Acquired hemolytic anemia

European Hematology Association - Pegcetacoplan Maintains a Durable Response in Patients with Paroxysmal Nocturnal Hemoglobinuria Through Week 48

Retrieved on: 
Saturday, June 12, 2021
Medical specialties, Clinical medicine, Acquired hemolytic anemia, Complement system, Orphan drugs, Organ systems, Paroxysmal nocturnal hemoglobinuria, Eculizumab, Pegcetacoplan, Hemoglobinuria

After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.

Key Points: 
  • After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.
  • Here, we show the efficacy and safety of pegcetacoplan in 77 patients through 48 weeks of treatment.
  • Improvement of hemoglobin (Hb) level was maintained through Week 48 in all patients treated with pegcetacoplan monotherapy.
  • In conclusion, patients with suboptimal response to eculizumab experienced durable treatment effect when switched to pegcetacoplan, suggesting that pegcetacoplan is an effective therapeutic option for patients with paroxysmal nocturnal hemoglobinuria.

European Hematology Association - Pegcetacoplan Maintains a Durable Response in Patients with Paroxysmal Nocturnal Hemoglobinuria Through Week 48

Retrieved on: 
Saturday, June 12, 2021
Medical specialties, Clinical medicine, Acquired hemolytic anemia, Complement system, Orphan drugs, Organ systems, Paroxysmal nocturnal hemoglobinuria, Eculizumab, Pegcetacoplan, Hemoglobinuria

After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.

Key Points: 
  • After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment.
  • Here, we show the efficacy and safety of pegcetacoplan in 77 patients through 48 weeks of treatment.
  • Improvement of hemoglobin (Hb) level was maintained through Week 48 in all patients treated with pegcetacoplan monotherapy.
  • In conclusion, patients with suboptimal response to eculizumab experienced durable treatment effect when switched to pegcetacoplan, suggesting that pegcetacoplan is an effective therapeutic option for patients with paroxysmal nocturnal hemoglobinuria.

Apellis and Sobi Report Positive Top-line Results from the Phase 3 PRINCE Study of EMPAVELI™ (pegcetacoplan) in Treatment-naïve Patients with PNH

Retrieved on: 
Tuesday, May 25, 2021
Complement system, Organ systems, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, Pegcetacoplan, PNH, Medical specialties, Clinical medicine, Paroxysmal attack, Hemoglobinuria, Ham test

Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment.

Key Points: 
  • Combined with previous studies, these results emphasize the potential of EMPAVELI to provide disease control for all adults with PNH regardless of prior treatment.
  • At Week 26, 9% of patients in the EMPAVELI group experienced a serious adverse event (SAE) compared to 17% on standard of care.
  • The PRINCE study results reinforce the efficacy and safety profile of EMPAVELI in PNH, said Ravi Rao, head of research and development and chief medical officer at Sobi.
  • The PRINCE study (NCT04085601) is a 2:1 (EMPAVELI: standard of care) randomized, multi-center, open-label, controlled Phase 3 study in 53 treatment-nave adults with paroxysmal nocturnal hemoglobinuria (PNH).

Omeros Announces Extension of FDA Review Period for Narsoplimab in HSCT-TMA

Retrieved on: 
Thursday, May 20, 2021
Research, Managed care, Pharmaceutical, Oncology, Genetics, Clinical trials, Science, Biotechnology, Stem cells, Practice Management, FDA, Health, Medical specialties, Clinical medicine, Medicine, Food and Drug Administration, Acquired hemolytic anemia, Blood disorders, Complement system, Lectin pathway, Hematology, Thrombotic microangiopathy, Prescription Drug User Fee Act, Atypical hemolytic uremic syndrome

The new Prescription Drug User Fee Act (PDUFA) target action date is October 17, 2021.\nAs part of the ongoing BLA Priority Review, Omeros recently submitted a response to an FDA information request.

