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‘Keytruda will become most sold orphan drug worldwide by 2024’
  • By Kim Yun-mi
  • Published 2019.06.19 11:11
  • Updated 2019.06.19 11:11
  • comments 0

The Korea Health Industry Development Institute (KHIDI) recently forecast that the global orphan drug market, which stood at $125 billion in 2017, will grow 11.3 percent a year to $262 billion by 2024.

MSD Korea’s immunotherapy Keytruda

This is more than double the overall prescription drug growth rate of 5.3 percent, and will account for 20 percent of the world prescription drug sales in 2024, it added.

The KHIDI report also predicted Celgene would top the list of global orphan drug makers in 2024, followed by Johnson & Johnson, Novartis, Roche and MSD. The top seven pharmaceutical companies will account for more than 35 percent of the total orphan drug sales, widening the financial gap that already exists in the market.

Also, the top 10 global products will account for about 25 percent of the 2024 orphan drug market, with anti-PD-1 immunotherapeutic drugs such as Keytruda and Opdivio accounting for about 10 percent of the total. “Of all the orphan drugs, Keytruda will grow as the world's best-selling drug by 2024 with estimated sales of $12.7 billion," the report claimed, adding that more than half of its sales will occur in the U.S.”

Cancer therapies have led research and development of rare treatments in the global market because there is high unfulfilled demand for cancer treatments and there are various niche diseases in which the numbers of patients are small as the disease-developing mechanisms and symptoms are highly subdivided, the report noted.

According to a 2016 report from the Pharmaceutical Research and Manufacturers of America, 596 projects to develop 566 drugs were either under review by the U.S. Food and Drug Administration or conducting clinical trials with FDA approval in that year. Out of the total, about 40 percent of the projects targeted oncology, followed by genetic diseases (26 percent), neurological disorders (7 percent) and infectious diseases (5 percent).

Also, data at the Ministry of Food and Drug Safety, Korea Drug Development Fund, FDA, and EMA showed that Korean drugmakers are conducting 106 projects to develop 93 drugs, which are registered as orphan drugs or are candidates for orphan drug designation.

Of the pipelines surveyed, 60 percent (64) are biopharmaceuticals, of which 31 percent (20) are cell therapy products (immune or stem cells).

Primary research and development areas consisted of oncology (33 percent), genetic diseases (22.6 percent), neurological diseases (10.4 percent), autoimmune diseases (8.5 percent) and infectious diseases (7.5 percent). Regarding development phase, phase 1 clinical trials accounted for most of the drugs (30), followed by phase 2 clinical trials (26).

Notably, 36 companies, or 76 percent of the total, which are developing orphan drugs (36, 76.6 percent) were small- and medium-sized bio ventures aiming to transfer their technology. Larger firms, such as Hanmi and SK Biopharmaceutical, have also started to focus on orphan drug development. In comparison, Handok, Bukwang and Yuhan are cooperating with bio ventures by licensing in treatments or investing in the ventures.

As of April 2019, 36 pipeline products for 39 types of research in Korea had received orphan drug designation (ODD) from the FDA, while nine drug candidates received the ODD status from the EMA.

Regarding indication, 17 out of the 48 of the ODD drugs designated by the FDA and EMA targeted oncology, followed by genetic disease and infectious disease.

“The global orphan drug market is growing faster than the growth rate of the overall pharmaceutical industry because of the policy support for the development process and the economic advantages such as high drug prices and monopoly rights,” the report wrote. “Such a trend is expected to continue in the future.”

Considering that domestic orphan drug research is dominated by small and midsize venture companies, with most of them still in phase I clinical trials, it is necessary to encourage research and development through extensive administrative support, including tax benefits, the report concluded.


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