A partnership between contract development and manufacturing organization (CDMO) Almac Group and Amicus Therapeutics has resulted in the European approval of Amicus’ first orphan drug product and precision medicine Galafold (migalastat, also referred to as AT1001). Galafold was approved as a first line therapy for long-term treatment of adults and adolescents—aged 16 years and older—with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and who have an amenable mutation.

Amicus and Almac began a partnership in 2009 when Amicus sought an outsourcing partner for Phase III clinical manufacture and packaging of its solid oral AT1001 compound. Almac’s pharmaceutical development and clinical teams worked with Amicus to advance the drug product through scale up, registration and now onwards into commercial supply. Almac is manufacturing and packaging the drug product and supporting Galafold’s launch across Europe from its UK headquarters in Northern Ireland.

Leveraging the Almac advantage of integrated development and commercial services ensured a smooth transition from development to commercialization for Amicus.  Utilizing the experience of Almac’s multidisciplinary product launch team incorporating quality, packaging design, regulatory and distribution, aided Amicus in navigating EU market requirements. Upon marketing authorization, Galafold was packed and distributed to the German market.

“It’s great to see the launch of this precision medicine addressing a debilitating unmet medical need and it is gratifying to have played our part in its development and commercialization,” said David Downey, vice president, commercial operations, Almac. “Amicus drew upon many of the services Almac has to offer, from development, through clinic, and into commercialization. We look forward to a growing partnership as Amicus services the needs of the Fabry patient population who have an amenable mutation.”

“Providing quality drug product and sufficient drug supply is critical to ensuring a successful launch of Galafold for Fabry patients who have an amenable mutation,” said Enrique Dilone, senior vice president, technical operations, Amicus Therapeutics. “Almac has been an outstanding outsourcing partner for Amicus and we look forward to continuing the relationship as Galafold becomes available across the EU.”

Visit Almac at Tables 43, 44