It’s rather unfamiliar to Koreans, but Recordati is a pharmaceutical company with a history of nearly 100 years. It acquired Orphan Europe headquartered in Paris in 2007, and launched the Recordati Rare Disease business unit within the group. In 2022, it also took over U.K.-based EUSA Pharma, which had rare disease treatments, before turning toward the Asian market in earnest. Particularly, Recordati noted the potential of the Korean market. That was because the market share of Sylvant (siltuximab), a multicentric castleman disease (MCD) treatment, was highest in Korea worldwide, following only the United States.
“In the short term, we want to become the hope of desperate patients, and in the mid- to long-term, we want to become a company that comes to people’s mind when they think about rare diseases,” Recordati Korea CEO Lee Yeon-jae said in a recent interview with Korea Biomedical Review. “We will strive to develop and supply rare disease treatments with high unmet demands and limited options.”
Lee worked at Novartis Korea, served as CEO of UCB Korea, and was the managing director for the rare blood disease business unit at Sanofi Genzyme. She also headed EUSA Pharma Asia-Pacific Regional Headquarters before leading Recordati Korea. Lee doubles as Recordati’s Asia-Pacific regional headquarters because Recordati Korea is the Italian company’s regional headquarters encompassing 10 other regional markets.
most other global pharma companies with regional headquarters in Singapore or Hong Kong, Recordati has bet on Korea. KBR met with CEO Lee, who said she wanted to show the headquarters office’s selection of Korea as regional headquarters was right through the success of Recordati Korea, to learn about the company’s rare disease treatment pipelines and its strategy to address the Korean rare disease treatment market.
Answer: Recordati is a pharmaceutical company established in 1926, headquartered in Milan, Italy. It operates branches in Europe, the Middle East, the U.S., Canada, Russia, Australia, Japan, and some Latin American countries, supplying primary care, such as hypertension and diabetes, specialty care, and rare disease treatments to about 150 countries. To strengthen its rare intractable disease portfolio, the company acquired U.K.-based EUSA Pharma in March 2022. Recordati Korea is the Korean branch of Recordati and its Asia-Pacific headquarters, encompassing countries such as Taiwan, Hong Kong, and Singapore. Recordati Korea will supply and market MCD treatment Sylvant (siltuximab) and hyperammonemia treatment Carbaglu (carglumic acid).
Q: Why Recordati chose Korea?
A: EUSA Pharma, which Recordati acquired to strengthen its rare intractable disease portfolio, was a small company focusing only on the European and U.S. market in the initial stages. Afterward, it began to pay attention to emerging markets, establishing Asian and Latin American branches. However, EUSA Pharma had no recognition in Korea and other Asian countries. In March 2022, Recordati took over EUSA Pharma, but Recordati also had little recognition in Asian countries. This notwithstanding, the market share of MCD treatment Sylvant was second-highest in Korea worldwide, following the U.S. Accordingly, the company regarded Korea as a significant market and wanted to create good examples in Asia by starting from Korea. That is why we decided to maintain a foothold in Korea.
Multicentric castleman disease is a pre-stage lymphoma, a type of blood cancer. It is caused by excessive expression of interleukin-6, an immune protein that regulates our body’s immune and inflammatory responses, attacking various body organs. It can occur anywhere there are lymph nodes. Along with lymph node enlargement, it is accompanied by symptoms, such as extreme chronic fatigue that makes daily life impossible, weight loss, fever, night sweating, organ hypertrophy at the liver and spleen, skin changes, and neuropathy.
Q: Patients with rare diseases experience a “diagnostic odyssey” for about seven years before being confirmed. Although the period is getting shorter due to the development of rare disease diagnosis technology, they still have to wander around many hospitals. MCD patients may not be an exception. Right?
A: MCD patients undergo a diagnosis odyssey for four to five years before confirmation, wandering around internal medicine, family medicine, and rheumatology departments. One of its typical symptoms is extreme fatigue. However, everyone experiences fatigue, so people tend to overlook it. However, MCP patients’ fatigue is so severe that they can hardly hold a spoon or cross the threshold of bathrooms. Moreover, even if a patient complains of chronic fatigue, it cannot be diagnosed if the medical workers are unaware.
Also, patients with multiple castle diseases sweat a lot at night. However, even if they sweat a lot at night, neither the patient nor the medical workers suspect MCD. As it is difficult to be diagnosed with one or two symptoms, they go through a diagnostic odyssey and miss the golden time of treatment. To diagnose MCD, comprehensive interpretation and analysis focusing on various lab parameters are needed, which, in turn, requires experience.
Q: If so, do you think the unmet demand for treating MCD in Korea is in the “diagnosis” part?
