Samoh Pharm’s expansion after Voxzogo reimb…orphan drug portfolio

The first achondroplasia treatment is now reimbursed…the first leap towards rare disease business

Through collaboration with BioMarin Pharmaceutical, the company leverages its global innovation and domestic execution capabilities

​"Rare disease business is about creating a treatment setting, not just selling a product"

The reimbursement of 'Voxzogo (vosoritide),' the first treatment for pediatric achondroplasia in South Korea, under the national health insurance has opened a new treatment option for patients. At the same time, it has provided a turning point for Samoh Pharm to expand its rare disease business, spanning over 20 years, to the next level.

Chang-Deok Ji, Managing Director of the Orphan Drug Business Unit at Samoh Pharm, who spearheaded the domestic introduction and launch of Voxzogo, sought to establish a new commercialization model through this project, connecting global innovative therapeutics with the domestic healthcare environment.

​DailyPharm met with Ji from Samoh Pharm's Orphan Drug Business Unit to discuss the significance of the Voxzogo launch, the company's rare-disease business strategy, and its future vision.

​"Voxzogo is a project that takes our accumulated experience to the next level"

Chang-Deok Ji, Managing Director of the Orphan Drug Business Unit at Samoh Pharm

​Director Ji evaluated Voxzogo as a project with a distinctly different nature from previous rare-disease drug launches.

​Over the past 20 years, Samoh Pharm has accumulated specialized expertise by introducing and supplying a range of orphan drugs in South Korea, including treatments for Fabry disease, Gaucher disease, Pompe disease, mucopolysaccharidosis (MPS), and phenylketonuria (PKU).

Ji explained that while Voxzogo is an extension of this trajectory, its significance lies in the fact that it served as an opportunity to upgrade the company's business operations and commercialization capabilities rather than simply adding a new product to the portfolio.

​"Rather than just being a single new product, Voxzogo is part of a project that implements our accumulated experience and know-how in a new way, and it marks a turning point for our rare disease business to leap forward to the next level," Ji said.

​Voxzogo is the first pharmacological treatment option approved in South Korea for pediatric patients with achondroplasia aged 4 months and older whose growth plates are not yet closed. It is recognized as an innovative therapeutic that regulates the FGFR3 signaling pathway through a CNP analog mechanism.

"While being the country's first treatment option is important, the greater significance lies in offering patients and caregivers the possibility that 'treatment is now a choice,'" Ji said. "Since achondroplasia accompanies not only short stature but also recurrent surgeries and various complications, we can expect long-term health management and improved quality of life beyond just growth enhancement."

​"Rare disease business is about creating a treatment setting, not just selling a product"

​Ji has built a 25-year career in the pharmaceutical industry, dedicating more than 15 years to the rare disease sector. He has led organizational setup and new product launches at global rare-disease enterprises, executing a range of commercialization projects. The Voxzogo project is an example of how to optimize and apply this extensive experience in the domestic medical landscape.

​"Our role is to connect globally accumulated innovation with the domestic healthcare environment so that it translates into patient treatment," he explained.

​The Voxzogo business was implemented through close collaboration with BioMarin Pharmaceutical. The structure leverages BioMarin's R&D capabilities and global experience, alongside Samoh Pharm's domestic execution capabilities, to successfully integrate the innovative therapeutic into local clinical practice.

However, Ji emphasized that the core of the rare disease business lies in building a comprehensive therapeutic environment rather than the product itself.

​"The rare disease sector is not an area where a market is formed simply by launching a single product. From patient identification and diagnosis to the initiation of treatment and long-term management, every stage must be organically linked for the therapy to reach the patient successfully," Ji noted.

Based on this philosophy, Ji established an operational framework within the Orphan Drug Business Unit that seamlessly connects medical affairs, marketing, and patient support functions.

​"What matters more than the size of the organization is expertise and execution," Ji said. "Our competitiveness lies in providing rapid and sophisticated support to healthcare providers and patients based on a lean yet powerful organization."

​"Voxzogo is not the end, but a greater beginning"

​Ji assessed that the impact of Voxzogo extends beyond the performance of a single product, serving as a blueprint for the future direction of the rare disease business.

"What we confirmed through Voxzogo was not merely the market potential of a single innovative therapeutic," he shared. "Rather, it proved that we could realize a patient-centric rare disease commercialization model by establishing a sophisticated Pricing and Reimbursement (P&R) process based on a Risk Sharing Agreement (RSA), creating a performance evaluation framework utilizing Real-World Data (RWD), and securing agile execution through a lean and robust organization."

Based on this milestone, Samoh Pharm plans to expand its commercialization capabilities by leveraging its accumulated rare disease expertise across its broader ethical drug (ETC) portfolio and pipeline.

Furthermore, the company envisions continuously developing an integrated collaboration model that connects medical professionals, patients, the government, academic societies, and global partners.

Ji said, ​"In the rare disease business, the process of connecting patients with treatment opportunities holds far greater meaning than short-term financial performance," and added, “We will continue collaborating with global partners so that more patients can gain access to innovative treatments."

Ji concluded by noting, ”The very moment a patient begins their treatment is when our role begins,” and added, “Voxzogo is not just a single project, but a starting point for our new therapeutic opportunities for an even broader population of rare disease patients."