FDA Grants Fast Track Designation to Nintedanib for Scleroderma with Lung Disease

MedicalResearch.com Interview with:

Dr. Thomas Leonard, Ph.D. Executive director, Clinical Development and Medical Affairs, Specialty Care Boehringer Ingelheim Pharmaceuticals, Inc.

Dr. Thomas Leonard

Dr. Thomas Leonard, Ph.D.
Executive director, Clinical Development and Medical Affairs, Specialty Care
Boehringer Ingelheim

MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by systemic sclerosis? What are the disease symptoms and manifestations?

Response: The FDA recently granted Fast Track designation to nintedanib for the treatment of systemic sclerosis with interstitial lung disease (SSc-ILD) – paving the way for Boehringer Ingelheim to take an important step in advancing this potential therapy for those affected by this disease. The designation was based on Boehringer Ingelheim’s Investigational New Drug application (IND) and the anticipated efficacy and safety data from SENSCIS™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS), a double-blind, randomized, placebo-controlled global Phase III trial which is fully enrolled and includes more than 520 patients from 32 countries.

The FDA’s Fast Track designation facilitates the development of new therapies that treat serious conditions and fulfill an unmet medical need in an effort to get treatments to those in need sooner, like those living with systemic sclerosis.

Systemic sclerosis, also known as scleroderma, is a rare disease characterized by thickening and scarring of connective tissue of multiple organs in the body, typically affecting women between ages 25 and 55. Most people with the disease will develop some degree of lung scarring, or interstitial lung disease (ILD), which is the leading cause of death among people with systemic sclerosis.

Nintedanib, currently marketed as Ofev®, is approved for treatment of a rare lung disease called idiopathic pulmonary fibrosis, or IPF, and has been shown to slow disease progression as measured by annual rate of decline in lung function. Because SSc-ILD and IPF share similarities in how the underlying lung scarring, or fibrosis, forms in people with the disease, Boehringer Ingelheim is evaluating the impact of nintedanib on SSc-ILD.

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Systemic Sclerosis: Chemokine CXCL4 as a Biomarker of Progression and Phenotype

Prof. Dr. T.R.D.J. Radstake, MD, PhD Staff Rheumatologist / head translational Immunology Department of Rheumatology & Clinical Immunology EULAR Center of Excellence Director, UMC Utrecht Infection and Immunity FOCIS Center of Excellence University Medical Center Utrecht, The NetherlandsMedicalResearch.com Interview with:
Prof. Dr. T.R.D.J. Radstake, MD, PhD
Staff Rheumatologist / head translational Immunology
Department of Rheumatology & Clinical Immunology EULAR Center of Excellence
Director, UMC Utrecht Infection and Immunity FOCIS Center of Excellence
University Medical Center Utrecht, The Netherlands

MedicalResearch.com: What are the main findings of the study?

Prof. Radstake: We observed that the chemokine CXCL4 is highly produced by so-called plasmacytoid dendritic cells in systemic sclerosis (Ssc). CXCL4 is associated with the progression and clinical phenotype of Ssc and thus provides a tool for clinicians to identify those patients in need for aggressive therapy and on the other hand, avoid unnecessary side-effects for those who have mild disease. Moreover, the identified roles for CXCL4 in SSc sparks our knowledge on the pathogenic pathways at hand in this terrible conditions. Now, we and other groups will have to further unravel the precise roles for CXCL4 in SSc and possibly other fibrotic and immune mediated conditions that cover the spectrum of medicine.
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