Breaking News
June 21, 2018 - Christiana Care Health System opens first Epilepsy Monitoring Unit in Delaware
June 21, 2018 - CDC: Obesity Prevalence Higher in Non-Metropolitan Counties
June 21, 2018 - Youths Treated for Non-Suicidal Self Harm at Increased Risk of Suicide Within a Year
June 21, 2018 - Pediatric kidney recipients often have subclinical inflammation
June 21, 2018 - OHSU Knight Cancer Institute Director wins 2018 Tang Prize in Biopharmaceutical Science
June 21, 2018 - Researchers study broader effects of neonics on wildlife
June 21, 2018 - Study provides new insight on how antibiotics affect the gut microbiome
June 21, 2018 - InHealth Technologies becomes exclusive distributor of RENÚ Voice, RENÚ Gel in the United States
June 21, 2018 - New analysis links higher BMI to lower breast cancer risk for younger women
June 21, 2018 - Interactive preclinical PET-MR workshop demonstrates benefits of multi-modality imaging
June 21, 2018 - Gene signature could improve early diagnosis of TB
June 21, 2018 - Psychiatric Drug Lithium Tied to Birth Defect Risk
June 21, 2018 - Preclinical study suggests ARID1a may be useful biomarker for immunotherapy
June 21, 2018 - Risks of cancer and mortality found to be lowest in light drinkers
June 21, 2018 - Fetal immune cells are fast-acting first responders to microbes in adulthood
June 21, 2018 - Researchers invent medical device for proliferation, differentiation of neural stem cells
June 21, 2018 - Study explores current understanding of human physiology, pathology, trauma and surgery in space
June 21, 2018 - Scientists explore interactions between chromosomes 12 and 17
June 21, 2018 - People with severe obesity constantly try to reduce or control their weight
June 21, 2018 - Recovery and Treatment | NIH MedlinePlus the Magazine
June 21, 2018 - Study finds cell-free DNA profiling as versatile method to monitor UTIs
June 21, 2018 - ‘Hidden’ driver discovered that helps prime the anti-tumor immune response
June 21, 2018 - Groundbreaking discovery could be key to preventing cancer metastasis
June 21, 2018 - Impulse control disorders found to be more common in people taking Parkinson’s drugs
June 21, 2018 - Study finds possible link between Type 2 diabetes and common white pigment
June 21, 2018 - Most emergency department patients wish to be involved in medical decision-making
June 21, 2018 - Study highlights growing problem of ‘iPad neck’ among young adults and women
June 21, 2018 - UT Southwestern scientists identify gene mutation linked with perplexing brain condition
June 21, 2018 - Probiotics could cut age-related bone loss in elderly women
June 21, 2018 - New study sheds light on role of vitamin D in healthy pregnancy
June 21, 2018 - Teva Provides Update on Clinical Trial of Fremanezumab for Use in Chronic Cluster Headache
June 21, 2018 - Unlocking the secrets of HIV’s persistence
June 21, 2018 - New guidelines recommend newborns with Down’s syndrome to receive leukemia test
June 21, 2018 - BetterYou’s new Magnesium Bone Lotion helps maximize bone health
June 21, 2018 - UH scientist receives grant to examine understudied part of glaucoma
June 21, 2018 - Lifestyle intervention could normalize unhealthy behaviors that lead to cancer, chronic disease
June 21, 2018 - Combining two anti-malarial vaccines could greatly reduce number of infections
June 21, 2018 - By 2030, prostate and lung cancers expected to be most common cancers among HIV population
June 21, 2018 - Researchers evaluate patient satisfaction and well-being after breast reconstruction
June 21, 2018 - Combining stem cell technology and artificial intelligence to diagnose genetic cardiac diseases
June 21, 2018 - Monash study reveals why older people have reduced immune responses to cancer therapy
June 21, 2018 - Researchers develop new microscope system that can image living tissue in real time
June 21, 2018 - Long-term estrogen therapy alters microbial composition and activity in the gut
June 21, 2018 - Study points to dangers of feeding non-dairy drink to infants
June 21, 2018 - Cannabis Use Linked to Psychosis Symptoms in Adolescents
June 21, 2018 - Inadequate sleep could cost countries billions
June 21, 2018 - Inhibiting epigenetic proteins with drugs could prevent development of breast cancer
June 21, 2018 - Study identifies factors that contribute to vaginal dryness
June 21, 2018 - Researchers employ nucleotide-based gene silencing to mitigate common ataxia
June 21, 2018 - New tool determines best treatment plan for adults with severe asthma
June 21, 2018 - Identifying gene variants that contribute to ovarian reserve may improve female fertility
June 21, 2018 - Religious involvement has no significant effect on misuse of prescription opioids, study finds
June 21, 2018 - Researchers characterize key signaling network that drives growth of triple negative breast cancers
June 21, 2018 - AHA: Big Weight Gain in 1st Pregnancy Could Boost Preeclampsia Risk
June 21, 2018 - How vaping helps even hardened smokers quit
June 21, 2018 - Gaming disorder an official disease condition says WHO
June 21, 2018 - Oxygen consumption in human BAT increases after a meal, shows research
June 21, 2018 - Research finds addictions to be diseases of the brain, not criminal behavior or personality disorders
June 21, 2018 - New study is testing safety and efficacy of glycoside in breaking up kidney stones
June 21, 2018 - Biosimilar competition may offer hope for cheaper, better psoriasis treatments in the future
June 21, 2018 - Cells form cage-like structures that trap viruses
June 21, 2018 - Wound protector use linked to significant reduction in surgical site infection
June 21, 2018 - African Americans and Latinos are more likely to be at risk for depression than Whites
June 21, 2018 - Genome study presents new way to track historical demographics of US populations
June 20, 2018 - Study sheds light on the early stages of tumorigenesis
June 20, 2018 - Quick adoption of new diagnostic tests could help NHS to save nearly £7 billion
June 20, 2018 - Mayo Clinic researchers find genetic mutations that increase person’s risk for pancreatic cancer
June 20, 2018 - Chemists invent new method for remote chiral induction
June 20, 2018 - Methadone or buprenorphine associated with reductions in opioid related mortality
June 20, 2018 - New simulation approach predicts efficacy of HIV-prophylaxis medications
June 20, 2018 - Rhythm Pharmaceuticals Completes Pivotal Enrollment in Two Ongoing Phase 3 Clinical Trials Evaluating Setmelanotide in Rare Genetic Disorders of Obesity
June 20, 2018 - Threat of malaria left its mark on the immune system in people with African ancestry
June 20, 2018 - Recent-onset type 2 diabetes may be early expression of pancreatic cancer
June 20, 2018 - Why are vaccines less effective in the developing world?
June 20, 2018 - Study opens new window into cellular events that occur in the brain during absence seizures
June 20, 2018 - Humana and Walgreens to provide easier access to primary care and other services for seniors
June 20, 2018 - ANU research could help find life in Mars and other planets
June 20, 2018 - Multidisciplinary Human-Focused Research
June 20, 2018 - ‘Mind-minded’ parents contribute greatly to development of infants’ emotion regulation capacity
June 20, 2018 - New study finds increase in use of alternative medicines among children
Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