Key Points: 
  • The new Prescription Drug User Fee Act (PDUFA) target action date is October 17, 2021.\nAs part of the ongoing BLA Priority Review, Omeros recently submitted a response to an FDA information request.
  • FDA has classified the response as a major amendment, which requires additional time to review.\n\xe2\x80\x9cWe\xe2\x80\x99re pleased with our ongoing interactions with FDA on the narsoplimab BLA,\xe2\x80\x9d said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros.
  • Omeros\xe2\x80\x99 lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application under priority review by FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy.
  • Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19.

Homology Medicines Announces Presentations on its Expanding Genetic Medicines Platform and Internal GMP Manufacturing at the American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Thursday, May 13, 2021
Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, PNH, Paroxysmal attack, Hemoglobinuria, Clinical medicine, Medicine, Medical specialties

Among Homology\xe2\x80\x99s presentations are data from its new GTx-mAb platform targeting complement protein 5 (C5), which showed proof of principle in paroxysmal nocturnal hemoglobinuria (PNH).

Key Points: 
  • Among Homology\xe2\x80\x99s presentations are data from its new GTx-mAb platform targeting complement protein 5 (C5), which showed proof of principle in paroxysmal nocturnal hemoglobinuria (PNH).
  • Presentations also focused on the Company\xe2\x80\x99s internal commercial GMP manufacturing process and platform.
  • \xe2\x80\x9cThese data demonstrated once again that our vectors are highly efficient in targeting the liver.
  • We are on track to name our first development candidate from the GTx-mAb platform in PNH this year.\xe2\x80\x9d\nDr.

Alexion Announces Upcoming Data Presentations at the 26th European Hematology Association Virtual Congress

Retrieved on: 
Wednesday, May 12, 2021
Health, Other Science, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Orphan drugs, Life sciences, Rare diseases, Health sciences, Health care, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, Alexion Pharmaceuticals, Ravulizumab, PNH, Paroxysmal attack, Hemoglobinuria, Paroxysmal Nocturnal Hemoglobinuria (PNH), ULTOMIRIS®, SOLIRIS®, Alexion, PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH), ULTOMIRIS®, SOLIRIS®, ALEXION

b"Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that five abstracts have been accepted for presentation at the 26th annual European Hematology Association (EHA) 2021 virtual Congress, taking place from June 9 through June 17, 2021.

Key Points: 
  • b"Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that five abstracts have been accepted for presentation at the 26th annual European Hematology Association (EHA) 2021 virtual Congress, taking place from June 9 through June 17, 2021.
  • PNH occurs when the complement system\xe2\x80\x94a part of the body\xe2\x80\x99s immune system\xe2\x80\x94over-responds, leading the body to attack its own red blood cells.
  • The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body\xe2\x80\x99s immune system.
  • ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Aplastic Anemia and MDS International Foundation and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
Thursday, May 6, 2021
Blood, Medical specialties, Hematopathology, Clinical medicine, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, Hematology, Aplastic anemia, Bone marrow failure, PNH, Paroxysmal attack, Anemia

and WASHINGTON, May 6, 2021 /PRNewswire/ -- The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD) today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare bone marrow failure disease.

Key Points: 
  • and WASHINGTON, May 6, 2021 /PRNewswire/ -- The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD) today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare bone marrow failure disease.
  • PNH is characterized by the destruction of red blood cells, blood clots and impaired bone marrow function.
  • "\nThe Global PNH Patient Registryis a natural history study that consists of electronic surveys to collect information about the patient experience and disease progression.
  • We are so pleased to welcome the Aplastic Anemia and MDS International Foundation to our IAMRARE Registry Community!

