Almac Clinical Services, part of the global contract pharmaceutical development and manufacturing organization (CDMO), the Almac Group, has released a case study detailing how the organization’s successful LEAN packaging process supported Pfizer’s urgent mission to dose a child diagnosed with Duchenne Muscular Dystrophy (DMD), with a life enhancing gene therapy.
 
Pfizer has made a significant effort to extend the life expectancy and improve the quality of life for patients with Duchenne Muscular Dystrophy (DMD), one of nine types of muscular dystrophy. In 2017, Pfizer’s solution—a new mini-dystrophin gene therapy drug (PF-06939926)—was granted Orphan Drug and Pediatric Rare Disease Designations by the FDA and Orphan Medical Product Designation by the European Medicines Agency. The following year, Pfizer embarked upon a Phase Ib, US-based clinical study to assess the safety, tolerability and efficacy of the product.
 
The case study released by Almac is centered on Pfizer’s urgent need to dose the trial’s first pediatric patient an exact quantity of the mini-dystrophin gene therapy in two weeks, three times less than the standard timeframe. In response, Almac developed a bespoke strategy based on the organization’s industry-leading LEAN packaging and labeling principals, that resulted in the patient being dosed in just twelve days, an unprecedented four weeks quicker than the standard.
 
“We are delighted to present our efforts alongside Pfizer and share insights from our collaboration through this case study,” said Natalie Balanovsky, JIT manufacturing solutions manager, Almac. “As clinical trials become more complex with the rise of biologics, we believe that the benefits associated with adapting a patient-centric approach to supply strategy are significant and we are therefore pleased to share our successes with the industry.”
 
The full case study can be downloaded here.