FOLD). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=FOLD as the Company's latest news hit the wire. On February 12, 2018, the Company announced that the US Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for filing under priority review for the oral precision medicine for migalastat HCl ("migalastat") for the treatment of patients 16 years and older with Fabry disease who have amenable mutations. The Prescription Drug User Fee Act (PDUFA) goal date for the FDA decision is August 13, 2018. Register today and get access to over 1000 Free Research Reports by joining our site below:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for Immunomedics, Inc. (NASDAQ: IMMU), which also belongs to the Healthcare sector as the Company Amicus Therapeutics. Do not miss out and become a member today for free to access this upcoming report at:

www.active-investors.com/registration-sg/?symbol=IMMU

Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Amicus Therapeutics most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

www.active-investors.com/registration-sg/?symbol=FOLD

NDA Submitted in December 2017

The Company submitted the NDA to the FDA on December 14, 2017. The NDA submission is based on existing clinical data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT) as well as other completed clinical studies.

EC Granted Approval for Migalastat Under Trade Name Galafold™, for Treatment of Fabry Disease

In May 2016, the European Commission (EC) granted full approval for migalastat under the trade name Galafold™ as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with Fabry disease (alpha-Gal A deficiency) and who have an amenable mutation. The EC approval was based on clinical data from two Phase-3 pivotal studies (FACETS and ATTRACT) as well as ongoing long-term extension studies. Outside the EU, migalastat is approved in Switzerland, Israel, Australia, South Korea, and Canada, with regulatory submissions under review in the US, Japan, and Taiwan.

FDA Fast Tracks Treatment for Fabry Disease

On January 31, 2018, FDA granted Fast Track designation to Protalix BioTherapeutics' pegunigalsidase alfa (PRX-102) for treatment for Fabry disease. PRX-102 also has orphan drug status in the United States. PRX-102 is the Protalix's plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate, and has demonstrated enhanced circulatory half-life, with higher enzyme activity in target organs affected by Fabry disease.

About Fabry Disease

Fabry disease is a rare genetic disease with a deficiency of an enzyme called alpha-galactosidase A. The disease affects many parts of the body including the skin, eyes, gastrointestinal system, kidney, heart, brain, and nervous system. The symptoms of the disease include episodes of pain and burning sensations, dark red spots on the skin, gastrointestinal problems, hearing loss, joint pain, etc., to name a few. Fabry disease is caused by a defect on the X chromosome. X-linked genetic mutations tend to occur mostly in males, and rarely in females.

About Migalastat

Migalastat, Amicus' lead product candidate, is an oral pharmacological chaperone that works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. A proprietary in vitro assay (Galafold Amenability Assay) has been used to classify more than 1,000 known GLA mutations as "amenable" or "not amenable" to treatment with migalastat. Migalastat has previously received both Orphan Drug Designation and Fast Track designation from the FDA.

About Amicus Therapeutics, Inc.

Founded in 2002, Amicus Therapeutics is a global, patient-centric biotechnology company focused on discovering, developing, and delivering novel high-quality medicines for people living with rare metabolic diseases. Headquartered in Cranbury, New Jersey, the Company's footprint spans to 27 countries.

Stock Performance Snapshot

February 13, 2018 - At Tuesday's closing bell, Amicus Therapeutics' stock fell 1.25%, ending the trading session at $14.62.

Volume traded for the day: 2.19 million shares.

Stock performance in the last three-month – up 13.42%; previous six-month period – up 17.71%; past twelve-month period – up 141.65%; and year-to-date - up 1.60%

After yesterday's close, Amicus Therapeutics' market cap was at $2.46 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

Active-Investors:

Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.

A-I has not been compensated; directly or indirectly; for producing or publishing this document.

PRESS RELEASE PROCEDURES:

The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst [for further information on analyst credentials, please email info@active-investors.com. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable-effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.

NO WARRANTY

A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.

NOT AN OFFERING

This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.

