CPF, PFF applaud PF patients' efforts to plead with FDA Advisory Panel regarding lack of treatment options

Mar 15 2010

PF Patients Plead with FDA Advisory Panel to Hear Their Voices, Understand Their Plight

The Coalition for Pulmonary Fibrosis (CPF) and the Pulmonary Fibrosis Foundation (PFF) are applauding the efforts of Pulmonary Fibrosis (PF) patients and family members for their work to convey to the FDA and an FDA Advisory Committee the sense of urgency and desperation regarding the disease and the lack of treatment options.  Their words were heard yesterday by the 11-member Advisory Committee and representatives of the FDA during a public hearing portion of the FDA Pulmonary-Allergy Drugs Advisory Committee discussion of a potential new therapy in the fight against PF.  The committee voted 9 to 3 to recommend approval for the drug by the FDA.

If approved, the therapy, Pirfenidone, would be the first FDA approved drug for PF.  PF is a progressive, relentless and irreversible scarring of the lungs which renders patients unable to breathe and is almost invariably fatal within two to five years.

"There's no question that the patient perspective was heard by the FDA and the panel," said Mishka Michon, Chief Executive Officer of the Coalition for Pulmonary Fibrosis.  "Family members and patients spoke from their hearts and conveyed the sense of urgency they feel in desperately waiting for a treatment for this horrific disease."

"We are humbled by the courage and determination of our patients," said Daniel Rose, M.D., President of the Pulmonary Fibrosis Foundation.  "The voice of the patient community is a critical component in finding solutions to PF."

The panel listened to testimony of 15 PF patients and family members for an hour. It also received a stack of letters from patients and families from across the country who could not attend the meeting in person.

Advocate Joy McBride opened the public hearing portion of the meeting by sharing with the panel the loss of her father and uncle to PF.  "The hardest part was hearing from the doctors 'there is nothing I can do for you,'" she said.  "Today, there is still no known cause, no cure, and no hope."

Patient Tommy Spivey traveled with his wife from Wilmington, North Carolina to speak to the panel.  "I have one grandchild and one on the way and I would like to live long enough to get to know them," he said.  Spivey, who says his disease is currently not progressing, traveled to Japan in 2009 to gain access to Pirfenidone and is currently taking the drug.

Kaitlyn Bergen shared with the panel details of her father's illness and subsequent death to PF in 2006.  "Pulmonary Fibrosis will continue stealing valuable and meaningful years from families," she said.

Though Timothy Cooney's father is one of a small number of survivors of the disease as a result of a successful lung transplant more than a year ago, he delivered his message to the panel on behalf of his family and in memory of his grandmother who was claimed by PF.  "On a sinking ship, if the life raft has a few holes in it, people will still take that raft," he said.

At the end of the day-long meeting, one panelist, Rodney Mullins, consumer representative for the committee, echoed Cooney's comments in his final remarks. "We don't know how leaky the lifeboat is, but it's a lifeboat."

The Advisory Committee's recommendations are not binding but will be considered as the FDA completes its review of the New Drug Application (NDA) for the drug.  Pirfenidone received Orphan Drug, Fast Track and Priority Review designations by the FDA. A target date of May 4, 2010 for FDA action has been set.