FDA approves first treatment for patients with rare type of lung disease

FDA The U.S. Food and Drug Administration has approved Ofev (nintedanib) capsules to slow the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis or scleroderma, called SSc-ILD. It is the first FDA-approved treatment for this rare lung condition.

"Patients suffering from scleroderma need effective therapies, and the FDA supports the efforts of drug companies that are designing and conducting the clinical trials necessary to bring treatment options to scleroderma patients," said Nikolay Nikolov, M.D., associate director for Rheumatology of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research. "Nintedanib is now a treatment option to slow the rate of decline in pulmonary function in patients who have interstitial lung disease from scleroderma."

Scleroderma is a rare disease that causes tissue throughout the body, including the lungs and other organs, to thicken and scar. Interstitial lung disease or ILD is a condition affecting the interstitium, which is part of the lung’s structure, and is one of the most common disease manifestations of sclerderma. SSc-ILD is a progressive lung disease in which lung function declines over time, and it can be debilitating and life-threatening. ILD is the leading cause of death among people with scleroderma, typically resulting from a loss of pulmonary function that occurs when the lungs cannot supply enough oxygen to the heart. Approximately 100,000 individuals in the United States have scleroderma, and approximately half of scleroderma patients have SSc-ILD.

The effectiveness of Ofev to treat SSc-ILD was studied in a randomized, double-blind, placebo-controlled trial of 576 patients ages 20-79 with the disease. Patients received treatment for 52 weeks, with some patients treated up to 100 weeks. The primary test for efficacy measured the forced vital capacity, or FVC, which is a measure of lung function, defined as the amount of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. Those who took Ofev had less lung function decline compared to those on placebo.

The overall safety profile observed in the Ofev treatment group was consistent with the known safety profile of the therapy. The most frequent serious adverse event reported in patients treated with Ofev was pneumonia (2.8% Ofev vs. 0.3% placebo). Adverse reactions leading to permanent dose reductions were reported in 34% of Ofev-treated patients compared to 4% of placebo-treated patients. Diarrhea was the most frequent adverse reaction that led to permanent dose reduction in patients treated with Ofev.

The prescribing information for Ofev includes warnings for patients with moderate or severe hepatic (liver) impairment, those with elevated liver enzymes and drug-induced liver injury and patients with gastrointestinal disorders. Ofev may also cause embryo-fetal toxicity that can result in fetal harm, arterial thromboembolic events (blood clots), bleeding and gastrointestinal perforation. P-gp and CYP3A4 inhibitors may increase nintedanib exposure, and patients taking these inhibitors should be closely monitored for tolerability of Ofev. Common side effects noted with Ofev include diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, weight loss and hypertension (high blood pressure).

Ofev was originally approved in 2014 for adult patients with idiopathic pulmonary fibrosis (IPF), which is another interstitial lung condition.

Ofev received Priority Review designation, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition. Ofev also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Ofev to treat SSc-ILD to Boehringer Ingelheim Pharmaceuticals Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Study finds lack of racial diversity in cancer dru…

New research published in JAMA Oncology has found a lack of racial and ethnic diversity in clinical trials for cancer drugs. The study - conducted by researchers from UBC...

Preventing tumor metastasis

Researchers at the Paul Scherrer Institute, together with colleagues from the pharmaceutical company F. Hoffmann-La Roche AG, have taken an important step towards the dev...

A new drug could revolutionize the treatment of ne…

The international team of scientists from Gero Discovery LLC, the Institute of Biomedical Research of Salamanca, and Nanosyn, Inc. has found a potential drug that may pre...

Interactions discovered in cells insulating nerve …

Schwann cells form a protective sheath around nerve fibres and ensure that nerve impulses are transmitted rapidly. If these cells are missing or damaged, severe neurologi...

Anniversary of the pivotal RE-LY® trial marks a de…

Boehringer Ingelheim today announces the ten-year anniversary of the RE-LY® trial publication(1-3) recognising the contribution made in the decade since by patients, heal...

AstraZeneca agrees to buy US FDA Priority Review V…

AstraZeneca announced that it has agreed to buy a US Food and Drug Administration (FDA) Priority Review Voucher (PRV) for a total cash consideration of $95m from a subsid...

The Pfizer Foundation invests in 20 organizations …

The Pfizer Foundation announced 20 grants* to help non-governmental organizations (NGOs), non-profits and social enterprises address critical health challenges related to...

FDA grants Fast Track designation for Farxiga in c…

AstraZeneca today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for the development of Farxiga (dapagliflozin) to delay the ...

Pfizer invests half billion dollars to advance sta…

Pfizer announced an additional half billion dollar investment for the construction of its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina...

Breast cancer can form 'sleeper cells' after drug …

Breast cancer medicines may force some cancer cells into 'sleeper mode', allowing them to potentially come back to life years after initial treatment. These are the early...

Amgen and Allergan announce positive top-line resu…

Amgen (NASDAQ:AMGN) and Allergan plc. (NYSE:AGN) today announced positive top-line results from a comparative clinical study evaluating the efficacy and safety of ABP 798...

Amgen to acquire Otezla® for $13.4 billion in cash

Amgen (NASDAQ: AMGN) announced today that it has entered into an agreement with Celgene Corporation (NASDAQ: CELG) in connection with its previously announced merger with...