CONTACT

For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:

Email: info@active-investors.com

Phone number: 73 29 92 6381

Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: Active-Investors

ReleaseID: 489216

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MarketersMEDIA / Newsroom / Blog Exposure – FDA Accepts Amicus Therapeutics’ NDA for Migalastat for Treatment of Fabry Disease

Blog Exposure – FDA Accepts Amicus Therapeutics’ NDA for Migalastat for Treatment of Fabry Disease

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Stock Monitor: Immunomedics Post Earnings Reporting

LONDON, UK / ACCESSWIRE / February 14, 2018 / Active-Investors.com has just released a free research report on Amicus Therapeutics, Inc. (NASDAQ: FOLD). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=FOLD as the Company's latest news hit the wire. On February 12, 2018, the Company announced that the US Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for filing under priority review for the oral precision medicine for migalastat HCl ("migalastat") for the treatment of patients 16 years and older with Fabry disease who have amenable mutations. The Prescription Drug User Fee Act (PDUFA) goal date for the FDA decision is August 13, 2018. Register today and get access to over 1000 Free Research Reports by joining our site below:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for Immunomedics, Inc. (NASDAQ: IMMU), which also belongs to the Healthcare sector as the Company Amicus Therapeutics. Do not miss out and become a member today for free to access this upcoming report at:

www.active-investors.com/registration-sg/?symbol=IMMU

Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Amicus Therapeutics most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

www.active-investors.com/registration-sg/?symbol=FOLD

NDA Submitted in December 2017

The Company submitted the NDA to the FDA on December 14, 2017. The NDA submission is based on existing clinical data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT) as well as other completed clinical studies.

EC Granted Approval for Migalastat Under Trade Name Galafold™, for Treatment of Fabry Disease

In May 2016, the European Commission (EC) granted full approval for migalastat under the trade name Galafold™ as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with Fabry disease (alpha-Gal A deficiency) and who have an amenable mutation. The EC approval was based on clinical data from two Phase-3 pivotal studies (FACETS and ATTRACT) as well as ongoing long-term extension studies. Outside the EU, migalastat is approved in Switzerland, Israel, Australia, South Korea, and Canada, with regulatory submissions under review in the US, Japan, and Taiwan.

FDA Fast Tracks Treatment for Fabry Disease

On January 31, 2018, FDA granted Fast Track designation to Protalix BioTherapeutics' pegunigalsidase alfa (PRX-102) for treatment for Fabry disease. PRX-102 also has orphan drug status in the United States. PRX-102 is the Protalix's plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate, and has demonstrated enhanced circulatory half-life, with higher enzyme activity in target organs affected by Fabry disease.

About Fabry Disease

Fabry disease is a rare genetic disease with a deficiency of an enzyme called alpha-galactosidase A. The disease affects many parts of the body including the skin, eyes, gastrointestinal system, kidney, heart, brain, and nervous system. The symptoms of the disease include episodes of pain and burning sensations, dark red spots on the skin, gastrointestinal problems, hearing loss, joint pain, etc., to name a few. Fabry disease is caused by a defect on the X chromosome. X-linked genetic mutations tend to occur mostly in males, and rarely in females.

About Migalastat

Migalastat, Amicus' lead product candidate, is an oral pharmacological chaperone that works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. A proprietary in vitro assay (Galafold Amenability Assay) has been used to classify more than 1,000 known GLA mutations as "amenable" or "not amenable" to treatment with migalastat. Migalastat has previously received both Orphan Drug Designation and Fast Track designation from the FDA.

About Amicus Therapeutics, Inc.

Founded in 2002, Amicus Therapeutics is a global, patient-centric biotechnology company focused on discovering, developing, and delivering novel high-quality medicines for people living with rare metabolic diseases. Headquartered in Cranbury, New Jersey, the Company's footprint spans to 27 countries.

Stock Performance Snapshot

February 13, 2018 - At Tuesday's closing bell, Amicus Therapeutics' stock fell 1.25%, ending the trading session at $14.62.

Volume traded for the day: 2.19 million shares.

Stock performance in the last three-month – up 13.42%; previous six-month period – up 17.71%; past twelve-month period – up 141.65%; and year-to-date - up 1.60%

After yesterday's close, Amicus Therapeutics' market cap was at $2.46 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

Active-Investors:

Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.

A-I has not been compensated; directly or indirectly; for producing or publishing this document.

PRESS RELEASE PROCEDURES:

The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst [for further information on analyst credentials, please email info@active-investors.com. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable-effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.

NO WARRANTY

A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.

NOT AN OFFERING

This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.

