Trientine tetrahydrochloride

New Delhi: In a bid to address the steep cost of imported rare disease therapies , the government is considering modifications to its Promotion of Research and Innovation in Pharma MedTech Sector ( PRIP ) scheme to support domestic development, said Amit Agrawal, Secretary, Department of Pharmaceuticals. Delivering the special guest address at the FICCI Rare Disease Conference 2025, Agrawal said, “Rare diseases in India are as significant as any other public health issue. While we are known as the pharmacy of the world, the need of the hour is to invest in research and development." “Under this vision we launched PRIP and we are at very final stages of making some tweaks to make it better and one of the key focus is the inclusion of rare diseases and orphan drugs,” he said adding that the government is considering rare diseases as a strategic focus with a higher policy support under the scheme.” Launched in 2023 with an allocation of Rs 5,000 crore, PRIP is a Research-Linked Incentive (RLI) initiative aimed at driving innovation in pharmaceuticals and MedTech. Of the scheme’s total outlay Rs 700 crore is earmarked under component ‘A’ for establishing 7 CoEs at NIPER and the rest Rs 4,250 crore is under component ‘B’ for promoting drug development in areas like New Chemical Entities, Complex generics including biosimilars, medical devices, stem cell therapy, orphan drugs, Anti-microbial resistance etc.” Agrawal also highlighted progress under the Production-Linked Incentive (PLI) scheme, noting that eight rare disease drugs—including Trientine (Wilson’s disease), Nitisinone (Type 1 Tyrosinemia), Eliglustat and Miglustat (Gaucher’s), Hydroxyurea (sickle cell anemia), Sapropterin (Phenylketonuria), Sodium Phenylbutyrate, and Carglumic Acid (Hyperammonemia) are now produced domestically. Stating that PLI scheme has brought down treatment costs significantly, he noted, “the annual cost of therapy for Gaucher’s disease has dropped from around Rs 3.6 crore to Rs 3 to 6 lakhs." The production of these drugs began in 2023 following the addition of pharmaceuticals in the PLI scheme with a financial outlay of ₹15,000 crore for the production period of fiscal year 2022-2023 to 2028-29. However, patient groups argue that the therapies currently covered are largely off-patent small molecules, whereas the the greater hurdle for patients is posed by advanced therapies such as large molecules, biologics, and gene therapies , that are still under patent protection. For instance, a disease-modifying therapy Risdiplam marketed by Swiss drugmaker Roche under the brand name Evrysdi for spinal muscular atrophy (SMA)–a neuromuscular disease–costs Rs 60–80 lakh annually, while Novartis’ one-time gene therapy Zolgensma (onasemnogene abeparvovec) entails Rs 14–16 crore. India’s National Policy for Rare Diseases (NPRD), introduced in 2021, has established 13 CoEs—including the latest at AIIMS Patna—to provide treatment and counselling for 63 listed rare diseases. According to the Ministry of Health and Family Welfare, as of August 2024, over 1,100 patients have benefited. Yet advocacy groups stress that the Rs 50 lakh financial support cap is inadequate for advanced therapies , and access to centers remains limited. By Abhijeet Singh ,

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