Chinese treatment granted 'orphan drug' designation by US FDA

Chinese biotech company Junshi Biosciences announced on Friday that the company's domestically developed drug Toripalimab (anti-PD-1 mAb), in combination with Axitinib for treatment of mucosal melanoma, has been granted orphan drug designation by the United States Food and Drug Administration.

Mucosal melanoma is one type of melanoma. Mucosal melanoma is not responsive to traditional chemotherapy and responds poorly to immunosuppressive monotherapy. Preclinical studies have shown immunity combined with the treatment of Vascular endothelial growth factor receptor can generate a synergistic effect on inhibiting the growth of tumors.

In late 2018, Toripalimab nabbed China's first approval for a homegrown anti-PD-1 treatment, an emerging cancer therapy that boosts the immune system to help the body target and kill tumors.

The use of Toripalimab in combination with Axitinib to treat patients with advanced mucosal melanoma who have not received chemotherapy can achieve an objective response rate of 48.3 percent and a disease control rate of 86.2 percent. The median progression-free survival time was 7.5 months, according to a study conducted by a group of scientists from Peking University Cancer Hospital. The study results were published in the Journal of Clinical Oncology on August 12, 2019.

The orphan drug designation granted by the FDA is beneficial for the continuous development of the combination therapy and the enjoyment of certain policy support in terms of registration and commercialization in the US, including but not limited to tax credits for clinical trial costs, application fee waivers for new drugs and 7 years of market exclusivity without being affected by patent.

The designation will reduce R&D investment of the Combination Therapy to a certain extent and accelerate the progress of clinical trials and market registration, the company said.