Almac Clinical Services, part of the global CDMO Almac Group, has released a case study detailing how its lean packaging process supported Pfizer’s urgent mission to dose child diagnosed with Duchenne Muscular Dystrophy (DMD), with life-enhancing gene therapy.
Natalie Balanovsky, JIT Manufacturing Solutions Manager at Almac, commented: “We are delighted to present our efforts alongside Pfizer and share insights from our collaboration through this case study. As clinical trials become more complex with the rise of biologics, we believe that the benefits associated with adopting a patient-centric approach to supply strategy are significant and we are therefore pleased to share our successes with the industry.”
Mini-dystrophin gene therapy drug
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 Paediatric 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 centred on Pfizer’s urgent need to dose the trial’s first paediatric 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 organisation’s industry-leading LEAN packaging and labelling principals, that resulted in the patient being dosed in just twelve days, an unprecedented four weeks quicker than the standard.
The full case study can be downloaded from the company website.