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Orphalan

Jan 18
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Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

Paris, France 18 January, 2024 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the approval of its trientine tetrahydrochloride product by China’s National Medical Products Administration (NMPA). This product, already marketed as Cuprior® in the EU, the UK, Saudi Arabia, Switzerland, and Colombia, and as Cuvrior® in the US, represents the first trientine approved by the NMPA. It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.
China is home to an estimated 80,000 individuals with Wilson disease, of whom approximately half have their diagnosis confirmed, and 25,000 are receiving regular treatment. The introduction of new treatment alternatives such as Orphalan’s trientine tetrahydrochloride, particularly for patients who are intolerant to currently available therapies, presents a significant opportunity to meet the needs of a broader segment of the Wilson disease population in China. Orphalan anticipates its product to be available in the country in the coming months.

Dr Naseem Amin, Chief Executive Officer at Orphalan, said: “We are pleased with the marketing authorisation approval of our trientine tetrahydrochloride product in China, which offers a well-tolerated and effective treatment option for patients with Wilson disease. This approval is a crucial step towards providing a much-needed treatment option for the Wilson disease community in China and reaffirms our commitment to improving the experience for patients living with rare diseases worldwide.”

Yan Qin, Director of Wuhan Tongxin (Wilson disease patient group in China) added: “We were thrilled when we heard that NMPA approved Orphalan’s trientine tetrahydrochloride in China. It is a significant step for those of us living with Wilson disease, representing a much-needed alternative treatment option. This approval, a result of Orphalan’s patient-focused approach, offers us a chance to better manage our condition with a new, effective treatment.”

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Early treatment with legacy agents such as D-penicillamine (DPA), trientine HCI, and zinc salts can effectively slow the progression of the disease, however these options may not be suitable for long-term care due to adverse events associated with DPA and the need for close monitoring during treatment with zinc salts and DPA.

About Orphalan
Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the Company develops and deliver innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product has been approved for the treatment of Wilson disease and is available in more than 20 countries, branded as Cuprior® in EU, the UK, Saudi Arabia, Switzerland and Columbia, and as Cuvrior® in the United States. For more information visit www.orphalan.com and follow us on LinkedIn.

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
ICR Consilium:
Tracy Cheung, Sukaina Virji, Davide Salvi
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Apr 21
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Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

– 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.”

 

                                                                         ENDS                        

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

References

[1] Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomized, open-label, non-inferiority, phase III trial, Schilsky et al, Lancet Gastroenterol Hepatol 2022, Published Online September 29, 2022

[2] CuvriorTM USPI, 215760s000lbl.pdf (fda.gov)

May 02
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Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

By Orphalan, Press Release, Private Companies
Press Release.

 

Paris, France 2 May 2022 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialisation company, today announces approval of Cuvrior™, a new salt of trientine (trientine tetrahydrochloride) by the United States Food and Drug Administration (FDA). Cuvrior™ is approved for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the US with about one third of patients developing intolerance1.

Orphalan recently completed a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by non-ceruloplasmin copper (NCC). In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.

Wilson’s disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Orphalan commercializes its trientine tetrahydrochloride product in Europe under the name of Cuprior® and expects to launch Cuvrior™, which has been granted Orphan Drug Designation by the FDA, in the US by early 2023. The approval follows the New Drug Application (NDA) submission for the company’s product last year.

Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented:

“We are delighted with the approval of our product, Cuvrior™, which provides a well-tolerated and effective option for Wilson’s disease patients. At Orphalan, we are committed to delivering innovative therapies, with our drug Cuprior® already approved and launched in European countries, and we look forward to launching Cuvrior™ in the United States. We also plan further national submissions to make our product available to patients globally.”

Mary L Graper, Vice President of Scientific Affairs, Wilson Disease Association, added:

“Wilson’s disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments. The approval of Orphalan’s Cuvrior™ is extremely promising and is reflective of Orphalan’s patient-driven approach. This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease.”

Professor Michael Schilsky, MD, Director, Center for Excellence for Wilson Disease at Yale University, said:

“As a physician, I have seen first-hand how Wilson’s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan’s Cuvrior™ by the FDA is backed by positive data from Orphalan’s multicenter, multinational CHELATE trial – the first head-to-head controlled study of a new trientine salt versus penicillamine. For patients in need, Cuvrior™ represents a well-tolerated and effective alternative to penicillamine, the current standard of care.”

1 Weiss KH, et al. Efficacy and Safety of Oral Chelators in Treatment of Patients With Wilson Disease. Clin Gastroenterol Hepatol. 2013 Aug;11(8):1028-35.e1-2.
– ENDS –

About Trientine Tetrahydrochloride
Trientine tetrahydrochloride is an oral trientine formulation. In the US, trientine tetrahydrochloride has been granted with Orphan Drug Designation for the treatment of Wilson’s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson’s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug Application (NDA).

