Galapagos’s Investigational Treatment for IPF Granted EU Orphan Drug Designation


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IPF therapy's EU orphan drug status

The European Commission (EC) has granted Galapagos’ investigational product GLPG1690 orphan drug designation for the treatment of patients with idiopathic pulmonary fibrosis (IPF).

GLPG1690 is a selective autotaxin inhibitor that was demonstrated to have a favorable safety and tolerability profile in a Phase 1 study in healthy volunteers. Autotaxin plays an important role in the production of the bioactive lipid lysophosphatific acid (LPA), which in turn controls several cellular activities. Researchers have suggested that autotaxin and LPA are involved in conditions like asthma, fibrosis, and lung disease.

“We are happy to see that the EC recognizes the potential of GLPG1690 as a new treatment for IPF patients in Europe. Next step will be the application for orphan drug designation with the Food and Drug Administration (FDA) in the U.S.,” Galapagos CSO Piet Wigerinck said in a recent press release.

GLPG1690 is currently being evaluated in the FLORA study, a randomized, double-blind, placebo-controlled Phase 2a clinical trial testing the drug’s tolerability, safety, pharmacodynamics, and pharmacokinetics in 24 IPF patients for 12 weeks. The drug is given orally once a day. Topline data from FLORA is expected by mid-year 2017.

Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union, which is the case in IPF.

To encourage the pharmaceutical industry to develop and market medicines for a small number of patients, the EU offers financial incentives such as 10 years of extended exclusivity, regulatory fee reductions, access to the centralized procedure for marketing authorization, and protocol assistance, all intended to stimulate the development of drugs which might otherwise lack a sufficient profit motive.

Galapagos recently gave a presentation titled “Strong reversal of the lung fibrosis disease signature by autotaxin inhibitor GLPG1690 in a mouse model for IPF” at the 2016 European Respiratory Society (ERS) Congress Sept. 3-7 in London.

IPF is a rare chronic disease that currently has no cure. It is characterized by a progressive decline in lung function, and the disease typically affects adults over the age of 40. The prevalence of IPF is fewer than 30 per 100,000 persons in both Europe and the United States.