved 2012-2018

Peak

Sales

Potential

Drug (Company) $ billion Indication

Biktarvy (Gilead

Sciences)

6.6 HIV

Ultomiris (Alexion) 2.3 Paroxysmal nocturnal

hemoglobinuria (PNH)

Symdeko (Vertex

Pharmaceuticals)

2.3 Cystic fibrosis

Orilissa (AbbVie) 1.9 Endometriosis

Crysvita (Ultragenyx) 1.8 X-linked hypophosphatemia

(XLH)

Erleada (Johnson &

Johnson)

1.7 Non-metastatic prostate

cancer

Takhzyro (Shire) 1.7 Hereditary angioedema

Olumiant (Eli Lilly) 1.5 Moderate to severe

rheumatoid arthritis

Epidiolex (GW

Pharmaceuticals)

1.3 Dravet & Lennox-Gastau

syndrome

Aimovig (Amgen) 1.3 Migraine

Libtayo (Regeneron

Pharmaceuticals)

1.2 Metastatic cutaneous

squamous cell carcinoma

(CSCC)Onpattro (Alnylam

Pharmaceuticals)

1.1 Familial Amyloid

Neuropathies

Lokelma

(AstraZeneca)

1.0 Hyperkalemia

▪ Average peak sales potential of new drugs approved

in 2018 dropped below $1 billion.

▪ Only 13 potential new blockbuster drugs were

approved in 2018 (as compared to 23 in 2017) …

▪ … with only 2 new potential blockbusters in oncology

in 2018 (vs. 10 in 2017)!

New Drugs Approved in 2018

With Largest Peak Sales Potential


Cancer and Genetic Diseases with Most Approvals

0

5

10

15

2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# of New Drugs Approved

Cancer

Genetic

CNS/Pain/Behaviour

Immunology

Infections viral


New Drug Approvals 2006-2018 in Selected Therapeutic Areas

▪ Highest number of new drug approvals in cancer

(16) and rare genetic diseases (11, a new record!).

▪ Approval numbers in viral infections, CNS and

immunology are also trending higher.

Anti-virals are a lucrative field for

new drug development. Drugs in

this area usually have a high sales

potential.

Since 2011, around 30% of

approvals have been in oncology

(as compared to approx. 20% in

previous years.

11 new orphan drugs to treat rare

genetic diseases were approved in

2018 with a total peak sales

potential of almost $10 billion!

In 2018, 4 new antibiotics were

approved (all with QIDP designation).

The total market potential of these

new drugs is rather modest ($1.2

billion expected peak sales).


New Drug Approvals in Oncology 2009-2018


▪ Considering the high number of cancer drug approvals in 2018,

potential revenues from these new drugs seems modest.

▪ The 16 new cancers drugs approved in 2018 amount to 28% of

all drug approvals (in-line with 5-year average).

3.01.4

6.2

10.6

14.312.3

17.7

7.5

16.9

9.05

2

8

13

8

10

16

4

13

16

0

5

10

15

20

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

Ap

pro

vals

Worldwide

Peak Sales

Potential

($ billion)

# of New Cancer

Drugs Approved

High Number of New Cancer Drug Approvals in 2018,

But “Modest” Total Peak Sales Potential

10 new cancer drugs with

“blockbuster” potential in

2017, but only 2 oncology

“blockbusters” in 2018!


New First-in-Class Drugs Approved 2009-2018


4 713

19

916 16

914

21

22 14

15

16

16

2429

10

31

36

15%

33%

46%

54%

36%40%

36%

47%

31%39%

0

5

10

15

20

25

30

35

40

45

50

55

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

Ap

pro

vals

# of

First-In-Class

Drugs

Approved

# of All

ApprovalsOther

Drugs

Approved

The High Number of “First-in-Class” Drugs Approved in 2018

Indicate that Biopharma is Focussing on “True Innovations”

First-in-class drugs are

“drugs which use a new

and unique mechanism

of action for treating a

medical condition”

(FDA definition).

The number of new

first-in-class drugs

approved is often used

as an indicator of true

innovation in pharma.

