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FDA Approves Two Drugs For Idiopathic Pulmonary Fibrosis

OCTOBER 15, 2014
James Radke

Today, the U.S. Food and Drug Administration (FDA) approved two orphan drugs for the treatment of idiopathic pulmonary fibrosis – Intermune’s Esbriet (pirfenidone) and Boehringer Idelheim’s Ofev (nintedanib).

Idiopathic Pulmonary Fibrosis

Idiopathic Pulmonary Fibrosis is a chronic, progressive fibrosing interstitial pneumonia that does not have a clear cause. In most cases, the disease is fatal within 2-3 years of initial symptoms and treatment options are limited. Current treatments (until now) included oxygen therapy, pulmonary rehabilitation, and lung transplant.


Esbriet (pirfenidone)

Esbriet acts on multiple pathways that may be involved in the scarring of lung tissue. In a statement, the FDA noted that the drugs safety and effectiveness were established in three clinical trials of 1,247 patients with idiopathic pulmonary fibrosis. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Esbriet compared to patients receiving placebo.

Curtis J. Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research said:

“Esbriet provides a new treatment option for patients with idiopathic pulmonary fibrosis, a serious, chronic lung disease.”

The FDA granted Esbriet fast track, priority review, orphan product, and breakthrough designations. Esbriet is being approved ahead of the product’s prescription drug user fee goal date of Nov. 23, 2014.

Esbriet is not recommended for patients who have severe liver problems, end-stage kidney disease, or who require dialysis. Esbriet should be taken with food to minimize the potential for nausea and dizziness. Patients should avoid or minimize exposure to sunlight and sunlamps and wear sunscreen and protective clothing, as Esbriet may cause patients to sunburn more easily.

The most common side effects of Esbriet are nausea, rash, abdominal pain, upper respiratory tract infection, diarrhea, fatigue, headache, dyspepsia, dizziness, vomiting, decreased/loss of appetite, gastro-esophageal reflux disease, sinusitis, insomnia, decreased weight, and arthralgia.


Ofev (nintedanib)

Ofev is a tyrosine kinase inhibitor that blocks multiple pathways that may be involved in the scarring of lung tissue. In a press release, the FDA stated Ofev’s safety and effectiveness were established in three clinical trials of 1,231 patients with IPF. The decline in forced vital capacity – the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible – was significantly reduced in patients receiving Ofev compared to patients receiving placebo.

Mary H. Parks, M.D., deputy director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research said:

“Today’s Ofev approval expands the available treatment options for patients with idiopathic pulmonary fibrosis, a serious, chronic condition.”

The FDA granted Ofev fast track, priority review, orphan product, and breakthrough designations. Ofev is being approved ahead of the product’s PDUFA date of Jan. 2, 2015.

Ofev is not recommended for patients who have moderate to severe liver problems. Ofev can cause birth defects or death to an unborn baby. Women should not become pregnant while taking Ofev. Women who are able to get pregnant should use adequate contraception during and for at least three months after the last dose of Ofev.

The most common side effects of Ofev are diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, headache, decreased weight, and high blood pressure.


Advocacy Response

In a press release, the Coalition for Pulmonary Fibrosis (CPF) applauded the two FDA approvals.  Mishka Michon, CEO of the CPF said:

"It is a historic for IPF patients with two first-ever treatments for IPF being approved by the FDA." 

"With these therapies comes hope for a brighter future for patients and renewed optimism regarding the battle against IPF amongst physicians and researchers."


Drug Availability

The makers of Esbriet have stated it will be available in 2 weeks and the makers of Ofev have stated it will be available in 10 days.

Coalition for Pulmonary Fibrosis (CPF) applauds the two U.S. Food and Drug Administration (FDA) approvals - See more at: http://www.globenewswire.com/news-release/2014/10/15/673554/10102869/en/CPF-Applauds-Two-FDA-Approvals-Announced-Today-for-First-Drugs-for-Idiopathic-Pulmonary-Fibrosis.html#sthash.aVbDyzEV.dpuf

 



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