Paroxysmal Nocturnal Hemoglobinuria News and Research

RSS

Insilico Medicine's new AI tool shows promise in predicting clinical trial outcomes

Insilico Medicine ("Insilico"), a clinical-stage end-to-end generative artificial intelligence (AI) drug discovery company, has demonstrated that it can predict the outcome of Phase II to Phase III clinical trial success using its proprietary transformer-based AI clinical trial prediction tool called inClinico with a high degree of accuracy.

3 Aug 2023

AAMDSIF Patient and Family Conference to be held in Memphis

The University of Tennessee Health Science Center is partnering with the Aplastic Anemia & MDS International Foundation (AAMDSIF), along with St. Jude Children's Research Hospital, Regional One Health, and Baptist Memorial Hospital, to hold the AAMDSIF Patient and Family Conference for the first time in Memphis on July 23.

22 Jul 2022

FDA approves first drug for treatment of neuromyelitis optica spectrum disorder

The U.S. Food and Drug Administration today approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder in adult patients who are anti-aquaporin-4 antibody positive.

27 Jun 2019

Drug reduces risk of relapse with neuromyelitis optica spectrum disorder, study shows

The drug eculizumab, a synthetic antibody that inhibits the inflammatory response, significantly reduced the risk of relapse with neuromyelitis optica spectrum disorder.

3 May 2019

New treatment for adults with rare, life-threatening blood disease receives FDA approval

The U.S. Food and Drug Administration today approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.

21 Dec 2018

Alexion initiates new global Uncommon Strength campaign to raise awareness of rare diseases

Alexion Pharmaceuticals, Inc. today announced the launch of Uncommon Strength, a global campaign to raise awareness of rare diseases through the celebration of the extraordinary resilience and inner strength of those impacted by these diseases.

15 Sep 2016

Scientists use X-rays to study how eculizumab prevents immune system from damaging kidney tissue

In collaboration with Alexion Pharmaceuticals, Inc., scientists from Aarhus University have used X-rays to understand how the therapeutic antibody eculizumab prevents our immune system from destroying red blood cells and damaging kidney tissue.

6 Jun 2016

Apellis Pharmaceuticals completes $47.1M Series D preferred stock financing

Apellis Pharmaceuticals, Inc., today announced that it has completed a $47.1 million Series D preferred stock financing, co-led by new investors Cormorant Asset Management, Hillhouse Capital Group and venBio Global Strategic Fund, joining existing investors Morningside Venture Investments, AJU IB Investment, and Epidarex Capital.

12 Feb 2016

Pabetalone (RVX-208) to be tested in proof-of-concept pilot study in complement mediated diseases

Resverlogix Corp. announced today the commencement of an Orphan Disease Program specific for Complement Mediated Diseases. New data generated by Resverlogix has demonstrated that BET inhibition by apabetalone (RVX-208) has effects on multiple biological pathways that underlie disease pathology.

24 Sep 2015

Apellis Pharmaceuticals completes $33M private placement of Series C Preferred Stock

Apellis Pharmaceuticals, Inc., a leading biotechnology company applying immunotherapy to autoimmune disease, today announced that it has completed a $33M private placement of its Series C Preferred Stock.

2 Dec 2014

Penn Medicine-developed drug gets FDA orphan status for PNH treatment

A Penn Medicine-developed drug has received orphan status from the Food and Drug Administration (FDA) this month for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.

28 Oct 2014

Penn receives orphan status in Europe for treatment of PNH

A Penn Medicine-developed drug has received orphan status in Europe this week for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.

27 Aug 2014

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation.

17 Jun 2014

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases.

10 Jun 2014

Alexion Pharmaceuticals obtains orphan drug designation from EC for Soliris

Alexion Pharmaceuticals, Inc. today announced that the European Commission has granted an orphan drug designation (ODD) to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of graft rejection following solid organ transplantation. Graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.

23 Apr 2014

Penn research team discovers new cost-effective way to fight rare blood disorder

A University of Pennsylvania research team has defined a possible new way to fight a disease that is currently treatable only with the most expensive drug available for sale in the United States.

28 Feb 2014

Penn researchers identify possible new way to fight rare blood disorder

A University of Pennsylvania research team has defined a possible new way to fight a disease that is currently treatable only with the most expensive drug available for sale in the United States. In a study published this month in Blood, the Penn team describes the strategy, based on the oldest part of the human immune system - called "complement" -- that could turn out to be less costly and more effective for the majority of patients with a rare blood disorder.

27 Feb 2014

Alexion Pharmaceuticals reports full year 2013 GAAP and non-GAAP financial results

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter and year ended December 31, 2013. For the three months ended December 31, 2013, Alexion Pharmaceuticals, Inc. ("Alexion" or the "Company") reported net product sales of Soliris® (eculizumab) of $441.9 million, compared to $320.5 million for the same period in 2012.

30 Jan 2014

Alexion's Soliris gets FDA orphan drug designation for DGF prevention in renal transplant patients

Alexion Pharmaceuticals today announced that the U.S. Food and Drug Administration has granted an orphan drug designation to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function in renal transplant patients.

21 Jan 2014

Viropharma announces financial results for third quarter 2013

ViroPharma Incorporated reported today its financial results for the third quarter ended September 30, 2013. Net sales were $113 million for the third quarter ended September 30, 2013, as compared to $91 million in the same three months of 2012.

31 Oct 2013