For 2018, FDA lists 20

new first-in-class drugs.

Including Epidiolex, the

first active ingredient

derived from marijuana,

generally considered a

“first-in-class” drug by

the industry, we count

21 new first-in-class

drugs in 2018.

“USA First”:

42 of the 59* novel drugs approved in 2018 (71%) were

approved in the US before getting approval in any other country.

* FDA count


Strong Increase in Orphan Drug Approvals since 2009

2 3 2 2 1 25

25

113

75 6

8

12

3

9

13

43

2 4 2

7

4

2

3

9

9

6

11 119

17

21

7

17

33

35%

29%

39%

31%36%

43%47%

37% 38%

58%

0

10

20

30

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# of New OrphanDrugs Approved

by FDA

Otherindications

Cancer

Geneticdiseases

% Share of All

Drugs Approved

New Orphan Drugs Approved by FDA 2009-2019


Note: Some other products approved by CBER also had Orphan Designation

2014 2015 2016 2017 2018 2014-18 $ billion

All Orphan Drugs 7 8 3 5 6 29 1.01

Cancer 4 4 1 4 - 13 0.97

Genetic - 2 1 - 5 8 1.05

Other Indications 3 2 1 1 1 8 1.05

New Orphan Drugs Approved 2014-2018

with Blockbuster Potential

Orphan drugs have (on

average) a somewhat

lower peak sales

potential than other

(non-orphan) drugs.

Still high prices paid for

genetic and other

orphan disease

treatments, quite a

number of orphan

drugs have “block-

buster” potential.

Orphan cancer drugs

are often expanded

into larger indications

thus leading to

substantial additional

revenues.

Average Peak Sales

Potential 2014-18

Orphan drug approvals

almost doubled from 2017

to 2018 (from 17 to 33)!


In 2018, 58% of New Drugs Approved Had “Orphan Drug Designation”

Genetic/11/19%

Cancer/13/23%

Other Orphan/9/16%

Non-Orphan/24/42%

Genetic/9%

Cancer/22%

Other Orphan/10%Non-Orphan/59%

(as compared to approx. 40% in the years before)

Orphan Drug Approvals in 2018 …

(as % of all approvals)

… and for years 2013-2017

Legend: Drug category or indication / Number of approvals / % of all approvals

Legend: Drug category / % of all approvals

33 New Orphan Drugs:

58% of all approvals

Orphan Drugs:

41% of all approvals

Companies with Highest

Number of Orphan Drug

Approvals 2009-2018

Companies outside of the

largest pharma companies

Company

# of Orphan

Drug

Approvals

Novartis 7

Pfizer 7

Roche 6

Johnson & Johnson 5

Bristol-Myers Squibb 5

AstraZeneca 5

Shire 4

GlaxoSmithKline 4

Alexion 3

Biomarin 3

Vertex Pharmaceuticals 3

Eli Lilly 3

Boehringer Ingelheim 3


Over 80% of Drugs Approved in 2018 Took Advantage of Some

Expedited Pathway (Priority Review, Fast Track etc.)

9 1014 13

10

24 24

13

28

44

35%

48% 50%

37% 40%

60%53%

68%62%

77%

0%

20%

40%

60%

80%

100%

0

10

20

30

40

50

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

Priority Reviews % of all Approvals

Drugs Approved under Priority Review 2009-2018

Source: FDA, HBM analysis

58%77%

40%25%

7% 7%

82%

42%23%

60%75%

93% 93%

18%

0%

20%

40%

60%

80%

100%

OD PR FT BT AA QIDP Any EP

% o

fN

ME

s A

pp

rove

d

Drugs Approved in 2018 Using Expedited Pathways (% of all approvals)

OD = Orphan Designation

PR = Priority Review

FT = Fast Track

BT = Breakthrough Therapy

AA = Accelerated Approvals

QIDP = Qualified Infectious

Disease Product

Any EP = Any expedited

pathway granted/ used

Significant increases seen in

approvals with Priority Review,

Fast Track, Breakthrough

Therapy and Orphan

Designation (both in absolute

numbers and % of all

approvals).

