{"id":521042,"date":"2021-08-02T07:03:17","date_gmt":"2021-08-02T11:03:17","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-european-commission-approval-of-galafold-migalastat-for-adolescents-with-fabry-disease\/"},"modified":"2021-08-02T07:03:17","modified_gmt":"2021-08-02T11:03:17","slug":"amicus-therapeutics-announces-european-commission-approval-of-galafold-migalastat-for-adolescents-with-fabry-disease","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/amicus-therapeutics-announces-european-commission-approval-of-galafold-migalastat-for-adolescents-with-fabry-disease\/","title":{"rendered":"Amicus Therapeutics Announces European Commission Approval of Galafold\u00ae (migalastat) for Adolescents with Fabry Disease"},"content":{"rendered":"

\nGalafold\u00ae is the First and Only Oral Therapy Approved in the EU for the Long-term Treatment of Adolescents with Fabry Disease Aged 12 years and older with an Amenable Mutation
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PHILADELPHIA, Aug. 02, 2021 (GLOBE NEWSWIRE) — Amicus Therapeutics<\/u><\/a> (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced the European Commission has approved Galafold\u00ae<\/sup> (migalastat) for use in adolescents aged 12 to <16 years weighing \u2265 45 kg with a confirmed diagnosis of Fabry disease and who have an amenable mutation. Galafold is already approved in multiple geographies around the world, including the U.S., EU, and Japan, for adults who have an amenable\u00a0variant, or mutation.<\/p>\n

Bradley Campbell, President and Chief Operating Officer of Amicus Therapeutics, Inc., stated, \u201cThis approval of Galafold is a transformative moment for the Fabry community in the EU, as it gives those patients as young as 12 years of age with an amenable mutation a new treatment option for the first time in more than 15 years. We are grateful to this wonderful and passionate community, particularly the patients, physicians, and families who have made this research possible through their participation in the clinical trials, as well as to the regulators and our dedicated Amicus team. This achievement highlights our company\u2019s mission, which is focused on transforming the lives of people living with rare diseases.\u201d<\/p>\n

\u201cThis expanded approval is a significant step forward for the Fabry community, as we work towards increasing awareness of this rare disease in young people. It ensures patients, both paediatric and adult now have a convenient oral disease modifying therapy option available,\u201d said Uma Ramaswami, MD, Lysosomal Storage Disease Unit, Royal Free London NHS Foundation Trust.<\/p>\n

The extension of the indication was supported by 1-month interim safety and pharmacokinetics\u00a0data from Study AT1001-020 which was a 2-stage, open-label, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects aged 12 to <18 years and weighing \u2265 45 kg with Fabry disease and with amenable mutations to the gene encoding \u03b1-galactosidase A (GLA<\/em>).<\/p>\n

Amicus will work closely with all relevant government authorities to secure access for eligible patients as quickly as possible. Galafold is not approved for adolescents outside of Europe.<\/p>\n

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\n About Fabry Disease<\/u>
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\n Fabry disease<\/a>\u00a0is an inherited lysosomal 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 irreversible organ damage regardless of the time of symptom onset.<\/p>\n

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\n About Galafold<\/u>
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\n \u00a0<\/strong>
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Galafold\u00ae<\/sup>\u00a0(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\u00a0galactosidase alpha gene (GLA)<\/em>\u00a0variants. In these patients, Galafold works by stabilizing the body\u2019s 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\u00a0GLA<\/em>\u00a0variants, though amenability rates within this range vary by geography. Galafold is approved in over 40 countries around the world, including the U.S., EU, U.K., Japan and others.<\/p>\n

\n U.S. INDICATIONS AND USAGE<\/strong>
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Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA<\/em>) variant based on\u00a0in\u00a0vitro<\/em>\u00a0assay data.<\/p>\n

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.<\/p>\n

\n U.S. IMPORTANT SAFETY INFORMATION<\/strong>\n <\/p>\n

\n ADVERSE REACTIONS<\/strong>
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The most common adverse reactions reported with Galafold (\u226510%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.<\/p>\n

\n USE IN SPECIFIC POPULATIONS<\/strong>
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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.<\/p>\n

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\u2019s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.<\/p>\n

Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.<\/p>\n

The safety and effectiveness of Galafold have not been established in pediatric patients.<\/p>\n

To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at\u00a01-800-FDA-1088 or\u00a0www.fda.gov\/medwatch<\/a>.<\/p>\n

For additional information about Galafold, including the full U.S. Prescribing Information, please visit\u00a0https:\/\/www.amicusrx.com\/pi\/Galafold.pdf<\/a>.<\/p>\n

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\n EU Important Safety Information<\/u>
\n <\/strong>
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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.<\/p>\n