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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Stem Cell Transplantation Offers Hope For Severe Scleroderma

MedicalResearch.com Interview with:

“Breastfeeding welcome here” by Newtown grafitti is licensed under CC BY 2.0

Picture of a female patient’s left arm, showing skin lesions caused by Scleroderma
Wikipedia image

Keith M. Sullivan, M.D.
James B. Wyngaarden Professor Of Medicine
Division of Cellular Therapy
Duke University Medical Center
Durham, North Carolina 27710, USA 

MedicalResearch.com: What is the background for this study? What are the main findings?

  • Scleroderma with internal organ involvement is a devastating  autoimmune disorder with considerable morbidity and high mortality which have not changed in 40 years of reporting. Effective new therapies are needed.
  • Despite 2 prior randomized trials showing benefit for reduced-intensity stem cell transplant vs. conventional cyclophosphamide immune suppression, clinical practice in the US did not change due in part due to concern about patient safety and durability of response (attached).
  • The current randomized trial compares 12 monthly infusions of cyclophosphamide with high-dose chemotherapy plus whole-body irradiation designed to wipe-out (myeloablate) the defective, self-reactive immune system and replace with the patients own stem cells which had been treated to remove self-reacting lymphocytes. This was the first study to test if myeloablative autologous could re-establish a normal functioning immune system in patients with scleroderma.

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Systemic Sclerosis: Autologous Hematopoietic Stem Cell Transplantation vs IV Pulse Cyclophosphamide

MedicalResearch.com Interview with:
ProfProf. Dr. Jacob M. van Laar Professor and Chair Dept of Rheumatology & Clinical Immunology University Medical Center Utrecht . Dr. Jacob M. van Laar
Professor and Chair
Dept of Rheumatology & Clinical Immunology
University Medical Center Utrecht

MedicalResearch: What are the main findings of the study?

Prof. van Laar: The results of the ASTIS-trial demonstrate that stem cell transplantation in selected patients with early, diffuse cutaneous systemic sclerosis, a rare, autoimmune connective tissue disease, prolongs long-term survival and improves clinical manifestations (skin, lung) and quality of life, when compared to monthly infusions with cyclophosphamide. The benefits must be weighed against the risks which include early  treatment-related mortality (10% in the ASTIS-trial) and viral infections.

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