The FDA Approves Two Orphan Blood Disorder Drugs

The U.S.Food and Drug Administration (FDA) approved two drugs for separate blood disorders, Alexion Pharmaceuticals’ Ultomiris for the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), and Stemline Therapeutics’ Elzonris for blastic plasmacytoid dendritic cell neoplasm (BPDCN). For Alexion, the FDA approval was two months ahead of its February PDUFA date, although share prices of company stock do not reflect the good news, as they have been caught up in the overall market negativity stemming from the government shutdown and other factors. Ultomiris, a long-acting C5 complement inhibitor, is considered the successor to the company’s blockbuster PNH drug Soliris.
PNH is a rare blood disease where the red blood cells are destroyed by a part of the body’s immune system known as the complement system. Ultomiris is an improved version of Soliris. Trial results show that patients could safely be dosed once every eight weeks with Ultomiris (formerly known as ALXN1210) compared to every two weeks with Soliris – a key point that the FDA honed in on for approval. Richard Pazdur, head of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products, said the greater length of time between doses for patients on Ultomiris will benefit the quality of life in patients. Dosing every two weeks can be burdensome for patients and their families, he said.