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ALLMedicine™ Paroxysmal Nocturnal Hemoglobinuria Center - News


Soliris® is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. Soliris® works by inhibiting terminal complement, a part of the immune system that, when activated in an uncontrolled manner, plays a role in serious diseases like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (...

Registry: Blood Disorder Drug Safe for Pregnant Women

Sep 10th, 2015 - Action Points Note that this observational study of women with paroxysmal nocturnal hemoglobinuria treated with eculizumab found generally good pregnancy outcomes. While this was not a trial, a randomized study of this therapy would likely be deemed unethical. Women treated with eculizumab (Soliris) for the rare stem cell disorder paroxysmal nocturnal hemoglobinuria (PNH) experienced minimal ma...

FDA OKs New Options for Two Rare Blood Diseases

Dec 21st, 2018 - WASHINGTON -- The FDA announced new approvals for two rare and life-threatening blood diseases on Friday, as blastic plasmacytoid dendritic cell neoplasm (BPDCN) gets its first approved agent and paroxysmal nocturnal hemoglobinuria (PNH) gets a new drug that drastically cuts the frequency of treatments. BPDCN Gets First Approved Drug The FDA approved the infusion therapy tagraxofusp (Elzonris) ...

Rare Blood Disorder Tx Succeeds in Trial (Reuters)

Mar 15th, 2018 - Reuters reported that Alexion's investigational therapy for a rare and potentially fatal blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), showed non-inferiority to the current treatment. The new treatment -- ALXN1210 -- met the study's primary endpoint of reducing high levels of cellular damage in blood and reduced the need for blood transfusions. Unlike eculizumab (Soliris), Alexion'...

Meningitis Vaccine No Protection in Rare Diseases

Jul 7th, 2017 - Healthcare providers should have a high index of suspicion for meningitis if they are treating patients for two rare blood diseases, even if the patients have been vaccinated, the CDC is warning. The terminal complement inhibitor eculizumab (Soliris) is used to treat patients with two rare, life-threatening illnesses -- paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome ...

C3 inhibitor shows potential in PNH and AIHA
Will Pass

Apr 18th, 2019 - GLASGOW – APL-2, a complement factor 3 (C3) inhibitor, may be a future treatment option for paroxysmal nocturnal hemoglobinuria (PNH) and autoimmune hemolytic anemia (AIHA), according to investigators from two separate studies. Will Pass/MDedge News Dr.

The evolving landscape of complement inhibition therapy
I. Romina Sosa, MD, PhD

Jan 21st, 2020 - The introduction of eculizumab, a monoclonal antibody targeting C5 of the complement cascade, revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by complement-mediated intravascular hemolysis, bone marrow failure, and thrombophilia. Treatment options for PNH were limited before eculizumab was approved by the Food and Drug Adminis.

NORD Publishes Physician Guides to PNH and aHUS

Jan 8th, 2016 - Two new physician guides have been published in NORD’s free online series for physicians and other medical professionals on the NORD Physician Guides website. The new guides are the NORD Physician Guide to Paroxysmal Nocturnal Hemoglobinuria (PNH) and the NORD Physician Guide to Atypical Hemolytic Uremic Syndrome (aHUS).

Aplastic Anemia & MDS International Foundation Research Portal Is Open

Feb 17th, 2017 - AAMDSIF has announced that its 2017 research grant portal is now open. Each year, the foundation’s medical advisors review research proposals from new and established investigators in the topic areas of aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria.

FDA approves ravulizumab for PNH
HT Staff

Dec 25th, 2018 - Photo from Business Wire Ravulizumab (Ultomiris) The U. S.

Drug is convenient alternative for PNH, doc says
Jen Smith

Jun 22nd, 2018 - Photo from EHA Session at the 23rd Congress of the European Hematology Association (EHA) STOCKHOLM—Results of a phase 3 study suggest the long-acting C5 complement inhibitor ravulizumab produces similar results as eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH). Treatment with ravulizumab every 8 weeks proved noninferior to treatment with eculizumab every 2 weeks for the c.

FDA grants priority review to drug for PNH
HT Staff

Aug 22nd, 2018 - Red blood cells The US Food and Drug Administration (FDA) has accepted for priority review the biologics license application (BLA) for ALXN1210, a long-acting C5 complement inhibitor. With this BLA, Alexion Pharmaceuticals, Inc.

Drug granted orphan status for PNH in EU
HT Staff

Aug 29th, 2014 - Red blood cells A novel compound has received orphan status in the Europe Union to treat paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening disease that causes severe anemia and confers a high risk of thrombosis. The compound, AMY-101, works by inhibiting C3, a central component of the complement immune system.

Drug granted fast track designation for PNH
HT Staff

Dec 22nd, 2016 - Red blood cells The US Food and Drug Administration (FDA) has granted fast track designation for the complement C3 inhibitor APL-2. The designation applies to APL-2 in the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) who continue to experience hemolysis and require red blood cell transfusions despite receiving therapy with eculizumab.

FDA grants drug orphan designation for PNH
HT Staff

Sep 13th, 2016 - Red blood cells The US Food and Drug Administration (FDA) has granted orphan drug designation to Coversin as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). Coversin is a recombinant small protein (16,740 Da) derived from a native protein found in the saliva of the Ornithodoros moubata tick.

Upcoming regional education meetings

Apr 8th, 2015 - In its ongoing series of regional educational meetings for patients and caregivers, NORD will be hosting the following events: May 1-2, Orlando, short bowel syndrome; May 14-16, Denver, short bowel syndrome and hypoparathyroidism; June 26-27, Teaneck, NJ, paroxysmal nocturnal hemoglobinuria (PNH); and July 24-25, Mobile, AL, PNH. For information or to register, visit the NORD website (www.

EMA recommends drug receive orphan designation for PNH
HT Staff

May 11th, 2017 - Red blood cells The European Medicines Agency (EMA) has recommended orphan drug designation for the complement C3 inhibitor APL-2 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, blocking all 3 pathways of complement activation (classical, lectin, and alternative).

EC grants drug orphan designation for PNH
HT Staff

Nov 26th, 2016 - Red blood cells The European Commission (EC) has granted orphan drug designation to RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). RA101495 is a synthetic macrocyclic peptide inhibitor of complement component C5.

COMP recommends orphan status for drug to treat PNH
HT Staff

Jul 13th, 2016 - Red blood cells The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending orphan designation for Coversin for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Coversin is a second-generation complement inhibitor that acts on complement component-C5, preventing release of C5a and formation of C5b-9 (also known as the memb.

FDA approves ravulizumab for treatment of paroxysmal nocturnal hemoglobinuria
Hematology News; Lucas Franki

Dec 21st, 2018 - The Food and Drug Administration has approved ravulizumab (Ultomiris) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Wikimedia Commons/FitzColinGerald/Creative Commons License “The approval of Ultomiris will change the way that patients with PNH are treated.