About Orphalan
Orphalan is an international orphan drug development and commercialisation company. The company delivers worldwide innovative therapies for people living with orphan diseases and is a pioneer in the space. Orphalan was founded in 2011 and has launched Cuprior®. across Europe with its own commercial organization. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:
Orphalan Tel: +33 (0)1 42 49 82 64
info@orphalan.com

Consilium Strategic Communications:
Mary-Jane Elliott, Davide Salvi, Genevieve Wilson
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Sep 02
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Orphalan announces FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease

By Orphalan, Press Release, Private Companies
Press Release.

 

  • NDA supported by positive data from Phase 3 CHELATE clinical trial
  • TETA 4-HCl previously granted Orphan Drug Designation for first-line treatment of Wilson’s Disease

Paris, France – 02 September 2021 – Orphalan SA, a Company that identifies, develops and delivers worldwide therapies for orphan diseases, today announces that the US Food and Drug Administration (FDA) has accepted for review the Company’s New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the first-line treatment of Wilson’s Disease.

Wilson’s Disease is a rare inherited disorder of copper transport primarily affecting the liver and brain, affecting about 1 in every 30,000 people worldwide. For the last 70 years d- Penicillamine has been the only approved first-line treatment of Wilson’s Disease and to which about a third of patients develop intolerance.

TETA 4HCl is proposed as an alternative copper chelating agent to d-Penicillamine as a first line treatment. The Company’s NDA submission follows its previous Orphan Drug Designation by the FDA. The filing is based upon positive data from the CHELATE Phase 3 clinical trial that met its primary efficacy endpoint by demonstrating that TETA 4HCl was non-inferior to d-Penicillamine as measured by copper speciation evaluation of nonceruloplasmin copper (NCC).

Treatment with TETA 4HCl is supported by Orphalan’s novel NCC assay, for which the Company plans to file for FDA approval as a companion diagnostic. The in vitro NCC assay has the potential to provide an important additional tool to support physicians in identifying patients and monitoring their treatment with TETA 4HCl.

Dr Naseem Amin, Chief Executive Officer at Orphalan, commented:

“The FDA’s acceptance of the NDA brings us one step closer to providing first-line treatment to patients with Wilson’s Disease, who currently do not benefit from alternative methods of treatment, beyond D-Penicillamine”.

Notes to Editors

About the CHELATE Study

CHELATE was a Phase 3, multicentre, randomised, open label, active-controlled, noninferiority study conducted in 9 countries at 15 centres designed to evaluate efficacy and safety of trientine tetrahydrochloride compared to d-Penicillamine in patients with stable Wilson’s Disease. Fifty-three adult Wilson’s Disease patients with clinically stable disease for over one year and who met specific inclusion criteria, including laboratory measures of serum non-ceruloplasmin copper (NCC), 24-hour urinary copper excretion (UCE) and liver function tests, were followed for a baseline period for 12 weeks before being randomised 1:1 to either trientine tetrahydrochloride or d-Penicillamine twice daily. The study’s primary endpoint was serum NCC as measured using Orphalan’s proprietary method using copper speciation at 24-weeks post-randomisation. A secondary composite efficacy endpoint was NCC and 24-hour UCE.

Additional secondary endpoints included were: clinical Global Impression of Change (CGIC) score; serum copper and ceruloplasmin levels; the unified Wilson’s Disease Rating Scale (UWDRS); modified Nazer score; cognitive assessments and standard safety assessments. In addition, an independent adjudication committee blinded to the allocated treatment, and study centres assessed key efficacy and safety parameters to determine clinical stability of the patient. Trientine tetrahydrochloride was well tolerated and during treatment, more patients achieved the pre-specified composite endpoint of NCC and 24-hour Urinary Copper Excretion (UCE) within therapeutic target ranges, compared to patients treated with d- Penicillamine, 50% versus 24%. Data from the trial was presented during an oral presentation at EASL’s The International Liver Congress™ 2021.

About Orphalan

At Orphalan, our mission is clear: we are pioneers in orphan diseases. Orphalan identifies, develops and delivers worldwide innovative therapies for people living with Orphan diseases. Orphalan was founded in 2011 and has launched Cuprior™ across Europe with its own commercial organisation. For more information get in touch or follow us on LinkedIn.

For more information, please contact:

Orphalan
Tel: +33 (0)1 42 49 82 64
Email: info@orphalan.health

Consilium Strategic Communications:
Mary-Jane Elliott, Allison Connolly, Genevieve Wilson
Tel: +44 (0) 203 709 5700
Email: orphalan@consilium-comms.com