Domestic pharmaceutical companies are targeting the U.S. market with rare diseases.
In 2017, six locally developed drugs received Orphan Drug Designation (ODD) by the FDA, while two more drugs – Oraxol and HM15136 -- received orphan drug designation this year.
Hanmi Pharmaceutical’s Oraxol, a novel oral formulation commonly used during chemotherapy treatment, received ODD status for angiosarcoma on April 20.
The drug is an anticancer treatment that transformed injectable anticancer medicines into oral medications. Hanmi Pharmaceutical had licensed out the cure to Athenex, a U.S. biopharmaceutical company, in 2011. The company had also received ODD status for HM15136, a congenital hyperinsulinemia drug, in February.
Hanmi plans to enter phase 1 clinical trials for the HM15136, a drug that used Hanmi’s Lapscovery technology, in the first half of this year. It is also developing Poziotinib, a drug candidate technology transferred to Spectrum, a U.S. biopharmaceutical firm, to treat non-small cell lung cancer, which is a rare disease.
Youngjin Pharmaceutical also received ODD status from the FDA recently for KL1333, a drug the company recently transferred technology to Neurovive, a Swedish pharmaceutical company.
Youngjin recently transferred exclusive rights for KL1333, a treatment for a mitochondrial genetic disease, to Neurovive except for Korea and Japan.
Medpacto, a subsidiary of Theregen ETEX, is undergoing phase 2 clinical trials for TEW-7197, an anticancer treatment with indications for myelodysplastic syndrome and liver cancer., after receiving ODD status.
Viromed has also received approval to conduct phase 2 clinical trials for VM202-ALS, a treatment for amyotrophic lateral sclerosis (Lou Gehrig's disease). Approximately 30,000 patients are suffering from the disease in America.
Winning an ODD status in the U.S. is noteworthy as it is also related to early market entry of a product.
The U.S. government is implementing the Orphan Drug Act and has expedited screening programs to encourage the development of rare disease treatments.
The ODD status provides specific benefits, including a seven-year period of market exclusivity after approval, as well as a tax reduction. Also, if the program receives ODD status, it minimizes the approval process for the drug and opens up possibilities for a huge licensing out agreement or even a merger and acquisition.
“The U.S. gives ODD status much quicker than Korea. If a company manages to receive ODD status it has a high chance of also obtaining an orphan drug status in Korea,” a domestic pharmaceutical official said. “Also, having a new drug means that a company can get a big contract before long.”
Korean companies have stepped up efforts to receive ODD status over the years. FDA's designation of rare pharmaceuticals for domestic pharmaceutical companies was only one in 2006 but has since steadily increased to two in 2012, three in 2013, and six in 2017.