Food and Drug Administration holds meeting on Alpha-1 Antitrypsin Deficiency
On September 29, 2015, Alphas and Alpha-1 caregivers packed a public meeting held by the US Food and Drug Administration to discuss Patient-Focused Drug Development for Alpha-1 Antitrypsin Deficiency. About 250 people filled the room, while about 600 attended online.
Alpha-1 Foundation Board member Liz Johnson explained the impact of Alpha-1 on daily life based on results of a survey of just under 1,700 response: 1. Nearly 100% of those with Alpha-1 affecting their lungs reported shortness of breath. 2. 37% of these individuals reported that shortness of breath had a significant or extremely significant effect on their daily lives, including dressing, washing, bending down, or tying shoes. 3. 72% of the total noted breathlessness with exercise. 4. Among those affected with liver involvement, 74% reported abdominal pain as a symptom, and 73% had abdominal swelling.
AlphaNet coordinator Fred Walsh commented that: “Pulmonary rehab is essential and should be widely available and inexpensive. A rescue inhaler can help everybody. I can’t believe there is no generic version of albuterol.”
Most speakers said that they were satisfied with augmentation therapy, but nearly all expressed the need for new therapies, especially for Alpha-1 liver disease.
Pujita Vaidya, MPH, gave an overview of the Patient-Focused Drug Development Initiative and said that the FDA will summarize patient testimony with a summary of “What We Learned,” from the Alpha-1 hearing.