image_pdfDownload PDFimage_print

migalastat

Treatment for Fabry Disease

Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

CRANBURY, N.J., Dec. 14, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to request approval of the oral precision medicine migalastat HCl (“migalastat”) for the treatment of patients 16 years and older with Fabry disease who have amenable mutations. 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.

Migalastat previously received both Orphan Drug Designation and Fast Track designation from the U.S.FDA. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing, after which the Agency will notify the Company. Amicus plans to communicate following written receipt of the Agency’s decision.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Today marks the very first Amicus submission of a new drug application to the U.S.FDA. This important milestone is the culmination of a strong collaboration and commitment among the patients, physicians and Amicus employees who have spent more than a decade to advance migalastat. On the heels of our initial launch success for migalastat in Europe, our goal is to further expand access for more Fabry patients with amenable mutations in the U.S., Japan and other global geographies. We look forward to working with the FDA during the review process and to a potential U.S. approval of migalastat in 2018.”

An estimated 3,000 people in the U.S. are currently diagnosed with Fabry disease, more than any other country. Fabry disease is a progressive, inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The disease causes accumulation of specific lipids, primarily GL-3, in tissues including the heart, kidneys, central nervous system, and skin. This abnormal accumulation can lead to debilitating consequences including pain, kidney failure, heart disease, and stroke.

Migalastat works by stabilizing the body’s own dysfunctional alpha-Gal A enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations (an estimated 35% to 50% of Fabry patients globally). 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 Galafold. 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 a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation. The EC approval was based on clinical data from two Phase 3 pivotal studies in both treatment-naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT), as well as ongoing long-term extension studies.

Outside the EU, migalastat is approved in Switzerland, Israel, Australia and Canada, with regulatory submissions under review in Japan and additional geographies.

About Fabry Disease

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of 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 the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the clinical development, regulatory approval pathway, and prospects and timing of regulatory submission and approval of our product candidates for the treatment of Fabry disease. Any express or implied statements contained in this press release that are not statements of historical fact, including interpretation of guidance given by the U.S.FDA may be deemed forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved in a timely manner or at all. 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, 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, changes in FDA guidance for regulatory approval, risks regarding the FDA’s interpretation of our clinical trial results, including the risk that results from completed clinical trials that supported approval by regulators in other jurisdictions will not be sufficient for U.S.FDA purposes, the risk that the FDA will require additional studies or data, the risk that the timing of an NDA will be delayed or not be accepted for filing by the FDA, the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidate and the potential that we may not be successful in commercializing our product candidates for Fabry disease in Europe or any other country in which approval is ultimately obtained, if any. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2016 and the Quarterly Report for the quarter ended September 30, 2017. The FDA guidance described in this release was given as of a specific date and the FDA could change its position on the clinical end points or other standards for review and/or approval. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

Source: Amicus Therapeutics, Inc.

Posted: December 2017

Related Articles

migalastat FDA Approval History

Tagged with:

About author

Related Articles