Almac Pharma Services Supports Packaging and Supply of PTC Therapeutics’ Gene Therapy Upstaza™ for EU Launch

October 17, 2022

Tailored ultra-low temperature solutions enabled first and only approved disease-modifying treatment for AADC deficiency to be approved by EMA

Craigavon, Northern Ireland – 17 October 2022 – Almac Pharma Services, a member of the Almac Group providing innovative pharmaceutical development, manufacture and commercial services, has successfully supported the packaging and commercial supply of PTC Therapeutics, Inc.’s gene therapy product, Upstaza™ (eladocagene exuparvovec).

Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy infused directly into the brain. The product is approved for patients 18 months and older and marketing authorisation is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.

Almac Pharma Services began their partnership with PTC to support the Upstaza product introduction in 2019 through its innovative and tailored ultra-low temperature (ULT) solutions. The Almac team, located in Europe, worked directly with PTC to establish a deep understanding of the unique treatment, and every aspect of the special conditions required to handle, store and process the gene therapy product.

Together they agreed an appropriate strategic programme to efficiently, and effectively, prepare the specialised treatment including packaging, labelling and serialisation over dry ice to guarantee product integrity throughout.

“We are proud to partner with Almac to support packaging and supply of Upstaza for people living with AADC deficiency in Europe,” said Kylie O’Keefe, Senior Vice President, Head of Global Commercial and Corporate Strategy, PTC Therapeutics. “We look forward to continuing our partnership with Almac to bring the best innovation from PTC to the people living with rare diseases who are counting on us.

Grainne Hughes, VP Business Support Operations, Almac Pharma Services commented: “We are delighted to have supported the rapid launch of this ground-breaking gene therapy product which has been marked as a pivotal milestone for AADC deficiency patients. Our ULT specialists understand that each product is unique in its requirements to guarantee its integrity throughout the entire process.  Through our established relationship with PTC, the teams worked diligently to develop a plan and implement the most appropriate robust and timely processes, underpinned by Quality Risk Management, to protect the integrity of Upstaza whilst ensuring it was delivered on time. Many congratulations to our partners at PTC on this fantastic achievement.”

In March 2022, Almac announced the expansion of its dedicated Centre of Excellence and offers an expansive storage facility with specialist suites and freezer capacity to enable inventory stock of crucial product supply ready for rapid distribution when required. The CDMO currently provides solutions for over 25% of EU approved/pre-registration advanced therapeutic medicinal products (ATMPs) having processed and QP released over 700 batches to date.


About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, Instagram, LinkedIn and Twitter at @PTCBio.

About Almac Pharma Services

Tailormade pharmaceutical development and commercial solutions

With over 50 years’ experience, Almac Pharma Services is a world leading outsourcing partner to the global pharmaceutical and biotechnology industry. 

With facilities located in Europe and the US, we provide tailored, quality-led and timely solutions from early and late phase pharmaceutical development, clinical and commercial drug product manufacture, product launch through to commercial packaging and global distribution.

About Almac Group

A unique culture delivering exceptional solutions

The Almac Group is an established contract development and manufacturing organisation 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 commercialisation, API manufacture, analytical services, formulation development, clinical trial supply, IRT (IVRS/IWRS) through to commercial-scale manufacture.

The international company is a privately owned organisation which has grown organically over the past five decades now employing over 6,600 highly skilled personnel across 18 facilities including Europe, the US and Asia.

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