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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

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