Galapagos NV (Euronext & NASDAQ: GLPG) announces today that the US Food & Drug Administration (FDA) and the European Commission (EC) have granted investigational autotaxin inhibitor GLPG1690 ‘orphan drug designation’ for the treatment of systemic sclerosis (SSc).
In order to stimulate the pharmaceutical industry to develop and market medicines for diseases affecting a small number of patients, the EC and the FDA offer a range of incentives to encourage the development of these ‘orphan’ medicines for rare diseases in the European Union and the United States. These incentives include amongst others 7 to 10 years of market exclusivity once the medicine is on the market, regulatory fee reductions and fee waivers and access to the centralized procedure for marketing authorization in Europe.1,2
“We are happy to see that the EC and FDA recognize GLPG1690 as a potential new treatment for SSc patients. With the NOVESA Phase 2 trial in SSc fully recruited, we expect to see topline data in the second half of the year,” said Dr. Walid-Abi-Saab, CMO of Galapagos.
Diffuse cutaneous systemic sclerosis (SSc) is an autoimmune disease involving multiorgan fibrosis, which has one of the highest mortality rates among rheumatic diseases.3 One of the most visible manifestions is hardening of the skin. In diffuse cutaneous SSc, skin thickening affects several body areas, and patients have a higher risk of developing fibrosis of various internal organs, such as the lung. Currently, there are no approved drugs for this disease. SSc affects approximately 90,000 patients in the US and Europe, with a predominance of female patients (75%).
GLPG1690 is an investigational small molecule, selective autotaxin inhibitor that was inlicensed by Gilead Sciences, Inc. as part of the global R&D collaboration between Galapagos & Gilead. Autotaxin is the main enzyme responsible for lysophosphatidic acid (LPA) production. LPA is a well-known pro-fibrotic and pro-inflammatory lipid, acting through at least 6 g-protein coupled receptors. Galapagos identified the autotaxin target using its proprietary target discovery platform and developed molecule GLPG1690 as an inhibitor of this target. GLPG1690 is currently being studied in a global Phase 3 program in idiopathic pulmonary fibrosis (ISABELA) as well as in the NOVESA Phase 2 trial.
GLPG1690 is an investigational drug and its efficacy and safety have not been established by any regulatory agency.
Galapagos (Euronext & NASDAQ: GLPG) discovers and develops small molecule medicines with novel modes of action, three of which show promising patient results and are currently in late-stage development in multiple diseases. Galapagos’ pipeline comprises discovery programs through Phase 3 programs in inflammation, fibrosis, osteoarthritis and other indications. The Company’s ambition is to become a leading global biopharmaceutical company focused on the discovery, development and commercialization of innovative medicines.