A: That’s right. In the case of MCD, there is a cure, so there are few cases where treatment is unavailable due to the lack of treatment. It is also already covered by health insurance. However, since it is a disease not recognized by the public and medical workers, the diagnosis is difficult without medical professionals with extensive experience in rare diseases.
Multidisciplinary treatment is needed for accurate diagnosis of MCD. Medical workers in hematology, pathology, and radiology need to understand what MCD is.
The prevalence of MCD ranges from 2.5 to 5.8 per 1 million people. In the case of Korea, it is counted as 1.7 per million. The prevalence varies from country to country because there are many hidden patients due to difficulties in diagnosis.
explains why we need to focus on finding hidden patients. We seek ways to share experiences among doctors at various departments through “preceptorship.” We are considering applying a two-track approach by enhancing expertise in medical workers with a higher understanding of MCD while increasing its understanding among those with low awareness.
Q: You also have hyperammonemia treatment. What unmet needs does this disease have?
A: Unlike Europe, Carbaglu is reimbursed only for “emergency” use in Korea. However, in the case of hyperammonemia, ammonia levels in the blood fluctuate widely and do not fall immediately by taking drugs for three to five days. Therefore, as insurance benefits are given in the short term and only to emergency cases, although the disease heavily depends on the patients’ condition, patients must pay all expenses themselves after five days of administration.
Hyperammonemia is a disease that can cause brain damage when it worsens. Brain damage has permanent consequences, so consideration is needed for this aspect. The insurance coverage part is regrettable because it is a rare disease with a tiny number of patients.
Hyperammonemia is a disease that causes problems with the operation of individual enzymes and cannot process ammonia effectively, increasing its concentration and neurotoxicity. Abnormalities in the urea circuit are the most common cause of hyperammonemia due to metabolic diseases. The overall urea circuit metabolic disease occurs in about 1 per 35,000 people. However, the number of hyperammonemia patients with gene mutations capable of using Carbaglu is very small.
Q: Besides Sylvant and Carbaglu, what other rare disease treatments do you plan to release?
A: We will introduce treatments for high-risk neuroblastoma and Cushing’s syndrome in 2025. They are already in use with permission in Europe. The treatment for Cushing’s syndrome is a portfolio that Recordati had. However, neuroblastoma treatment is a portfolio expanded by acquiring and merging EUSA Pharma, and its launch in Korea has been delayed due to procedures such as mergers and acquisitions. Therefore, we will hurry to introduce the two treatments in Korea as soon as possible.
Q: Global pharma companies find it difficult to cope with Korea’s health insurance system. However, even good treatments are pie in the sky for patients without insurance benefits. Do you have any specific strategy for negotiating drug prices?
A: We will do our best based on the treatments’ clinical data. The government needs to find meaning in providing benefits, and pharmaceutical companies need to receive appropriate benefits to continue to invest.
Neuroblastoma accounts for 22 percent of solid cancer in infants under 12 months. Although the infant population is shrinking and the number of patients will decrease, the treatment is significant if it can improve the survival rate of patients with neuroblastoma notably. Because each patient has a value that the price of a drug cannot judge, I hope the authorities judge it in a greater sense rather than focusing only on economic feasibility.
‘Hope Recordati Korea will set an example of regional headquarters’
Q: How is the Korean market as global pharma see it?
A: Global pharmaceutical companies experience difficulty setting long-term strategies because the environment is changing rapidly in Korea, and the nation has many regulations. This notwithstanding, Korea is included in their priority list in releasing new drugs because the Korean market is important. Although the Korean pharmaceutical industry has been developed to an excellent level, I hope it will not be undervalued due to a handful of regulations, including drug prices. In addition, the domestic policy needs to become more flexible for Korea to represent the country and Asia and make its voice heard worldwide.
Q: After all, is drug price negotiation the highest barrier to the Korean market for global pharma?
A: The lack of flexibility in drug pricing is most regrettable. Although I am in charge of Recordati’s Asian headquarters, I have many opportunities to tell the headquarters about Korean situations. Every time, I emphasize that it is worth investing in the Korean market as a high unmet demand exists. However, the headquarters cannot help but consider what benefits the release of its drugs here will have. Korea has sufficient expertise of medical professionals, an excellent clinical environment, and high internal staff capabilities. So, it is most regrettable that the process does not go well due to the external environment. Still, the good news is that the negotiation period for permits and drug price negotiations is getting shortened.
Q: Do you have some concluding message?
A: I hope the success of Recordati Korea will set a good example for other global pharma companies to consider Korea when they choose Asian headquarters.
출처 : KBR(https://www.koreabiomed.com)