Omeros Corporation Announces Approval of ICD-10 Codes for HSCT-TMA Diagnosis and for Narsoplimab Administration

Retrieved on: 
Tuesday, May 4, 2021
FDA, Health, Stem cells, Clinical trials, Pharmaceutical, Biotechnology, Medical specialties, Clinical medicine, Medicine, Blood disorders, Rare diseases, Acquired hemolytic anemia, ICD-10, International Classification of Diseases, Diagnosis code, Hemolysis, Atypical hemolytic uremic syndrome, Hemolytic–uremic syndrome, Narsoplimab, Omeros Corporation, NARSOPLIMAB, OMEROS CORPORATION

The absence of a specific HSCT-TMA diagnosis code has led to underreporting of diagnoses and inaccurate coding, which has complicated reporting of the incidence and outcomes of the disease.

Key Points: 
  • The absence of a specific HSCT-TMA diagnosis code has led to underreporting of diagnoses and inaccurate coding, which has complicated reporting of the incidence and outcomes of the disease.
  • ICD-10-PCS codes are used by billers to identify procedures and services performed during an inpatient stay.
  • The new codes will be essential to facilitating billing and reimbursement and for tracking utilization, if narsoplimab is approved by the U.S. Food and Drug Administration (FDA).
  • Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19.

Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast to 2030: Two Roads Diverge - Treatment Choice in Coexisting Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria - ResearchAndMarkets.com

Retrieved on: 
Thursday, April 15, 2021
Biotechnology, Pharmaceutical, Health, Acquired hemolytic anemia, Paroxysmal nocturnal hemoglobinuria, PNH, Paroxysmal attack, Clinical medicine, Medicine, Hemoglobinuria, Medical specialties, Ham test

b'The "Paroxysmal Nocturnal Hemoglobinuria - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com\'s offering.\nThis \'Paroxysmal nocturnal hemoglobinuria (PNH) - Epidemiology Forecast - 2030\' report delivers an in-depth understanding of the historical and forecasted epidemiology of PNH in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.\nThe PNH epidemiology division provides insights about the historical and current patient pool, along with the forecasted trend for every seven major countries.

Key Points: 
  • b'The "Paroxysmal Nocturnal Hemoglobinuria - Epidemiology Forecast to 2030" report has been added to ResearchAndMarkets.com\'s offering.\nThis \'Paroxysmal nocturnal hemoglobinuria (PNH) - Epidemiology Forecast - 2030\' report delivers an in-depth understanding of the historical and forecasted epidemiology of PNH in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.\nThe PNH epidemiology division provides insights about the historical and current patient pool, along with the forecasted trend for every seven major countries.
  • It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders.
  • This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.\nThe disease epidemiology covered in the report provides historical and forecasted PNH epidemiology segmented as the Total diagnosed prevalent cases of Paroxysmal Nocturnal Hemoglobinuria and Gender-specific Cases of PNH.
  • The report includes the prevalent scenario of PNH in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.\n'

Glancy Prongay & Murray LLP Reminds Investors of Looming Deadline in the Class Action Lawsuit Against Immunovant, Inc. f/k/a Health Sciences Acquisitions Corporation (IMVT)

Retrieved on: 
Friday, April 9, 2021
Blood, Medical specialties, Acquired hemolytic anemia, Autoimmune diseases, Hematopathology, Clinical pathology, Hematology, Transfusion medicine, Warm antibody autoimmune hemolytic anemia, Autoimmune hemolytic anemia, AIHA, Hemolytic anemia

You can also contact Charles H. Linehan, of GPM at 310-201-9150, Toll-Free at 888-773-9224, or via email at [email protected] to learn more about your rights.

Key Points: 
  • You can also contact Charles H. Linehan, of GPM at 310-201-9150, Toll-Free at 888-773-9224, or via email at [email protected] to learn more about your rights.
  • On September 29, 2019, HSAC entered into an agreement with Immunovant Sciences Ltd. (Legacy Immunovant) to effect a merger between the two entities (the Merger).
  • The Company has also completed initiation of Phase II clinical trials of IMVT-1401 for the treatment of warm autoimmune hemolytic anemia (WAIHA).
  • Glancy Prongay & Murray LLP, Los Angeles