CONTACT

For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:

Email: info@active-investors.com

Phone number: 73 29 92 6381

Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: Active-Investors

ReleaseID: 489216

Stock Monitor: Immunomedics Post Earnings Reporting

LONDON, UK / ACCESSWIRE / February 14, 2018 / Active-Investors.com has just released a free research report on Amicus Therapeutics, Inc. (NASDAQ: FOLD). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=FOLD as the Company's latest news hit the wire. On February 12, 2018, the Company announced that the US Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for filing under priority review for the oral precision medicine for migalastat HCl ("migalastat") for the treatment of patients 16 years and older with Fabry disease who have amenable mutations. The Prescription Drug User Fee Act (PDUFA) goal date for the FDA decision is August 13, 2018. Register today and get access to over 1000 Free Research Reports by joining our site below:

www.active-investors.com/registration-sg

Active-Investors.com is currently working on the research report for Immunomedics, Inc. (NASDAQ: IMMU), which also belongs to the Healthcare sector as the Company Amicus Therapeutics. Do not miss out and become a member today for free to access this upcoming report at:

www.active-investors.com/registration-sg/?symbol=IMMU

Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Amicus Therapeutics most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:

www.active-investors.com/registration-sg/?symbol=FOLD

NDA Submitted in December 2017

The Company submitted the NDA to the FDA on December 14, 2017. The NDA submission is based on existing clinical data, including reduction in disease-causing substrate (GL-3), as well as the totality of data from two Phase 3 pivotal studies in treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT) as well as other completed clinical studies.

EC Granted Approval for Migalastat Under Trade Name Galafold™, for Treatment of Fabry Disease

In May 2016, the European Commission (EC) granted full approval for migalastat under the trade name Galafold™ as a first line therapy for long-term treatment of adults and adolescents aged 16 years and older with Fabry disease (alpha-Gal A deficiency) and who have an amenable mutation. The EC approval was based on clinical data from two Phase-3 pivotal studies (FACETS and ATTRACT) as well as ongoing long-term extension studies. Outside the EU, migalastat is approved in Switzerland, Israel, Australia, South Korea, and Canada, with regulatory submissions under review in the US, Japan, and Taiwan.

FDA Fast Tracks Treatment for Fabry Disease

On January 31, 2018, FDA granted Fast Track designation to Protalix BioTherapeutics' pegunigalsidase alfa (PRX-102) for treatment for Fabry disease. PRX-102 also has orphan drug status in the United States. PRX-102 is the Protalix's plant cell-expressed recombinant, pegylated, cross-linked α-galactosidase-A candidate, and has demonstrated enhanced circulatory half-life, with higher enzyme activity in target organs affected by Fabry disease.

About Fabry Disease

Fabry disease is a rare genetic disease with a deficiency of an enzyme called alpha-galactosidase A. The disease affects many parts of the body including the skin, eyes, gastrointestinal system, kidney, heart, brain, and nervous system. The symptoms of the disease include episodes of pain and burning sensations, dark red spots on the skin, gastrointestinal problems, hearing loss, joint pain, etc., to name a few. Fabry disease is caused by a defect on the X chromosome. X-linked genetic mutations tend to occur mostly in males, and rarely in females.

About Migalastat

Migalastat, Amicus' lead product candidate, is an oral pharmacological chaperone that works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations. A proprietary in vitro assay (Galafold Amenability Assay) has been used to classify more than 1,000 known GLA mutations as "amenable" or "not amenable" to treatment with migalastat. Migalastat has previously received both Orphan Drug Designation and Fast Track designation from the FDA.

About Amicus Therapeutics, Inc.

Founded in 2002, Amicus Therapeutics is a global, patient-centric biotechnology company focused on discovering, developing, and delivering novel high-quality medicines for people living with rare metabolic diseases. Headquartered in Cranbury, New Jersey, the Company's footprint spans to 27 countries.

Stock Performance Snapshot

February 13, 2018 - At Tuesday's closing bell, Amicus Therapeutics' stock fell 1.25%, ending the trading session at $14.62.

Volume traded for the day: 2.19 million shares.

Stock performance in the last three-month – up 13.42%; previous six-month period – up 17.71%; past twelve-month period – up 141.65%; and year-to-date - up 1.60%

After yesterday's close, Amicus Therapeutics' market cap was at $2.46 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

Active-Investors:

Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.

A-I has not been compensated; directly or indirectly; for producing or publishing this document.

PRESS RELEASE PROCEDURES:

The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst [for further information on analyst credentials, please email info@active-investors.com. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable-effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.

NO WARRANTY

A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.

NOT AN OFFERING

This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.

CONTACT

For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:

Email: info@active-investors.com

Phone number: 73 29 92 6381

Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: Active-Investors

ReleaseID: 489216

Source URL: https://marketersmedia.com/blog-exposure-fda-accepts-amicus-therapeutics-nda-for-migalastat-for-treatment-of-fabry-disease/300934

Source: AccessWire

Release ID: 300934

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