Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease

• Cuvrior™ now available in the US for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine

— Oral administration offers dosing flexibility and portability

Paris, France, April 20, 2023 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the commercial launch in the US of Cuvrior™, a new trientine tetrahydrochloride (TETA-4HCl). Cuvrior™ is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.

Cuvrior™ was approved by the United States Food and Drug Administration (FDA) and was granted Orphan Drug Exclusivity (ODE) in April 2022 for this patient group. Physicians can now discuss the availability of Cuvrior™ as a treatment option with their patients and can access important information here about prescribing Cuvrior™.

Compared with the standard of care, Cuvrior™ facilitates precise dosing by providing a small, scored tablet in a blister pack. Additionally, its room temperature stability eliminates the need for refrigeration, making it a more practical solution for patients with busy lifestyles.

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Since 2019, Orphalan has commercialized its trientine tetrahydrochloride product in Europe under the name of Cuprior® and approximatively 1,000 Wilson disease patients have received treatment, with a positive track record on safety and efficacy.

Cuvrior™ was approved by the FDA on the basis of data from the Company’s phase III CHELATE trial, the first prospective randomized trial comparing penicillamine with TETA-4HCl. During the trial, an innovative assay was developed to measure non-caeruloplasmin bound copper (NCC), the free and potentially toxic pool of copper in the blood. Using this NCC measurement in patients previously receiving maintenance penicillamine therapy, TETA-4HCl was determined to be non-inferior to penicillamine at the primary endpoint of the study (24 weeks), with the same observation at the end of the extension phase of the study (one year from randomization).

Dmitry Paramonov, President Orphalan US, commented: “We are thrilled to introduce Cuvrior™ in the US, a safe and innovative treatment option for patients and caregivers managing Wilson disease. Our distribution partners have Cuvrior™ stocked and ready for immediate use. At Orphalan, our focus is on providing targeted solutions for rare diseases, as demonstrated by the successful launch of Cuprior® in Europe. We are eager to expand availability of Cuvrior™ to as many patients as possible, underscoring our dedication to making a meaningful impact on the lives of rare disease patients worldwide.”

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About Orphalan

Orphalan is a pioneering, international orphan drug development and commercialization company. Founded in 2011, the company develops and delivers innovative therapies for people living with orphan diseases, and is initially focused on Wilson disease, a rare genetic disorder that can be life-threatening if untreated. Orphalan commercializes Cuprior^®, its trientine tetrahydrochloride product for the treatment of Wilson disease in Europe, and has launched Cuvrior™ in the US. For more information visit www.orphalan.com and follow us on LinkedIn.

About Cuvrior™

Cuvrior™ is an innovative new oral formulation of trientine and the first new advance in Wilson disease in over 30 years. Cuvrior™’s FDA approval was supported by a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by serum non-caeruloplasmin copper assay (NCC) ^[1,2]

About Wilson disease

Wilson disease is a rare genetic disease that is characterized by gradual accumulation of dietary copper over time, possibly to life-threatening levels. Affected individuals are unable to effectively excrete copper naturally through the digestive tract. Common symptoms associated with Wilson disease include progressive liver disfunction, neurological disorders such as severe tremors, and mental health deterioration. About 1 in 15 patients eventually need a liver transplant. Wilson disease affects nearly 1 in every 30,000 people worldwide.  

For more information, please contact: 

Orphalan:

Géraldine van den Broek, Head of Corporate & BD

Tel: +33 (0)1 42 49 82 64

info@orphalan.com

Consilium Strategic Communications: 

Mary-Jane Elliott, Tracy Cheung, Davide Salvi

Tel: +44 (0) 203 709 5700 

orphalan@consilium-comms.com