Reistone Biopharma Co., Ltd, a company incubated by Hengrui Medicine with an investment of USD 100 million in 2018, has announced the receipt of orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its Bruton’s tyrosine kinase (BTK) inhibitor, edralbrutinib. The designation is awarded for the drug’s potential use in treating neuromyelitis optica spectrum disorders (NMOSD).
Understanding NMOSD and Its Clinical Impact
NMOSD is a rare chronic autoimmune and inflammatory demyelinating disease of the central nervous system, primarily affecting the optic nerve and spinal cord. Clinical features include recurrent optic neuritis and long segment transverse myelitis, which can occur simultaneously or separately. NMOSD is characterized by high recurrence rates and disabling symptoms, with most patients experiencing severe visual impairment, limb dysfunction, and urinary and fecal disorders. The disease was included in China’s first rare disease list of 121 kinds in May 2018. Commercially available NMOSD therapies in China include Hansoh/Viela’s Uplizna (inebilizumab) and Roche’s Enspryng (satralizumab).
Edralbrutinib’s Mechanism of Action and Phase II Study Progress
Edralbrutinib, an oral BTK inhibitor co-developed by Hengrui Medicine, selectively inhibits the activation, proliferation, and survival of B cells and can also act on other immune cells such as macrophages. This mechanism affects the autoimmune inflammatory processes mediated by B cells and other immune cells, which are key pathological processes in NMOSD. The Phase II study for edralbrutinib is currently at the data analysis stage, indicating ongoing progress towards potential regulatory submissions and commercialization.-Fineline Info & Tech
