A T T H E F O R E F R O N T O F

T H E R A P I E S F O R R A R E D I S E A S E S

43rd Annual J.P. Morgan Healthcare Conference

January 13, 2025

Our Passion is for Patients

Our Mission:

We seek to deliver the highest quality therapies for people living with rare diseases

Our Vision:

Be a leader in rare disease drug development and commercialization leveraging our global capabilities in bringing life-changing therapies to patients

3

Definition:

əˈmēkəs (noun) Latin Friend

A Rare Company

A unique story in biotech with significant revenue growth and profitability

~$528M

Medicine for

10-15%

First Two-

for Pompe Disease

First Oral

Component Therapy

Precision

2024 Total Revenue1

Fabry Disease

FY 2025

(+32% Growth)2

Galafold Revenue

Growth2

65-85%

Non-GAAP

Global

$1B+

FY 2024

Leverageable

FY 2025

Profitability

Commercial

Total Revenues

Pombiliti+ Opfolda

Achieved

Organization

Expected in 2028

Revenue Growth2

4

1

Preliminary and unaudited

2

At CER: Constant Exchange Rates

2024 Strategic Priorities

Delivered

Galafold® revenue growth of 11-16% at CER1, raised to 16-18%

Execute multiple successful launches of Pombiliti® + Opfolda®

Advance ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases

Achieve non-GAAP profitability for the full year

5

5

1CER: Constant Exchange Rates;

2024 Key Milestones

Highest Patient Demand for Galafold

Since Early Launch

Galafold: A Fast Growing Treatment in Fabry

Disease & SoC in Amenable Population

Reduced OPEX Guidance and Judiciously

Managed Expenses

6

2025 Strategic Priorities

Deliver total revenue growth of 17-24% at CER1

Double-digit Galafold® revenue growth of 10-15% at CER1

Pombiliti®+ Opfolda® revenue growth of 65-85% at CER1

Advance ongoing studies to broaden labels and strengthen scientific leadership in Fabry and Pompe diseases

Deliver positive GAAP Net Income during H2 2025

7

1CER: Constant Exchange Rates

Galafold® (migalastat)

Continued Growth

Building a leadership position

in the treatment of Fabry disease

8

Fabry Disease Overview

Fabry is a rare inherited genetic disorder caused by mutation in GLA gene and deficiency of α-Gal A enzyme

  • >1,000 known mutations
  • >18,000 diagnosed WW
  • X-linkeddominant disease leading to diagnosis of multiple family members

Leading Causes of Death

TRANSIENT ISCHEMIC

ATTACK (TIA) & STROKE1

HEART DISEASE2

Irregular heartbeat (fast or slow)

Heart attack or heart failure

Enlarged heart

KIDNEY DISEASE3

Protein in the urine

Decreased kidney function

Kidney failure

Life-limiting Symptoms

GASTROINTESTINAL3

PAIN3

Nausea, vomiting,

cramping, diarrhea

FATIGUE3

Pain/bloating after eating,

feeling full

Constipation

ANHIDROSIS3

Difficulty managing weight

1 Desnick R, et al. Ann Intern Med. 2003

9

2

Yousef Z, et al. Eur Heart J. 2013

3

Germain D. Orphanet J Rare Dis. 2010

Global Fabry Market

Fabry market expected to grow to ~$3B by 2029

(millions)

$3,500

$3,000

$2,500

$2,000

$1,500

$1,000

$500

$0

Global Fabry market of ~$2.1B in 2024 and

tracking toward ~$3B+ by the end of the decade1

Significantly underdiagnosed

- Newborn screening studies suggest Fabry is one

of the more prevalent rare genetic diseases

(~1:1,500 to ~1:4,000 incidence)4

Continued market growth driven by increased

diagnosis

Anticipate market size for amenable patients

to surpass $1B in 2029

Galafold continues to be the greatest

//

//

contributor to market growth

2017

20242

2029E

Amenable to Galafold3

1 Global market measured by reported sales of approved therapies for Fabry disease - 2029 sales projected using ~7% CAGR

2

LTM ended September 30, 2024

10

3

Assumes ~40% amenability to Galafold

4 Burton 2017 J Pediatr 2017;190:130-5 ; Mechtler et al., The Lancet, 2011 Dec.; Hwu et al., Hum Mutation, 2009 Jun; Spada et al., Am J Human Genet.,

2006 Jul

Fabry Market Dynamics

Number of people on a Fabry treatment has more than doubled since 2015

2015 Fabry Market

2024 Fabry Market

5,000

6,000

~10K

18K

~

Patients

Patients

5,000

12,000

Treated

Diagnosed Untreated

6,000 diagnosed untreated patients remain

111 Based on Amicus data on file

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Disclaimer

Amicus Therapeutics Inc. published this content on January 13, 2025, and is solely responsible for the information contained herein. Distributed by Public, unedited and unaltered, on January 13, 2025 at 03:47:02.770.