On December 21, 2018, the United States Food and Drug Administration (USFDA) approved two new treatments Elzonris (tagraxofusp-erzs)32 infusion and Ultomiris (ravulizumab)33 injection for the treatment of rare blood diseases:

1. Elzonris (tagraxofusp-erzs) - for treatment of BPDCN

USFDA approved Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, two years of age and older. The FDA granted the approval of Elzonris to Stemline Therapeutics. The FDA also granted Breakthrough Therapy designation, Orphan Drug designation and Priority Review designation to Elzonris.

The efficacy of Elzonris was studied in two cohorts of patients in a single-arm clinical trial. The first trial cohort enrolled 13 patients with untreated BPDCN, and seven patients (54%) achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). The second cohort included 15 patients with relapsed or refractory BPDCN. One patient achieved CR and one patient achieved CRc.


BPDCN is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin. It often presents as leukemia or evolves into acute leukemia. The disease is more common in men than women and in patients 60 years and older.


ELZONRIS, a CD123-directed cytotoxin, was granted full approval by the FDA for the treatment of adult and pediatric patients, two years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN), in treatmentnaive and previously-treated settings. In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment for the upcoming marketing authorization application (MAA) submission, which is expected in the first quarter of 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF) and other CD123 positive diseases34.

2. Ultomiris (ravulizumab) - for treatment of PNH

The U.S. Food and Drug Administration approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease. The FDA granted the approval of Ultomiris to Alexion Pharmaceuticals. The FDA also granted Priority Review designation and Orphan Drug designation to Ultomiris.

Ultomiris is a long-acting complement inhibitor that prevents hemolysis. The efficacy of Ultomiris was studied in a clinical trial of 246 patients who previously had not been treated for PNH (treatment naive), who were randomized to be treated with Ultomiris or eculizumab, the current standard of care for PNH. The results of the trial demonstrated that Ultomiris had similar results to eculizumab (non-inferior) – patients did not receive a transfusion and had similar incidence of hemolysis measured by the normalization of LDH levels in patients' blood (lactate dehydrogenase, or LDH, is an enzyme required during the process of turning sugar into energy in the body's cells). In addition, Ultomiris was studied in a second clinical trial of 195 patients with PNH who were clinically stable after having been treated with eculizumab for at least the past six months. These patients were randomly selected to be treated with Ultomiris or to continue eculizumab. Ultomiris again demonstrated similar effects to eculizumab (non-inferior) based on several clinical measures including hemolysis and avoiding transfusion.

About PNH

PNH is a rare acquired disorder that leads to the rupture or destruction of red blood cells (hemolysis). Patients with PNH are missing a certain protein that normally protects red blood cells from being destroyed by the patient's immune system. Patients with PNH have sudden, recurring episodes where red blood cells are prematurely destroyed which may be triggered by stresses on the body, such as infections or physical exertion. During these episodes, the following symptoms may occur - severe anemia, profound fatigue, shortness of breath, and intermittent episodes of dark colored urine, kidney disease or recurrent pain. PNH can occur at any age, although it is most often diagnosed in young adulthood.


ULTOMIRIS" (ravulizumab-cwvz) is the first and only long-acting C5 inhibitor administered every eight weeks that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). ULTOMIRIS is approved in the U.S. as a treatment for adults with PNH. Regulatory authorities in the European Union (EU) and Japan have accepted and are reviewing applications for the approval of ULTOMIRIS as a treatment for adults with PNH35.


32 https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629020.htm

33 https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629022.htm

34 https://ir.stemline.com/news-releases/news-release-details/fda-approves-elzonristm-tagraxofusp-first-treatment-blastic

35 https://news.alexion.com/press-release/product-news/alexion-receives-early-fda-approval-ultomiris-ravulizumab-cwvz-adults-par

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