Pegcetacoplan

Sobi Receives Approval from Health Canada for Empaveli™ (pegcetacoplan) for the Treatment of Certain Patients with Paroxysmal Nocturnal Hemoglobinuria

Retrieved on: 
Friday, December 9, 2022
Eculizumab, Thrombosis, Neurology, Breathe Me, Quality of life, Incidence, Toronto General Hospital, Blood, Hospital, Fatigue, Safety, Erectile dysfunction, McKinley, The New England Journal of Medicine, Swedish Orphan Biovitrum, PEGASUS, Nephrology, Pain, PNH, Geographic atrophy, Paroxysmal nocturnal hemoglobinuria, SEK, Hemolysis, The Amazing Race Canada 4, Degenerative disease, United Arab Emirates, Hematology, Nasdaq Stockholm, Anemia, Health Canada, Pharmaceutical industry, Sales, Patient, Pegcetacoplan, EU

PNH is characterized by the destruction of red blood cells (hemolysis) and release of free hemoglobin that can cause thrombosis and require frequent transfusions.

Key Points: 
  • PNH is characterized by the destruction of red blood cells (hemolysis) and release of free hemoglobin that can cause thrombosis and require frequent transfusions.
  • Empaveli is approved in the United States and the United Arab Emirates for the treatment of adults with PNH.
  • In Australia, Empaveli is approved for the treatment of adult patients with PNH who have an inadequate response to, or are intolerant of, a C5 inhibitor.
  • McKinley C. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome.

NovelMed's Complement Alternative Pathway Specific Anti-Bb Antibody (NM8074) for Rare Diseases Achieves a Major Milestone

Retrieved on: 
Monday, January 31, 2022
AP, MAC, IND, Safety, Lists of diseases, Patient, Therapy, Pathology, Food, Infection, Conditional sentence, Hemolysis, BB, LDH, Thrombus, Inflammation, Properdin, CP, Pharmacokinetics, Serum, Disease, Complement component 5, C5a, Alternative-complement-pathway C3/C5 convertase, PNH, Paroxysmal nocturnal hemoglobinuria, C3b, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FDA, C3a, Pharmaceutical industry, Vaccine, C5, C3, Pegcetacoplan, Eculizumab, Ravulizumab

Through its targeted specificity to the complement alternative pathway (AP), NM8074 is expected to improve upon current treatments such as Soliris, Ultomiris, and Empaveli, which are approved for treatment of a limited number of rare diseases.

Key Points: 
  • Through its targeted specificity to the complement alternative pathway (AP), NM8074 is expected to improve upon current treatments such as Soliris, Ultomiris, and Empaveli, which are approved for treatment of a limited number of rare diseases.
  • NM8074 selectively binds to complement alternative pathway specific protein Bb and inhibits the formation of C3a/C3b, C5a/C5b, and MAC, products that mediate inflammation and tissue damage in numerous diseases.
  • NM8074 carries a mechanistic advantage over current platforms because it targets the disease-specific complement alternative pathway (AP) without blocking the classical pathway (CP) required for host defense.
  • NovelMed is a clinical-stage biopharmaceutical company committed to innovating and developing biologics to treat rare diseases caused by the overactivation of the complement alternative pathway.

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.

 Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
Monday, June 7, 2021
Biotechnology, Pharmaceutical, Teens, Health, FDA, Children, Consumer, Clinical trials, Life sciences, Health care, Orphan drugs, Biotechnology, Alexion Pharmaceuticals, AstraZeneca, Paroxysmal nocturnal hemoglobinuria, PNH, Ravulizumab, Paroxysmal attack, Eculizumab, Pegcetacoplan, Paroxysmal Nocturnal Hemoglobinuria (PNH), ULTOMIRIS®, Alexion, PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH), ULTOMIRIS®, ALEXION

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH).

Key Points: 
  • Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH).
  • ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH.
  • Since its initial approval in 2018, ULTOMIRIS has quickly become the standard of care in the U.S. for the treatment of adults with PNH.
  • This approval is based on interim Phase 3 study results, which showed that ULTOMIRIS was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age.

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).