AstraZeneca plc (ADR) (NYSE:AZN) and its R&D arm, MedImmune, disclosed that its investigational anti-CD19 monoclonal antibody, MEDI-551, was given Orphan Drug Status by the FDA. The company is developing the drug for the treatment of neuromyelitis optica and neuromyelitis optica spectrum disorders or NMOSD. The drug is currently in Phase IIb clinical development.
Life-Threatening Autoimmune Disease
AstraZeneca said that NMO is a rare and life-threatening autoimmune disease. It affects the central nervous system, attacking the healthy nerve cells of patients. The pharmaceutical company indicated that it was most commonly seen in optic nerves and the spinal cord, resulting in severe damage. The British firm said that NMO also causes severe muscle weaknesses, paralysis, respiratory failure, vision loss, neuropathic pain and causes functional issues with the bowel and bladder.
The disease affects approximately 5 in 100,000 people. However, currently, there is no approved medicine for treating NMO. AstraZeneca revealed that its research has suggested that NMO and NMOSD causes antibodies to develop against aquaporin-4 protein. The company believes that such antibodies play an important role in the pathogenesis of NMO disease. Its MEDI-551 was focused mainly on depleting the cells producing these antibodies.
The company’s Vice President for R&D Bing Yao said that the FDA move underscored the considerable need for an effective drug for NMO sufferers being such a devastating disease. He said that the disease can cause increased damage and disability with each successive attack. The drug maker said that its orphan drug status was a unique one targeting mechanism of action in providing the possible treatment for NMO.
The AstraZeneca official said that he was looking forward to working with regulators to advance its orphan drug status medicine to patients affected by NMO as soon as possible. In the United States, there are about 200,000 people affected by the disease.