In 2018, only 4 drugs were granted

Accelerated Approval (vs. 6 each in

2015, 2016 and 2017)


Highest Number of Priority Review Vouchers Awarded in 2018

10 0

10

2

5

3

67

0

5

10

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# of PriorityReview

Vouchers Awarded

Priority Review Vouchers (PRVs) Awarded 2009-2018

Includes PRVs awarded for new drugs/treatments approved by CDER and CBER.

Source: FDA, www.priorityreviewvoucher.org

11 PRVs were sold (so far)

to other companies.

Average PRV transaction

values have come down to

around $80 million.

Considering that a

significant number of

PRVs are still unused, we

do not expect a recovery

of PRV values.

The Priority Review Voucher (PRV) Program was established in 2007.

Until the end of 2018 25 PRVs were awarded:

▪ 18 for rare pediatric diseases,

▪ 6 for neglected tropical diseases and

▪ 1 as a “Material Threat Medical Countermeasure” priority review voucher

(TPOXX).

http://www.priorityreviewvoucher.org/


US and European Pharma Companies Still Dominate US Drug Approvals

147

1622

8

25 24

13

29359

11

9

11

12

1015

6

11

15

3

2

1

2

4

3

6 3

4

1

21

2

2

3

0

10

20

30

40

50

60

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

s A

pp

rove

db

yF

DA

ROW (incl. Canada)

Japan

Europe

USA

Drug Owner/Sponsor - Headquarters Location by Country/Region


Drug Owner/Sponsor = Company that owned drug at time of approval (or had US marketing rights)

Only very few new drugs

approved were sponsored/

owned by “ROW companies”

with most of them being

Canadian companies.

In 2018, Australian-based

Medicines Development (with

Moxidectin), Indian-based Sun

Pharma (with Ilumya) and

Canadian-based Theratech-

nologies (with Trogarzo) got

one product approved each.

An analysis by the originators

of new drugs shows an even

stronger dominance by US

companies: 40 of new drugs

approved in 2018 were initially

developed by US companies.


Drug Approvals by Types of Drug Owner/Sponsors


▪ The share of new drug approvals of the Top 10 pharma companies has

declined in recent years.

▪ Smaller biopharma companies (definitions see box on the right) are playing

an increasing role not only as originators, but also as developers and owners

of drugs all the way to approval.

94

10 12 1318 16

916 14

4

4

45

8

8 13

3

665

85

43

45

2

59

85

914

1

10

11

5

18 28

35%

19%

36% 34%

52%

45%

36%

47%

36%

25%

0

10

20

30

40

50

60

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

Ap

pro

vals

Smaller Biopharma Co's

Mid-Sized Biopharma Co's

Other Large Biopharma Co's

Top 10 Pharma Co's

Top 10

Pharma‘s

Share of

Approvals

49% of all

approvals

in 2018!

Top 10 Pharma’s Share of New Drug Approvals Declining,

More Drugs from Smaller/Emerging Companies Get Approved

Definitions:

The “Drug Sponsor/Owner” is

the company that owns the

drug at time of approval or

has licensed it for US or

worldwide markets

“Top 10 pharma companies”:

Pharma companies ranked

within the top ten worldwide

by pharma sales.

“Other large biopharma

companies”: Biopharma

companies ranked between

11 and 30 in terms of

worldwide sales.

“Mid-sized biopharma

companies”: Companies with

significant sales of usually

between $100 million and

$1 billion.

“Smaller biopharma

companies”: = Other smaller

biopharma companies.


Drug Approvals by Size Drug Originator*


▪ That small, innovative biopharma companies have been active in

discovering and developing novel drugs is not a new

phenomenon.

▪ In absolute and relative (%) terms, smaller companies now

dominate the early development of new medicines.

