Penicillamine

IN013 restores functional protein levels to reduce systemic copper build-up in patients with Wilson’s disease. Image credit: Shutterstock / Kateryna Kon. The US Food and Drug Administration (FDA) has granted rare paediatric disease and orphan drug designations for IN013, Innorna’s mRNA therapeutic for Wilson’s disease. China-headquartered Innorna will now be in line for tax credits for US-based clinical trials. If IN013 is approved, the potential for seven years of market exclusivity in the designated indication, in accordance with orphan drug status benefits. The rare paediatric disease designation, meanwhile, given to biotechs developing treatments for diseases that affect fewer than 200,000 US paediatric patients, could mean Innorna receives a priority review voucher (PRV) if the therapy is approved. This voucher would allow the company to fast-track a future candidate of its choice through FDA review, meaning it will reach the market four months sooner if approved.  Innorna could also sell the voucher for a quicker cash injection – vouchers have typically been sold for around $100m over the past few years. Wilson’s disease is a rare inherited disease that causes copper levels to build up in the body’s organs, including the liver and brain. The disease is caused by mutations in the ATP7B gene, which normally encodes for the protein responsible for eliminating excess copper. Symptoms of the illness include hepatic and neurological damage, ocular abnormalities, and kidney injury, among others. It is estimated that one in every 30,000 individuals is affected by the disease, with isolated populations at higher risk due to consanguineous marriages. The current standard of care is the administration of copper chelating agents – compounds that bind to copper ions to reduce toxicity and facilitate the removal. In 2022, the FDA greenlit the first new treatment for Wilson’s disease in 30 years, Orphalan’s Cuvrior (trientine tetrahydrochloride). Comprising a copper chelating agent, Cuvrior is for patients who are de-coppered and able to take D-penicillamine, another drug approved for the disease. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Innorna states that current treatments offer limited efficacy and carry risks, such as hepatorenal toxicity or exacerbated neurological symptoms. Some Wilson’s disease patients taking penicillamine have experienced nervous system deterioration . Unlike current approaches that regulate copper levels downstream, Innorna’s IN013 enables the production of the ATP7B protein. The biotech states its asset, developed on an mRNA lipid nanoparticle platform, can restore functional protein levels to reduce systemic copper build-up. Other companies are also developing new treatments for Wilson’s disease. Vivet Therapeutics and Pfizer are developing a gene therapy called VTX-801, currently in a Phase I/II trial (NCT04537377). The candidate acts by inserting a fully functional version of ATP7B into the body. The FDA designations for Innorna come at a time of regulatory spotlight on rare disease pipelines, amid agency job cuts by US President Donald Trump and programme expirations. The PRV programme for paediatric rare diseases, for example, entered its ‘sunsetting’ phase in December 2024 after US Congress failed to reauthorise it. Experts have said the programme’s expiration, along with reduced FDA capacity, could slow down rare disease development. mRNA vaccine coverage on Pharmaceutical Technology is supported by Trilink . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Enhancing Potency, Stability, and Efficiency in mRNA Read this technical note to explore the frontiers of mRNA capping in advanced therapeutic development. As mRNA-based treatments evolve, optimizing capping strategies becomes critical for enhancing stability, translation efficiency, and potency. This comparative analysis of TriLink’s CleanCap® technologies versus traditional enzymatic capping reveals the key innovations setting new benchmarks. Discover how co-transcriptional methods, structural modifications, and dose-sparing potential are accelerating the path from synthesis to impactful protein expression in both research and therapeutic settings. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Trilink Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Trilink Thematic and that your personal data will be used as described in their Privacy Policy

Strategic partnership with Kyuan Xinhai (Beijing) Medical Products Trade Co., Ltd ("SPH Kyuan Trade") to transform access to Wilson's disease treatment for Chinese patients PARIS, March 7, 2025 /PRNewswire/ -- Orphalan, a global pioneer in orphan drug development and commercialization, achieves a historic milestone by launching its Wilson's disease treatment, " Ke Pei Ou" ( 科佩欧 ®), in China. This landmark achievement makes Orphalan the first European pharmaceutical company to introduce an orphan drug to the Chinese Wilson Disease market marking a significant step forward in global rare disease care. This launch is made possible through a strategic partnership with SPH Kyuan Trade, a leading pharmaceutical distributor in China and a subsidiary of Shanghai Pharma Group, one of the country's largest healthcare conglomerates. Together, Orphalan and SPH Kyuan Trade will work to dramatically expand patient access to this critical treatment. A New era for Wilson's disease patients in China Wilson's disease is a rare, life-threatening genetic disorder that affects the body's ability to eliminate excess copper. Without proper treatment, it can lead to severe neurological and hepatic complications. While penicillamine has been a standard therapy for years, many patients cannot tolerate it, leaving them with limited therapeutic options—until now. Orphalan's trientine tetrahydrochloride, "Ke Pei Ou" (科佩欧®), is the first trientine-based treatment approved by the NMPA (National Medical Products Administration) in China. Already available in more than 20 countries under the names Cuprior® (EU, UK, Saudi Arabia, Switzerland, Colombia, New Zealand) and Cuvrior® (US), this treatment should become a life changing therapy for thousands of patients in China. Now, under the name " Ke Pei Ou" ( 科佩欧 ®), it is commercially available in China, bringing new hope to thousands of patients who previously had no alternative. Orphalan expands its presence in China to ensure success Recognizing the strategic importance of China in its global mission, Orphalan has established a local entity in Shanghai and appointed Ms. Grace Li as General Manager. Backed by a team of scientific and medical experts, she will oversee efforts to: Engage with leading healthcare professionals to ensure they have access to the latest clinical data on Wilson's disease management. Facilitate knowledge-sharing and medical education, ensuring informed decision-making for both physicians and patients. Strengthen Orphalan's presence in the Chinese healthcare landscape, ensuring long-term success for "Ke Pei Ou" (科佩欧®). A defining moment for Orphalan's global expansion This milestone is part of Orphalan's broader commitment to advancing rare disease treatments worldwide. By successfully entering the highly regulated and competitive Chinese pharmaceutical market, the company is solidifying its role as a leader in orphan drug innovation and global patient access. Dr. Naseem Amin, CEO of Orphalan, underlined the significance of this achievement: "This is more than a market expansion—this is a breakthrough for rare disease patients in China. Our collaboration with SPH Kyuan Trade represents a pivotal moment in Orphalan's journey. We are not just bringing a treatment to market; we are revolutionizing access to care for those who need it most. This launch reaffirms our commitment to delivering life-changing therapies globally." As Orphalan continues to push the boundaries of scientific excellence and therapeutic innovation, it remains fully committed to ensuring that patients and healthcare providers in China have access to the latest advancements in Wilson's disease treatment. With Ke Pei Ou" ( 科佩欧 ®) now available in China, Orphalan is setting a new standard for what is possible in orphan drug development and global accessibility. About Orphalan Orphalan is an international orphan drug focused pharmaceutical company headquartered in Paris. Founded in 2011, the Company's purpose is to develop and deliver innovative therapies for people living with rare and debilitating diseases. Our commitment is to work collaboratively with the rare disease and scientific communities to develop evidence-based treatments in areas of unmet need. 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, Columbia and New Zealand, as Cuvrior® in the United States, and as Ke Pei Ou" in China. For more information visit and follow us on Linkedin: Orphalan. For more information, please contact: Orphalan Géraldine van den Broek, Head of Corporate & BD Tel: +33 (0)1 42 49 82 64 [email protected] Bien Commun Advisory Marie-Charlotte Potet 06 70 91 25 23 [email protected] Logo - SOURCE Orphalan WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED