Almac and Pfizer celebrate success of patient-led clinical supply model in gene therapy trial
January 22, 2020
Craigavon, N.I., 22 January 2020 – Almac Clinical Services, part of the global contract pharmaceutical development and manufacturing organization, 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.
Natalie Balanovsky, JIT Manufacturing Solutions Manager at Almac comments “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 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: https://www.almacgroup.com/clinical-services/case-study-lean-packaging-process-supports-pfizers-urgent-mission-to-dose-child-with-life-enhancing-gene-therapy/
About Almac Group
‘Partnering to Advance Human Health’
The Almac Group is an established contract development and manufacturing organization providing an extensive range of integrated services across the drug development lifecycle to the pharmaceutical and biotech sectors globally.
Its innovative services range from R&D, biomarker discovery development and commercialization, API manufacture, formulation development, clinical trial supply, IRT (IVRS/IWRS) through to commercial-scale manufacture.
The international company is a privately owned organization which has grown organically over the past five decades now employing over 5,600 highly skilled personnel across 18 facilities including Europe, the US and Asia. Click here to view our facilities.
The company has a global reputation for excellence built over 50 years of client service, delivering expertise right across the drug development lifecycle and offering a tailored solution to each of our clients.