A string of new drug candidate materials and biobetters (new drugs for bio-pharmaceuticals) from Korean pharmaceutical and biotechnology companies have been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA). Domestic pharmaceutical and biotech companies are expected to make an early entry into the U.S. market, which is the largest pharmaceutical market in the world.
The Korean biotech and pharmaceutical companies that have recently received ODD from the U.S. FDA include Hanmi Pharmaceutical, GC Green Cross Cell, Shinpoong Pharmaceutical, Alteogen, Corestem, and iNtRON Bio.
The number of domestic medicines that receives ODD has been continuing to grow in recent years. This year, a total of 10 domestically developed new drug candidates have been granted ODD. The figure was one in 2014, two in 2015, three in 2016 and six last year.
The FDA's ODD grant is designed to support active development and approval of therapeutic drugs for rare diseases or life-threatening diseases. Therapeutic drugs that received ODD are granted a variety of benefits such as tax cuts, exemption of new drug approval examination fees, and recognition of monopoly rights for seven years after approval of marketing authorization.
For Hanmi Pharmaceutical, this is the third time that its new drug candidate material has received ODD. The company’s “LAPS Glucagon Analog,” which is developed as a congenital hyperinsulinemia treatment, “Oraxol,” an oral anticancer drug, and the new drug candidate material (HM43239) were granted ODD in February, April, and this month, respectively.
In particular, HM43239 is a drug candidate material for treatment of acute myelogenous leukemia (AML). Currently, it has received an approval for Phase I clinical trial from the FDA and will begin the trial at MD Anderson Cancer Center, USA.
GC Green Cross Cell’s immunotherapy drug “Immune cell LC” was also given an ODD by the FDA in September as a pancreatic cancer treatment. The company has received ODD for liver cancer and brain tumor (glioblastoma) in June and August, accordingly. This gives GC Green Cross cells the opportunity to enter the US liver cancer, brain tumor, and pancreatic cancer drug markets.
Shinpoong Pharmaceutical also received an ODD for the anti-malarial drug “Pyramax.”
The achievements of domestic biotech companies are also remarkable. In June, Corestem’s “Neuronata R,” a stem cell therapy for amyotrophic lateral sclerosis was approved as ODD by FDA, while in August, Alteogen’s “ALT-P7,” a biobetter of breast canacer and stomach cancer treatment “Herceptin,” received an approval. Prior to this, in June, “BAL200,” an Anthrax infection treatment developed by Intron Bio, was granted an ODD.
According to market analyst firm EvaluatePharma, the global market for ODDs, which reached 11 trillion won (US$10 billion) in 2015, will grow at a CAGR of 11.1%, reaching 23 trillion won (US$20.9 billion) in 2022. According to the “Bio in Global Global Trend Report” published by the Biotech Policy Research Center, the number of ODDs granted by the FDA was 75 cases in 2004, which is about three times more than that in the previous year (27 cases).