FDA Approves Galafold™ (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease
First Precision and First Oral Medicine for Fabry Disease Approved for 348 Amenable GLA Variants
Galafold is the First New Treatment Option for Fabry Disease in the U.S. in 15+ Years
Amicus Therapeutics to Host Conference Call on
Galafold was approved under the Subpart H Accelerated Approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. As a condition of accelerated approval, Amicus Therapeutics will continue to study Galafold in a confirmatory Phase 4 program. Galafold is a capsule taken once every other day, at the same time of day. The FDA has approved Galafold for 348 amenable GLA variants. Galafold is the first oral medicine for Fabry disease, and the first new therapy approved to treat Fabry disease in the United States in more than 15 years.
John F. Crowley, Chairman and Chief Executive Officer of
Fabry disease is a rare, progressive genetic disorder characterized by a defective gene (GLA) that causes an enzyme deficiency. This enzyme is responsible for breaking down disease substrate that, when deficient in patients with Fabry disease, builds up in the kidneys, which is one of the organ systems impacted by Fabry disease. In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated. Globally, it is estimated that 35 to 50 percent of Fabry disease patients may have an amenable GLA variant.
“People with rare diseases often have limited treatment options, so research leading to new therapies is critical. As a physician and researcher, I treated the first patient ever to receive Galafold in a clinical trial. Today, many years later, I am pleased that more patients will have access to this precision medicine,” said Raphael Schiffmann, M.D., M.H.Sc., Director,
The FDA approval was based on data from a Phase 3 pivotal Fabry disease study in treatment-naïve patients (Study 011, or FACETS), which demonstrated the efficacy of Galafold, including a reduction in the amount of damaging disease substrate accumulated in the kidney capillaries. The most common side effects related to Galafold were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
“Today is a long-awaited day of celebration for all of us living with and advocating for people with Fabry disease, especially those who have participated in the development of Galafold in the United States,” said Jack Johnson, Founder and
Amicus Therapeutics will launch Galafold immediately, and will begin shipping to a limited distribution network in the coming week. Amicus Assist, a service that will provide product assistance and support to patients to help gain access to Amicus Therapeutics’ medications, will also be initiated immediately; for more information please call +1-833-AMICUS-A (+1-833-264-2872). With today’s approval, patients in 20 countries worldwide have reimbursable access to Galafold. Galafold is approved in Australia, Canada,
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast
An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the website 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at
GalafoldTM (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, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in Australia, Canada,
INDICATIONS AND USAGE
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.
U.S. IMPORTANT SAFETY INFORMATION
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 Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/galafold.pdf.
About Fabry Disease
Fabry disease is a rare, progressive genetic disorder characterized by a defective gene (GLA) that causes an enzyme deficiency. This enzyme is responsible for breaking down disease substrate that, when deficient in patients with Fabry disease, builds up in the kidneys, which is one of the organ systems impacted by Fabry disease.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-centric biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. The cornerstone of the Amicus portfolio is Galafold, an oral precision medicine for people living with Fabry disease who have amenable GLA variants. The lead biologics program in the Amicus Therapeutics pipeline is AT-GAA, an investigational therapy for Pompe disease. Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to approval and commercialization plans for Galafold in the United States. 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, 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 we may not be successful in commercializing Galafold in the United States, the potential that public and commercial payors will not reimburse Galafold, the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete all of our commercialization and manufacturing activities. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended
A video accompanying this announcement is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/673926ca-4565-41cf-9f11-f1171f59fd90
Logos accompanying this announcement are available at http://www.globenewswire.com/NewsRoom/AttachmentNg/0e1eab28-2505-45f8-9660-b1e7d3d9aaba and http://www.globenewswire.com/NewsRoom/AttachmentNg/e5aa5f58-56a0-428c-8b7f-c214e103a3f5
A PDF accompanying this announcement is available at http://resource.globenewswire.com/Resource/Download/167e6127-f1ea-45ba-ac0a-a28bf35b6fdc
Associate Director, Investor Relations
Media / Multimedia Assets:
PP GA ALL 0002 0718
PP GA ALL 0004 0818
Source: Amicus Therapeutics, Inc.