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.
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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.
When the first patient enrolled in the study at a clinical site in North Carolina, Pfizer had no time to waste in administering the potentially transformational drug. But the necessity of using a patient demand-led drug supply model presented several behind-the-scenes, packaging-based challenges.
Here’s how, with help from Almac Clinical Services, Pfizer successfully overcame these challenges and kept a child in urgent need of life changing treatment at the heart of its operation.
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