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
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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
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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.