A Day with the FDA …
On January 12th I presented in front of the FDA Advisory Committee relating to the pending approval of Liprotamase from Alnara Pharmaceuticals (a wholly owned subsidiary of Eli Lilly) on behalf of CF parents/caregivers. It was a valuable learning experience for sure, but one with an unfortunate outcome.
Liprotamase is a pancreatic enzyme capsule that would largely benefit Cystic Fibrosis patients (30,000 people in U.S.) of which 90% suffer from pancreatic insufficiency. CF patients are required to swallow enzymes (presently only derived from ground up pig pancreases coated in plastic) with every meal and snack so that they can absorb fat and nutrients. It’s an exhausting, unpredictable and unfortunately necessary regiment.
My argument for FDA approval of Liprotamase boiled down to three points.
The next step is for the Alnara/Lilly/CFF team to meet with the FDA to see what exactly that panel needs to feel more comfortable about approving Liprotamase and also to continue educating them about those living with CF.
And while the meeting’s outcome wasn’t what I expected, I left with a much better understanding of the incredible effort that goes into the clinical process of developing a pharmaceutical and getting FDA approval.
I have an intensified admiration for the MD’s, Clinical Operations, Regulatory Affairs, and everyone else whose lives involve clinical trials and FDA approval. The Alnara clinical team that I saw in action on January 12th was the most organized group of warriors I could imagine.
One has to wonder about the decision-making process of a 12-member FDA advisory panel that has no CF-specific expertise. Are they really hearing and understanding the concerns of the thousands of parents and patients pleading for this product?
Clinical trials proved Liprotomase to be a very safe and effective product that could be of huge benefit to CF patients.
I just hope FDA scrutiny like this doesn’t unnecessarily burden the type of cutting-edge biotech and pharmaceutical development leaders that we at StratAcuity help move forward by making sure they are staffed with the most talented scientists.
I’m keeping my fingers crossed that the FDA will make the right decision by approving this groundbreaking capsule sooner than later.
Liprotamase is a pancreatic enzyme capsule that would largely benefit Cystic Fibrosis patients (30,000 people in U.S.) of which 90% suffer from pancreatic insufficiency. CF patients are required to swallow enzymes (presently only derived from ground up pig pancreases coated in plastic) with every meal and snack so that they can absorb fat and nutrients. It’s an exhausting, unpredictable and unfortunately necessary regiment.
My argument for FDA approval of Liprotamase boiled down to three points.
- Babies with CF could greatly benefit from an enzyme that could easily dissolve in liquid to ease the current parental/caregiver burden.
- The new drug could be a lifesaver in the event of a devastating viral outbreak in the world’s pig population. In short: No pigs means no enzymes for CF patients who would potentially starve to death.
- The new enzyme capsule could greatly improve CF patient’s quality of life. Today, my 11 year old daughter Kate takes about 20 porcine-based enzymes each day, while the approval of Liprotamase would result in her only having to take 1-2 pills per meal!
The next step is for the Alnara/Lilly/CFF team to meet with the FDA to see what exactly that panel needs to feel more comfortable about approving Liprotamase and also to continue educating them about those living with CF.
And while the meeting’s outcome wasn’t what I expected, I left with a much better understanding of the incredible effort that goes into the clinical process of developing a pharmaceutical and getting FDA approval.
I have an intensified admiration for the MD’s, Clinical Operations, Regulatory Affairs, and everyone else whose lives involve clinical trials and FDA approval. The Alnara clinical team that I saw in action on January 12th was the most organized group of warriors I could imagine.
One has to wonder about the decision-making process of a 12-member FDA advisory panel that has no CF-specific expertise. Are they really hearing and understanding the concerns of the thousands of parents and patients pleading for this product?
Clinical trials proved Liprotomase to be a very safe and effective product that could be of huge benefit to CF patients.
I just hope FDA scrutiny like this doesn’t unnecessarily burden the type of cutting-edge biotech and pharmaceutical development leaders that we at StratAcuity help move forward by making sure they are staffed with the most talented scientists.
I’m keeping my fingers crossed that the FDA will make the right decision by approving this groundbreaking capsule sooner than later.
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