Romiplostim: A breakthrough in rare disease treatment – An inside look with Abijar Bhori

In the world of rare diseases, every breakthrough is more than just a scientific achievement—it’s a beacon of hope for patients who have long struggled with limited treatment options. At Enzene, we are committed to pushing the boundaries of innovation to develop life-changing therapies. One such success story is Romiplostim, a thrombopoietin receptor agonist that has transformed the management of immune thrombocytopenia (ITP).

Behind this milestone is a dedicated team of scientists, researchers, and visionaries who worked tirelessly to bring Romiplostim to life. In this exclusive interview, we sit down with Abijar Bhori, the Project Champion for Romiplostim, to uncover the inspiration, innovations, and impact behind this rare disease therapy. From scientific advancements to patient benefits, Abijar shares insights into what makes Romiplostim a game-changer in the treatment landscape.

Join us as we explore a journey fueled by passion, innovation, and life.

1. Can you share the inspiration behind the development of Romiplostim?

The inspiration behind Romiplostim stemmed from Enzene’s vision to provide trusted, effective and affordable medicines for patients with immune thrombocytopenia (ITP). Traditional treatments, such as corticosteroids and splenectomy, often come with significant side effects. In India, the unavailability of the originator drug Nplate® (Romiplostim) posed a significant challenge for ITP patients, limiting access to an effective treatment option. The widespread adoption of originator biologics in India has been hindered by their high cost, the need for imports, and limited medical insurance coverage, making it essential to develop high-quality, cost-effective biosimilars like Romiplostim. Enzene’s Romiplostim biosimilar was developed to bridge this gap, offering an affordable and accessible alternative without compromising on efficacy or safety.

2. What excites you the most about the future of thrombopoietin receptor agonists?

TPO receptor agonists have a highly promising future. Since their introduction in 2008, they have been adopted in over 100 countries as a first-line treatment and are now the preferred choice for ITP patients due to their high response rates. Advancement in precision medicine is paving the way for more refined and personalized approaches to treating ITP and other platelet-related disorders. Very soon AI will further optimize dosing strategies and patient responses, leading to more individualized treatments. We must also note that the increasing availability of biosimilars like Enzene’s Romiplostim is making TPO-RAs more accessible in cost-sensitive markets like India. This is particularly exciting because it ensures that more patients can benefit from advanced biologics which are more specific in its mechanism without the financial burden associated with originator drugs.

3. How does Romiplostim improve the quality of life for ITP patients compared to alternative treatments?

Romiplostim provides a significant advantage for ITP patients by delivering a more predictable and sustained increase in platelet counts. With response rates of 40-60% and platelet levels consistently above 50×10⁹/L—without bleeding or the need for rescue therapy—it presents a realistic treatment goal. Additionally, it offers fewer side effects compared to conventional therapies such as steroids or immunosuppressants. Unlike corticosteroids, which can cause weight gain, mood swings, and bone loss, Romiplostim specifically targets platelet production without widespread immune suppression. For Indian patients, the availability of Romiplostim biosimilar is a game-changer.

4. If you could share one key message about Romiplostim’s impact, what would it be?

I believe Romiplostim has redefined the standard of care for ITP patients. It has not only provided a long-term solution that controls their condition but also treats them with minimal side-effects, and enables them to live a fuller, more active life. In India, availability of affordable Romiplostim by Enzene marks a significant step towards ensuring equitable access for Indian patients to advanced biologics.

5. Have there been any innovations in its production that enhance cost-effectiveness or sustainability?

At Enzene, our team is dedicated to finding innovative solutions to develop cost-effective and affordable medicines for the broader population. Romiplostim is a peptibody-class drug that presents significant production challenges. As a non-natural molecule, it has a high propensity to generate product-related variants. Enzene’s Romiplostim manufacturing process is carefully designed and thoroughly characterized – from fermentation and refolding to final polishing purification – resulting in a high-yield process and a product that closely matches the originator. The process is highly scalable, with a single manufacturing-scale batch capable of producing approximately 75,000 doses, ensuring both sustainability and affordability for patients.