OPEN LETTER IN SUPPORT OF MOLGRAMOSTIM FOR AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS (aPAP) - PATIENTS
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Division of Pulmonary, Allergy, and Critical Care
Office of Immunology and Inflammation
Office of New Drugs
Center for Drug Evaluation and Research
U.S. Food and Drug Administration
10903 New Hampshire Avenue, Silver Spring, MD 20993
Dear FDA:
We are
the PAP – Pulmonary Alveolar Proteinosis Patients & Caregivers Facebook page
comprised of 368 members. We are writing on behalf of people living with autoimmune pulmonary alveolar proteinosis (aPAP), an ultra-rare and chronic lung disease, and the families who care for them. We are grateful for the FDA’s consideration of molgramostim inhalation solution (molgramostim) for the treatment of people with aPAP. We are writing to express our support for FDA approval for Molgramostim, and share how it could greatly improve our lives, and possibly save a life.
We are the people whose quality of life has been severely degraded by aPAP. We who can no longer climb a flight of stairs or walk the dog without stopping to catch our breath. We who have had to give up playing sports, hiking, enjoying the outdoors, or participating in our family and children’s activities. We who struggle with the simplest of daily activities. We who suffer with near constant dyspnea, fatigue, and cough. We who may need to be on continuous supplemental oxygen. We who have lost our jobs and faced financial hardship. We who have taken the risks with undergoing Whole Lung Lavage (WLL) to remove the excess surfactant from our lungs. Some of us have had to endure many and frequent WLLs because an FDA approved drug is not available to treat aPAP. We who may have to travel great distances and at great expense to find a medical institution experienced and knowledgeable to safely perform WLL. We who have endured anxiety, depression, and entertained thoughts of suicide.
We are the caretakers, friends, and family who have watched our loved ones decline. We who share their pain, fear and frustration.
The PAP Foundation is the largest patient advocacy organization for aPAP in the United States. On May 7, 2025, the PAP Foundation with the National Organization for Rare Disorders (NORD), hosted the externally led Patient-Focused Drug Development (EL-PFDD) meeting on aPAP—the largest ever gathering of people affected by aPAP. Eight patients spoke on panels and 41 patients and caregivers answered polling questions, together reflecting the experience of 49 people living with aPAP. What follows is drawn from what was shared that day.
The completed molgramostim trials enrolled more aPAP patients than any other trial for our disease. So, there are patients that have already taken molgramostim in the IMPALA and IMPALA-2 trials or through the expanded access program. There are patients now after treatment that know what it feels like to breathe easier. Those of us on it want to stay on it. One IMPALA-2 participant told the EL-PFDD meeting, “I’ve been able to spend a lot more time doing outdoor activities, camping, riding my bike, hiking and doing the things that I did before I got diagnosed with this disease…I feel like I’m finally gaining some of that life back.”
Five patients treated with molgramostim outside the trials have now been described in the medical literature: one who had endured eight lavages in four years has not needed a single lavage since starting molgramostim; another came off oxygen and returned to work. Not one of the five has needed a lavage since starting the medicine. These are not numbers on a chart. They are stairs climbed, days worked, and lives given back.
When our community was asked what would matter most in a new treatment; 97% said improving symptoms or slowing their progression, 94% said improved quality of life, and 88% said ease of access and use. In clinical trials, molgramostim did what our community asked for; it improved symptoms and quality of life, and it’s a once-daily inhaled medicine that patients can take at home. Even a chance at symptom improvement or slowing disease progression over the long run would be meaningful progress.
The aPAP community have been waiting patiently for decades to have an FDA approved drug proven to be successful in the treatment of aPAP. We are grateful for the attention the FDA has already given to this ultra-rare (fewer than 10 per million) disease most people have never heard of. We sincerely ask you to approve Molgramostim and give us a chance to breathe freely again and reclaim our quality of life.
Respectfully,
The PAP Patient and Caregivers Facebook Group
Signed by the patients, caregivers, and family members who have added their names to this letter.
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