Orphan Drug Designation Granted to Nintedanib for Treatment of Systemic Sclerosis, including Associated Interstitial Lung Disease

Ridgefield, Conn., September 9, 2016 – Boehringer Ingelheim today announced that the European Commission (EC) and U.S. Food and Drug Administration (FDA) have granted Orphan Drug Designation to nintedanib for the treatment of systemic sclerosis (SSc, also known as scleroderma), including the associated interstitial lung disease (SSc-ILD).

SENSCIS™, the largest trial to date in this disease area, is evaluating nintedanib to understand the disease process and potential benefit of the compound to treat SSc-ILD.

Systemic sclerosis, commonly referred to as “scleroderma,” is a disfiguring, disabling and potentially fatal rare disease that can cause scarring of the skin, lungs (SSc-ILD) and other organs. Worldwide, an estimated two million people have systemic sclerosis (also known as scleroderma) and up to 90% may develop some degree of lung scarring. In the U.S., it is estimated that SSc-ILD affects up to 86,000 people. SSc-ILD indicates a poor prognosis and accounts for 35 percent of all disease-related deaths.

"To have nintedanib receive Orphan Drug Designation is an exciting step forward for people living with scleroderma and associated interstitial lung disease, as well as their families," said Robert Riggs, CEO, Scleroderma Foundation.

"This designation represents important progress towards addressing an unmet need and bringing a potential new treatment to those with this rare and devastating disease," said Luke Evnin, Ph.D., chairman, Scleroderma Research Foundation.

Orphan Drug Designation is generally granted by the EC for a therapeutic agent intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000, and for which there is no or only unsatisfactory treatment options or the medicine will be of significant benefit to those affected by that condition. In the U.S., the FDA grants the status to investigational compounds intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people.

“The FDA’s and European Commission’s Orphan Drug Designations for nintedanib are encouraging milestones in our SSc-ILD development program — and underscore our commitment to patients living with rare fibrotic lung diseases,” said Martina Flammer, M.D., vice president, Clinical Development & Medical Affairs Specialty Care, Boehringer Ingelheim Pharmaceuticals, Inc. “Building on our positive real-world clinical experience in idiopathic pulmonary fibrosis (IPF), exploring nintedanib’s potential as part of the SENSCIS™ study may revolutionize the way we approach this debilitating disease with unsatisfactory treatment options.”

About SENSCIS™ and nintedanib
The SENSCIS™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS) study has the potential to provide advances in the treatment of this devastating condition and to help address the unmet need for the many people living with systemic sclerosis or SSc-ILD. Nintedanib is currently being evaluated in a randomized, double-blind, placebo-controlled study designed to measure the efficacy and safety of nintedanib 150 mg twice daily over 52 weeks up to a maximum of 100 weeks in people with SSc-ILD. The primary endpoint is the annual rate of decline in forced vital capacity (FVC), a measure of disease progression. Key secondary endpoints include the absolute change from baseline in the modified Rodnan Skin Score (mRSS), which is an evaluation of people’s skin thickness, and the absolute change from baseline in the Saint George´s Respiratory Questionnaire (SGRQ) total score, which measures the health-related quality of life of people with lung diseases to assess the impact of treatment.

Additional information is also available at:
clinicaltrials.gov/ct2/show/NCT02597933.

The U.S. Food and Drug Administration (FDA) approved nintedanib, marketed as Ofev®, for the treatment of idiopathic pulmonary fibrosis (IPF) on October 15, 2014. Ofev is one of the first FDA-approved drug treatments for IPF and the only kinase inhibitor approved to treat this disease.

About Systemic Sclerosis and Interstitial Lung Disease
Systemic sclerosis (SSc, also known as scleroderma) is a rare and serious disease characterized by thickening and scarring of connective tissue of multiple organs in the body. It is difficult to diagnose because a lot of organ systems are involved and its symptoms are similar to other autoimmune diseases. The disease impacts four times as many women as men, and the onset of the disease typically occurs at a young age – between 35 and 55 years.

Nearly all people with systemic sclerosis have hardening and thickening of patches of skin. The disease also can cause scarring in the lungs, heart, and kidneys, which can be debilitating and may become life-threatening.

