Full-Year 2020 Galafold® Revenue of ~$261M Exceeds Guidance
Strengthened Galafold IP Portfolio Now Includes 24 Issued Patents Including 13 Patents through 2038
Pompe Phase 3 PROPEL Study Last Patient, Last Visit Complete with Data Expected in 1Q2021
AT-GAA Pompe Clinical and CMC Milestones On-Track to Support 2021 BLA and MAA
Multiple Data and Manufacturing Updates and Advancements Planned Across Industry-Leading Rare Disease Gene Therapy Portfolio
Cash Position Sufficient to Achieve Self-Sustainability
Over the previous year, Amicus substantially met or exceeded its strategic priorities, highlighted by:
$261 million of global product revenue for Galafold driven by strong adoption and patient adherence- Completed last patient, last visit of the AT-GAA Phase 3 PROPEL clinical study, advanced manufacturing activities, and initiated the rolling Biologics License Application (BLA) with the
U.S. Food and Drug Administration (FDA) - Advanced clinical development and manufacturing for CLN6 and CLN3 Batten programs
- Progressed Pompe gene therapy and disclosed Fabry as the next gene therapy IND candidate
- Maintained strong financial position
Amicus is focused on the following five key strategic priorities in 2021:
- Achieve continued double-digit growth and Galafold revenue of at least
$300 million+1 - Report data from the AT-GAA Phase 3 PROPEL study and complete BLA and MAA filings for regulatory approvals
- Advance clinical studies, regulatory discussions and scientific data across industry leading gene therapy pipeline
- Further manufacturing capabilities and capacity to build world-class technical operations to support all gene therapy programs
- Maintain strong financial position
1 Guidance range to be provided on full-year earnings call.
Full-Year 2020 Galafold Summary and 2021 Guidance
Global revenue for Galafold in full-year 2020 was approximately
For the full-year 2021, the Company anticipates total Galafold of revenue at least
Updates and Anticipated 2021 Milestones by Program
Galafold (migalastat) Oral Precision Medicine for People Living with Fabry Disease and have an Amenable Variant
- Continued revenue growth in 2021 of at least
$300 million+. Guidance range to be provided on full-year earnings call. - Following the issuance of 11 new patents covering a range of treatment methods, Galafold has 24 issued patents, 13 of which extend IP protection into 2038
- Plans to expand EU label to cover adolescent population
- Continued geographic expansion
- Registry and other Phase 4 studies
AT-GAA For Pompe Disease
- Report data from the AT-GAA Phase 3 PROPEL study in 1Q2021
- Complete the BLA submission in 1H2021 and the EU MAA submission to be completed in 2H2021
- Ongoing supportive studies, including an open-label study in 12- to <18-year-olds living with Pompe and plans to initiate additional pediatric studies in 2021
Gene Therapy Pipeline
- As part of the
J.P. Morgan Conference virtual presentation, Amicus will highlight initial preclinical proof of concept data in CLN1 Batten disease and its plans on initiating an early-stage gene therapy program in Angelman Syndrome - Advance manufacturing and regulatory discussions to finalize clinical and regulatory path for the CLN6 Batten disease gene therapy program and begin dosing additional patients with GMP grade material
- Report initial data from the CLN3 Batten disease Phase 1/2 study, advance manufacturing and regulatory discussions to finalize clinical and regulatory path, and submit IND for next clinical study
- Continue IND-enabling work in both Pompe and Fabry
- Additional preclinical data and potential IND candidate declarations across multiple preclinical programs
- Manufacturing advancements and updates across the portfolio
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable GLA variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally,
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact
For additional information about Galafold, including the full
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
- Galafold is not intended for concomitant use with enzyme replacement therapy.
- Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children 0–15 years of age have not yet been established.
- No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
- There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse.
- While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk.
- Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold.
- OVERDOSE: General medical care is recommended in the case of Galafold overdose.
- The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.
About Fabry Disease
Fabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which results from mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including heart, kidneys, and skin. Accumulation of GL-3 and progressive deterioration of organ function is believed to lead to the morbidity and mortality of Fabry disease. The symptoms can be severe, differ from person to person, and begin at an early age.
About Amicus Therapeutics
Forward Looking Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product. In addition to the impact of the COVID-19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in
Non-GAAP Financial Measures
In addition to financial information prepared in accordance with
CONTACT:
Investors:
Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Head of
dmoore@amicusrx.com
(609) 662-5079
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