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7 Success Attributes for a Super-Orphaned Drug Manufacturing Suite

Takeda manufactures one batch of infant botulism treatment every five years. Source plasma only comes from around 30 donors. Hear about their “batch right, first time” approach, because there is no second chance to get it right.

Takeda's Pat Gavit presented at the PDA Annual Meeting in Long Beach, CA.
Takeda's Pat Gavit presented at the PDA Annual Meeting in Long Beach, CA.

Biopharmaceutical manufacturing will always have challenges, even for products manufactured day in and day out in 24/7 operations. But ultra-rare drug manufacturing comes with its own unique hurdles. If a drug is only produced once every few years, how does a company retain knowledge among staff?

At the PDA Annual Meeting this week, Patrick Gavit, head of manufacturing science at Takeda, offered insights into how their Los Angeles manufacturing facility produces a super-orphaned drug once every five years for infants suffering from infant botulism, which can lead to muscle weakness, trouble breathing, and more. 

Takeda produces the drug on a contract basis for the California Department of Public Health (CDPH), who owns the license for this public service, not-for-profit orphan drug. It is filled by Emergent BioSolutions in Baltimore.

As Gavit explained, the drug is colloquially called BabyBIG because it's used to treat infants under one year of age who have infant botulism, and the BIG is an acronym for Botulism Immune Globulin. The drug is purified from human plasma, obtained from volunteer donors who have been inoculated with the botulinum toxin so that they produce antibodies targeted against the toxin.

BabyBIG is the only approved treatment for infant botulism, and there are on average 150 infant patients per year in the U.S. in need of the drug. Because Takeda manufactures one batch every five years, and source plasma comes from only 30 donors, Gavit upholds a “batch right, first time” approach because there is no second chance to get it right.

Gavit offered tips for success in producing ultra-rare drugs, given the infrequent manufacturing schedule.

1. Modern manufacturing suite

The manufacturing suite where BabyBIG is produced is shared with another product which is typically produced 24/7/365. To produce BabyBIG, they need a six-month window that entails moving out the equipment from the other product, moving in the equipment for BabyBIG, doing validation activities, training the operators, doing practice runs, and doing the actual manufacturing run. “There's a lot that is contained in that six-month window. What really enables us to do this well is a very flexible manufacturing suite. All of the equipment—with the exception of a few centrifuges—is mobile, on wheels,” said Gavit.

They wheel out tanks for the other product and wheel in equipment for BabyBIG. “There are three viral zones in the manufacturing suite, and both products need all three of those zones. We also have the ability to adjust the temperature in the rooms from either ambient to 5°C—some of the BabyBIG process needs 5°C, whereas the other product just needs ambient temperature. So having that flexibility is key,” he said.

Gavit explained that they can control temperature on the tanks by wheeling them to a thermal fluid line coming in through the wall. The modern, flexible manufacturing suite was awarded the ISPE Facility of the Year award in 2018.

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