When the disease affects the lungs, ultimately resulting in scarring, it is called SSc-ILD. Shortness of breath and difficulty in performing daily activities are common symptoms of SSc-ILD. Lung involvement, like ILD, is the leading cause of death among people with systemic sclerosis.

What is OFEV (nintedanib)?
OFEV is a prescription medicine used to treat people with a lung disease called idiopathic pulmonary fibrosis (IPF). It is not known if OFEV is safe and effective in children.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about OFEV (nintedanib)?

OFEV can cause harm, birth defects or death to an unborn baby. Women should not become pregnant while taking OFEV. Women who are able to become pregnant should have a pregnancy test before starting treatment and should use birth control during and for at least 3 months after your last dose. If you become pregnant while taking OFEV, tell your doctor right away.

What should I tell my doctor before using OFEV? Before you take OFEV, tell your doctor if you have:

• liver problems
• heart problems
• a history of blood clots
• a bleeding problem or a family history of a bleeding problem
• had recent surgery in your stomach (abdominal) area
• any other medical conditions.

Tell your doctor if you:

• are pregnant or plan to become pregnant.
• are breastfeeding or plan to breastfeed. It is not known if OFEV passes into your breast milk. You should not breastfeed while taking OFEV.
• are a smoker. You should stop smoking prior to taking OFEV and avoid smoking during treatment.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, herbal supplements such as St. John’s wort.

What are the possible side effects of OFEV?

OFEV may cause serious side effects.

TELL YOUR DOCTOR RIGHT AWAY if you are experiencing any side effects, including:

• Liver problems. Unexplained symptoms may include yellowing of your skin or the white part of your eyes (jaundice), dark or brown (tea colored) urine, pain on the upper right side of your stomach area (abdomen), bleeding or bruising more easily than normal or feeling tired. Your doctor will do blood tests regularly to check how well your liver is working during your treatment with OFEV.
• Diarrhea, nausea, and vomiting. Your doctor may recommend that you drink fluids or take medicine to treat these side effects. Tell your doctor if you have these symptoms, if they do not go away, or get worse and if you are taking over-the-counter laxatives, stool softeners, and other medicines or dietary supplements.
• Heart attack. Symptoms of a heart problem may include chest pain or pressure, pain in your arms, back, neck or jaw, or shortness of breath.
• Stroke. Symptoms of a stroke may include numbness or weakness on 1 side of your body, trouble talking, headache, or dizziness.
• Bleeding problems. OFEV may increase your chances of having bleeding problems. Tell your doctor if you have unusual bleeding, bruising, or wounds that do not heal and/or if you are taking a blood thinner, including prescription blood thinners and over-the-counter aspirin.
• Tear in your stomach or intestinal wall (perforation). OFEV may increase your chances of having a tear in your stomach or intestinal wall. Tell your doctor if you have pain or swelling in your stomach area.

The most common side effects of OFEV are diarrhea, nausea, stomach pain, vomiting, liver problems, decreased appetite, headache, weight loss, and high blood pressure.

These are not all the possible side effects of OFEV. For more information, ask your doctor or pharmacist. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Click here for full Prescribing Information, including Patient Information.

About Boehringer Ingelheim
Boehringer Ingelheim Pharmaceuticals, Inc., based in Ridgefield, CT, is the largest U.S. subsidiary of Boehringer Ingelheim Corporation.

Boehringer Ingelheim is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, the company operates globally with 145 affiliates and more than 47,000 employees. Since its founding in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel treatments for human and veterinary medicine.

Boehringer Ingelheim is committed to improving lives and providing valuable services and support to patients and their families. Our employees create and engage in programs that strengthen our communities. To learn more about how we make more health for more people, visit our Corporate Social Responsibility Report.

In 2015, Boehringer Ingelheim achieved net sales of about $15.8 billion (14.8 billion euros). R&D expenditure corresponds to 20.3 percent of its net sales.

For more information, please visit www.us.boehringer-ingelheim.com, or follow us on Twitter @BoehringerUS.

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