82 4 7

27 9 6 7 9

3

46

56

10 74

46

73

32

4 7

1

7 5

88

1519

14

1822

12

2736

31%

38%

54% 54%56%

45%49%

63%60%

63%

0

10

20

30

40

50

60

2009 2010 2011 2012 2013 2014 2015 2016 2017 2018

# o

fN

ME

Ap

pro

vals Institutions, Others

Smaller Biopharma Co's

Mid-Sized Biopharma Co's

Other Large Biopharma Co's

Top 10 Pharma Co's

% of New Drugs Approved That Were Initially

Developed by Smaller Biopharma Co’s

The Majority of New Drugs Approved Originated at or

Were Initially Developed by Smaller Biopharma Companies

* The “Drug

Originator” is the

company that

discovered the drug or

undertook the first

serious clinical

development effort.

Note: A significant

number of new drugs

were originally

discovered at

universities or

research institutions

and then transferred

to a biopharma

company for initial or

further development.

We have listed such

institutions only as

“originators” if the

transfer to a company

occurred after pre-

clinical development.


Over 50% of New Drugs Approved Have Been

In-licensed or Acquired

41% 44% 36% 47%

30% 28%24%

42%

29% 28%40%

11%

0%

50%

100%

Top 10 Other Large Mid-Sized Smaller

% o

fN

ME

s A

pp

rove

d

Acquisition/M&A

In-Licensed

Inhouse Develooed

Origins of New Drugs Approved 2014-2018 By Type of Company (Drug Owner/Sponsor)

Companies of all sizes use in-licensing and

acquisitions to get access to novel drugs!

Biopharma Companies

Smaller/71%

Mid-Sized/13%

Large /7%

Top 10/9%

Origin of New Drugs Licensed or Acquired

(Approved 2014-2019) by Company Sizes

113 drugs (54% of all that were approved 2014-

18) “changed hands” before approval. 80 (or

71%) of those stem from smaller biopharma

companies (50/50 licensing vs. trade sales). Company Size

Total 113 of

drugs licensed

or acquired


Drug Owner/Sponsor = Company that owned drug at time of approval (or had US marketing rights)


HBM New Drug Approval Report - Notes

Which data is in the report (and which is not)?

The data for new drugs approved has been collected from FDA (www.fda.gov) and other sources.

The term “new drug” in this report is used for therapeutic new molecular entities (“NMEs”). This includes

new chemical entities “NCEs” (also called small molecules) and new biologics such as antibodies etc.

(classified by FDA as NMEs and approved by CDER).

Our data does not include FDA approvals for (a) new non-therapeutic agents such as imaging agents,

preventive vaccines etc., some of which are included in FDA’s older approval numbers and reports, (b)

BLA approvals by CBER such as fractioned plasma products and other biologics products etc.,

(c) approvals of previously FDA-approved drugs for new indications, (d) new combinations or

formulations of previously approved NMEs and (e) biosimilars.

In 2017 and 2018, we do provide data/information on selected BLAs approved by CBER for NME-like

products, but products are not included in the analysis, graphs etc.

We thus follow the definition by the FDA for «Novel Drugs» (but exclude new imaging and other non-

therapeutic agents, which were included mainly in earlier years by FDA).

The use of data and charts is permitted with reference to

“HBM New Drug Approval Report”

Further Reading

FDA 2018 New Drug Approval Report:


Fierce Biotech – List of new drugs approved in 2018 with some details:

https://www.fiercebiotech.com/special-report/two-years-after-sluggish-2016-new-drug-approvals-hit-

their-stride-2018

http://www.fda.gov/




About HBM Partners AG

HBM Partners is among the global leaders in healthcare-focused investing with

approximately $1.7 billion in assets under management. HBM focuses on

development stage, growth and buy-out financings of private and public

biopharma, medical device and diagnostics companies. Investments in private

companies usually range between $10 million to $50 million.

HBM Partners advises HBM Healthcare Investments AG (SIX: HMBN) as well

as other specialized private & public equity funds.

HBM Partners has been an active contributor to value creation in its portfolio

companies, generating over 60 trade sales and IPOs since its inception in 2001.

HBM Partners AG

Bundesplatz 1

CH-6300 Zug / Switzerland

Phone: +41 43 888 71 71

www.hbmpartners.com

http://www.hbmpartners.com/

hbm new drug approval report...hbm new drug approval report 2019 4 approvals for 2004 and after...

Documents