Nobelpharma Leading the Development of Rare Disease Treatments

Hunterraze Joint Development Research Team Founded in 2017
Multiple Rare Pediatric Disease Treatment Pipelines
Clinical Trials Planned in the US Next Year... Preparing for Overseas Investment Attraction

[Asia Economy Reporter Hyungsoo Park] GC Green Cross's Hunter syndrome treatment 'Hunterase,' the second developed worldwide, has seen its sales rapidly increase every year. The domestic prevalence of 'Mucopolysaccharidosis type II,' known as Hunter syndrome, is reported to be 0.74 per 100,000 people. The annual drug cost per patient reaches 400 million KRW. Despite the extremely small number of Hunter syndrome patients, Hunterase sales exceeded 50 billion KRW last year.

The rare disease treatment market had already grown to 150 trillion KRW by 2020. It is expected to grow at an average annual rate of 10.8% until 2026, reaching a market size of 280 trillion KRW. This is why the global pharmaceutical industry is focusing on rare disease treatments.

In Korea, there are new drug developers specializing in rare disease treatments. The research team that co-developed Hunterase with GC Green Cross established Nobelpharma in January 2017. Based on therapies designed to cross the blood-brain barrier, they mainly develop treatments for rare pediatric diseases.

The criteria for defining rare diseases vary by country. In the United States, which has the largest rare disease treatment market, rare diseases are defined as those affecting fewer than 200,000 people. Although each rare disease has a small number of patients, there are about 7,000 types of rare diseases. Among rare diseases, 69.9% occur during childhood. Cases occurring in both childhood and adulthood account for 18.2%, and those occurring only in adulthood account for 11.9%. The global prevalence of rare diseases was 1.2 per 100,000 people based on the world population in 2017.

As interest in rare diseases grows, more countries are providing government-level support for treatment. The United States established legislative systems such as the Orphan Drug Act in 1983 and the Rare Diseases Act in 2001, creating government support organizations. They actively promote pharmaceutical industry support and registry research and development for rare disease treatments. In the U.S. healthcare system, rare disease patients can receive medical support depending on income level, economic activity status, and degree of disability.

In June 2017, the U.S. FDA announced the Rare Disease Drug Modernization Plan, committing to review all new rare disease drug designation requests within 90 days. The FDA planned to ensure timely responses through a special team of experts and improve efficiency through joint reviews with responsible departments. They also provide opportunities to directly hear and reflect patient experiences in collaboration with the private organization National Organization for Rare Disorders (NORD).

Until a few years ago, few multinational pharmaceutical companies attempted to develop rare disease treatments due to lack of economic feasibility compared to anticancer drugs. Physicians responsible for patient treatment in hospitals recognized the need for treatments, but rare disease treatments available on the market were very limited. This was the reason for establishing Nobelpharma. They are conducting joint research with the Mucopolysaccharidosis Research Society to solve social and daily difficulties faced by patients and families suffering from rare diseases.

Nobelpharma develops new drugs that can treat rare pediatric diseases with unfamiliar names such as Sanfilippo syndrome type A, GM1 gangliosidosis, and Morquio syndrome type B.

Sanfilippo syndrome type A is a hereditary lysosomal storage disease (LSD) in which heparan sulfate accumulates in the central nervous system (CNS) including the brain and spinal cord, causing progressive damage to the CNS. It is the most prevalent among mucopolysaccharidosis (MPS) type 3. Sanfilippo syndrome type A is reported to occur in 1 out of 100,000 newborns. There is currently no effective treatment, so patients rely on symptomatic therapy aimed at alleviating symptoms.

Sanfilippo syndrome type A is an autosomal recessive genetic disorder caused by a defect in the gene (SGSH) responsible for producing the heparan sulfate-degrading enzyme. Because physical changes at birth are mild, diagnosis is often delayed, but from ages 2 to 6, very aggressive behavior and developmental delays become apparent. As symptoms progress, patients lose motor and functional abilities and typically die around age 15.

Park Chan-ho, CEO of Nobelpharma, introduced, "We are researching a treatment for Sanfilippo syndrome type A that directly supplements the deficient enzyme in the central nervous system by applying the intracerebroventricular (ICV) administration method established through Hunterase clinical trials."

GM1 gangliosidosis is a hereditary lysosomal storage disease (LSD) in which GM1 ganglioside accumulates in the central nervous system (CNS), leading to destruction of nerve cells in both the central and peripheral nervous systems. It is an autosomal recessive genetic disorder caused by mutations in the GLB1 gene. It is known to occur in 1 out of 150,000 newborns. It is classified into three types based on the age at which signs and symptoms first appear, with the most severe infantile form accounting for over 60%. It is a severe disease causing death between ages 2 and 4.

Morquio syndrome type B, also called mucopolysaccharidosis type IVB (MPS IVB), is a rare lysosomal storage disease (LSD) inherited in an autosomal recessive manner. It broadly affects the musculoskeletal system and the entire body. Complications such as vision and hearing loss, organ enlargement, dislocation, and respiratory failure occur, reducing patients' quality of life.

CEO Park said, "Awareness of rare disease treatments is still low," and explained, "Investments up to Series C have mainly involved the Korea Development Bank and pharmaceutical companies."

He added, "We expect to start clinical trials for major pipelines in the U.S. next year and are currently discussing attracting overseas investment."

This content was produced with the assistance of AI translation services.