Almac Group’s Integrated CMC Services Support US Approval of Wellstat Therapeutics’ First Commercial Orphan Drug Product
September 9, 2015
Craigavon, N.I., UK, – 9 September 2015 – Almac Group, the global contract development and manufacturing organization, wishes to congratulate its long term client partner Wellstat Therapeutics on the commercial approval of their first orphan drug product XURIDEN™ (uridine triacetate). XURIDEN has been developed as a treatment for the rare disease Hereditary Orotic Aciduria (HOA).
The partnership began more than 5 years ago with Almac’s Sciences and Pharma Services business units providing expert support to Wellstat Therapeutics for the development of both the Active Pharmaceutical Ingredient (API) and formulation of the drug product. Having manufactured clinical, registration and PV batches, Almac will now produce the commercial product from its UK headquarter facilities, on behalf of Wellstat Therapeutics.
Following a successful Pre-Approval Inspection (PAI) of Almac’s UK facilities in May of this year and the subsequent FDA approval of the product on September 4, 2015, XURIDEN is forecast to be commercially available in early 2016.
Leveraging Almac’s unique integrated API and Drug Product expertise prior to and through NDA submission and approval, Wellstat Therapeutics worked with Almac to take advantage of a single partner approach with smooth transition through the drug development process and now commercialization, saving time, transfers and other uncertainties inherent in a multi-supplier process.
David Downey and Charles Shields, VP’s of Almac’s Drug Product and API teams respectively commented “It is great to see a product that Almac has supported long term in both development of the API and Drug Product achieve commercial approval. The value of integrating development and the flexibility it offers our partners cannot be overstated.”
“Until FDA approval of XURIDEN, there were no approved treatments for patients with hereditary orotic aciduria, a rare, potentially life-threatening disease”, said Michael Bamat, VP Research & Development for Wellstat Therapeutics. “Almac has been an integral part of the API and drug product development team for XURIDEN.”
Hereditary orotic aciduria (HOA) is a genetic disorder caused by mutations reducing the activity of uridine monophosphate synthase (UMPS), which converts orotic acid to UMP. Uridine deficiency underlies many of the symptoms of HOA, including hematologic abnormalities, developmental delays, failure to thrive, and if untreated, early mortality. Orotic acid biosynthesis is normally subject to feedback inhibition by intracellular uridine nucleotides and is therefore overproduced in HOA. The resulting increased levels of orotic acid can cause crystalluria and occasional obstructive uropathy.
The goal of uridine replacement therapy in HOA is to provide the uridine that these patients cannot make adequately themselves in order to restore intracellular uridine nucleotide concentrations, enabling and supporting the biosynthetic functions of pyrimidine nucleotides and their derivatives, and also normalizing orotic acid production.
XURIDEN is a pyrimidine analog for uridine replacement indicated for the treatment of hereditary orotic aciduria. Oral administration of XURIDEN delivers 4 to 7 times more uridine than oral administration of uridine itself.
Important Safety Information
- Contraindications – None
- Warning and Precautions – None
- Adverse reactions – No adverse reactions were reported with XURIDEN in patients with HOA
- Pregnancy – There are no available data on XURIDEN use in pregnant women to inform drug-associated risk
- Lactation – There are no data on the presence of XURIDEN in human milk, the effect on the breast-fed infant or the effect on milk production. The development and health benefits of breastfeeding should be considered along with the mother’s clinical need for XURIDEN and any potential adverse effects on the breastfed infant from XURIDEN or from the underlying maternal condition.
- Pediatric – The safety and effectiveness of XURIDEN have been established in pediatric patients.
For the full Prescribing Information go to http://www.xuriden.com/FPI.pdf.
About Almac Group
‘Partnering to Advance Human Health’
The Almac Group is an established contract development and manufacturing organization that provides an extensive range of integrated services to over 600 companies globally within the pharmaceutical and biotech sectors. The services range from R&D, biomarker discovery and development, API manufacture, formulation development, clinical trial supply, IXRS® technology (IVRS/IWRS) through to commercial-scale manufacture.
The international company is a privately owned organization that has organically grown over 40 years and now employs in excess of 4,000 highly skilled personnel. Almac is headquartered in Craigavon, Northern Ireland with operations across the US (Pennsylvania, North Carolina and California) and in Asia (Singapore and Tokyo).
About Wellstat Therapeutics
Wellstat Therapeutics Corporation is a privately-held biopharmaceutical company located in Gaithersburg, Maryland. Wellstat Therapeutics is committed to discovering, developing and commercializing products that will provide new and improved treatments for patients in the fields of oncology and metabolic, neurometabolic and neurodegenerative diseases. Wellstat Therapeutics is part of the Wellstat group of companies, http://www.wellstat.com. For more information, please visit the website at http://www.wellstattherapeutics.com.