FDA approves Ofev® as the first and only therapy in the U.S. to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated ILD

Ridgefield, Conn., September 9, 2019 – Boehringer Ingelheim announced that the U.S. Food and Drug Administration (FDA) approved Ofev® (nintedanib) as the first and only medicine to slow the rate of decline in pulmonary function in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD). Ofev is already approved in the U.S. and more than 70 countries for the treatment of patients living with idiopathic pulmonary fibrosis (IPF), and has been shown to slow IPF progression by reducing the annual rate of decline in lung function, as measured by forced vital capacity (FVC). 

“This is the first FDA-approved therapy to slow the rate of decline in pulmonary function for systemic sclerosis associated interstitial lung disease and provides hope to patients and their loved ones facing this devastating disease,” said Thomas Seck, M.D., senior vice president, Medicine & Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “This approval is supported by positive evidence from the Phase III study that showed Ofev significantly slowed the progression of lung function decline in this patient population and exemplifies Boehringer Ingelheim’s dedication to the rare disease community.”

Largest SSc-ILD study to date
The approval was based on results of SENSCIS®, a Phase III double-blind randomized, placebo-controlled trial, that involved 576 patients from 194 trial sites across 32 countries. The primary endpoint was the annual rate of decline in FVC in patients with SSc-ILD. Results shows that Ofev slowed the loss of pulmonary function by 44% (41 mL/year) in patients with SSc-ILD relative to placebo, as measured by FVC over 52 weeks.⁴

The safety and tolerability of Ofev were evaluated in SSc-ILD patients. The most common adverse reactions (occurring in greater than or equal to 5%) in Ofev-treated patients compared to placebo included: diarrhea, nausea, vomiting, skin ulcer, abdominal pain, liver enzyme elevation, weight decreased, fatigue, decreased appetite, headache, fever, back pain, dizziness and hypertension. 

“An approved anti-fibrotic medication for this condition is a major scientific advancement in the care of patients living with this rare disease,” said Kristin Highland, M.D., pulmonlogist with the Cleveland Clinic. “The option to offer a new therapy is welcome news for doctors and their patients.”

About the disease 
Systemic sclerosis, also known as scleroderma, is a rare autoimmune disease characterized by thickening and scarring of connective tissue throughout the body. The disease is estimated to affect about 100,000 people in the U.S.¹ and 2.5 million worldwide.⁵ Fibrosis, the hallmark of the disease, can affect the skin and internal organs, including the lungs. Interstitial lung disease (ILD), one of the most frequent disease manifestations, can be debilitating and may become life-threatening. Approximately 25 percent of patients develop significant lung involvement within three years of diagnosis.² ILD is the leading cause of death among people with SSc.³

Advocacy community perspective
Robert Riggs, chief executive officer of the Scleroderma Foundation, who has observed stronger interest in scleroderma research in the past few years said: “This approval brings hope to a community in need of new options to treat scleroderma-associated ILD.” 

Luke Evnin, Ph.D., a scleroderma patient and chairman of the Scleroderma Research Foundation commented: “When interstitial lung disease occurs in systemic sclerosis, the consequences can be severe. The availability of a new therapy for patients with this rare, chronic condition is truly exciting news for our community and at the core of the mission of the Scleroderma Research Foundation to improve the lives of patients.”

References

1. American College of Rheumatology (ACR). www.rheumatology.org/I-Am-A/Patient-Caregiver/Dis 
2. McNearney TA, Reveille JD, Fischbach M, et al. Pulmonary involvement in systemic sclerosis: associations with genetic, serologic, sociodemographic, and behavioral factors. Arthritis Rheum. 2007;57(2):318–326
3. Tyndall AJ et al. Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) database. Ann Rheum Dis. 2010 Oct;69(10):1809-15. doi: 10.1136/ard.2009.114264.
4. Distler O, et al. Nintedanib for Systemic Sclerosis-Associated Interstitial Lung Disease. NEJM.
5. Scleroderma & Raynauds UK (SRUK) https://www.sruk.co.uk/scleroderma/what-scleroderma/ Last accessed March 2019.