GENESIS Pharma announces a commercialization agreement with Amicus Therapeutics® for migalastat in Greece, Cyprus, Romania and Bulgaria
GENESIS Pharma, a leading regional biopharma company operating in the broader region of Southeast Europe, announces that Amicus Therapeutics, a biotechnology company at the forefront of rare and orphan diseases, has granted the company exclusive rights to commercialize migalastat in Greece, Cyprus, Romania and Bulgaria. The European Commission granted full approval of migalastat in May 2016. Under the terms of the agreement, GENESIS Pharma will be responsible for the sales and marketing, medical affairs, regulatory and reimbursement support for migalastat in the above markets.
Mr. Constantinos Evripides, Chief Executive Officer of GENESIS Pharma
stated: “We are very pleased and honored to further broaden our international partners’ network with a company as innovative and committed as Amicus Therapeutics. Our strategic goal has always been to enhance our collaborations so as to ensure patient access to innovative therapies, especially in challenging therapeutic areas where there are unmet medical needs. Rare diseases have always been a top priority for us, since medical innovation can make an essential difference in patients’ lives. Once available, migalastat will add to our broad orphan disease portfolio that currently includes medicines in the therapeutic areas of hematology, oncology and neurology.”
Migalastat, an oral small molecule pharmacological chaperone, is the first oral treatment as well as the first precision medicine for Fabry disease. In Europe, migalastat is approved 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 (Ref. 1). The broad label includes 313 Fabry-causing mutations which represent 35 percent to 50 percent of all patients with Fabry disease (Ref. 2).
“We are very pleased to be partnering with GENESIS Pharma to commercialize migalastat in Greece, Cyprus, Romania and Bulgaria,” commented Bradley L Campbell, President & Chief Operating Officer at Amicus Therapeutics
. “This collaboration provides a great opportunity to serve people living with Fabry in these regions as it brings together Amicus, a biotechnology company at the forefront of rare and orphan diseases and the extensive experience of GENESIS Pharma” he added.
About migalastat and Amenable Mutations
: Migalastat is a first-in-class chaperone therapy approved in the EU as a monotherapy for Fabry disease in patients with amenable mutations (Ref. 3). Migalastat works by stabilizing the body's own dysfunctional enzyme, so it can clear the accumulation of disease substrate in patients who have amenable mutations (Ref.4 ). A proprietary in vitro assay (Migalastat Amenability Assay) was used to classify more than 800 known GLA mutations as "amenable" or "not amenable" to treatment with migalastat (Ref. 5). The current label includes all 313 GLA mutations that have been identified and determined to be amenable based on the Migalastat Amenability Assay, which represent between 35% and 50% of the currently diagnosed Fabry population (Ref. 2). Healthcare providers in the EU may access a specialized electronic platform to quickly and accurately identify which mutations are categorized as "amenable" or "not amenable" to migalastat. Amicus expects to submit updates to the label as additional GLA mutations are identified and tested in the Migalastat Amenability Assay (Ref. 1,5). Migalastat is not currently commercial available in the Greek market.
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 are the result of mutations in the GLA gene5. 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) (Ref. 3). 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 (Ref. 5,6). 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 (Ref. 5,7). All Fabry disease is progressive and leads to organ damage regardless of the time of symptom onset (Ref. 3).
About Amicus Therapeutics
: Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, 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
, and follow them on Twitter and LinkedIn.
1. Migalastat Prescribing Information. Amicus Therapeutics, Inc., .http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/004059/human_med_001981.jsp&mid=WC0b01ac058001d124 , Accessed Oct 10, 2018
2. Amicus Therapeutics, Inc. Amicus Therapeutics launches Migalastat (migalastat) for Fabry disease in Japan. Available at: http://ir.amicusrx.com/news-releases/news-release-details/amicus-therapeutics-launches-galafoldr-migalastat-fabry-disease
, Accessed Oct. 10, 2018.
3. National Fabry Disease Foundation. Fabry Disease Treatment. Available at: https://www.fabrydisease.org/index.php/about-fabry-disease/fabry-disease-treatment
, Accessed Oct. 10, 2018.
4. Hou ZS, Ulloa-Aguirre A, Tao YX. Pharmacoperone drugs: Targeting misfolded proteins causing lysosomal storage-, ion channels-, and G protein-coupled receptors-associated conformational disorders. Expert Rev Clin Pharmacol
5. Germain DP, Hughes DA, Nicholls K, et al. Treatment of Fabry’s disease with the pharmacologic chaperone migalastat. N Engl J Med
6. Schiffmann R, Bichet DG, Jovanovic A, et al. Migalastat improves diarrhea in patients with Fabry disease: Clinical-biomarker correlations from the phase 3 FACETS trial. Orphanet J Rare Dis
7. Hughes DA, Nicholls K, Shankar SP, et al. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet