Hemoglobinuria

Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

Retrieved on: 
Monday, February 12, 2024
LDH, Infection, FDA, Food, Hemoglobinuria, Paroxysmal nocturnal hemoglobinuria, Alternative complement pathway, ODD, Safety, Atypical hemolytic uremic syndrome, ANCA, Death, Patient, Hyperlipidemia, AP, Diagnosis, AAV, PNH, Chelsea Handler, Hemolysis, Pharmaceutical industry

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.

Key Points: 
  • FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
  • The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV).
  • CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) --  NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition.

Summary of opinion: Aspaveli, 25/01/2024 Positive

Retrieved on: 
Sunday, February 4, 2024
Marketing, Hemoglobinuria, European Commission, Anemia, PNH, Civil service commission, Patient, Committee, CHMP, Medication package insert, Pharmaceutical industry

On 25 January 2024 the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Aspaveli.

Key Points: 
  • On 25 January 2024 the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Aspaveli.
  • The marketing authorisation holder for this medicinal product is Swedish Orphan Biovitrum AB (publ).
  • The CHMP adopted an extension to the existing indication to include patients with paroxysmal nocturnal haemoglobinuria who have haemolytic anaemia and have not had previous treatment with a C5 inhibitor.
  • 1New text in bold, removed text as strikethrough

Paroxysmal Nocturnal Hemoglobinuria Market to Climb Moderately at a CAGR of 7.6% During the Study Period (2019-2032), Assesses DelveInsight

Retrieved on: 
Monday, January 23, 2023
Safety, Therapy, CD55, EU4, Nitric oxide, LAS, Hemolysis, United Kingdom, Arrowhead Pharmaceuticals, III, CD59, Diagnosis, Death, BioCryst Pharmaceuticals, Immunosuppression, Eculizumab, Urine, Novartis, Haematopoietic system, Roche, Flow cytometry, GPI, Hemoglobinuria, HSC, US, Bone marrow failure, Drug development, FDA, Paroxysmal nocturnal hemoglobinuria, PIGA, Alnylam Pharmaceuticals, Venous thrombosis, SCR, Regeneron Pharmaceuticals, Patient, Alexion Pharmaceuticals, Czech Republic, Hemosiderin, Disease, PNH, Thrombosis, Pharmaceutical industry, Lithium, Medical device, MorphoSys

LAS VEGAS, Jan. 23, 2023 /PRNewswire/ -- DelveInsight's Paroxysmal Nocturnal Hemoglobinuria Market Insights report includes a comprehensive understanding of current treatment practices, paroxysmal nocturnal hemoglobinuria emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Points: 
  • In December 2022, Novartis released the efficacy and adverse event data from the phase III APPLY-PNH trial in paroxysmal nocturnal hemoglobinuria.
  • In September 2022, efficacy and safety data from a phase III trial in paroxysmal nocturnal hemoglobinuria were released by Alexion.
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare hematological disorder characterized by intravascular hemolysis, hemoglobinuria, hemolysis, and venous thrombosis.
  • To know more about paroxysmal nocturnal hemoglobinuria treatment guidelines, visit @ Paroxysmal Nocturnal Hemoglobinuria Management

KORU Medical Systems Receives FDA 510(k) Clearance for Delivery of EMPAVELI® (pegcetacoplan), Expands European Label for FreedomEdge®

Retrieved on: 
Monday, May 2, 2022
FDA, Health, Medical Supplies, Medical Devices, Linda, Flo, FREEDOM, Forward-looking statement, Mutation, NASDAQ, Fatigue, Patient, FDA, Risk, Annual report, Breathe Me, PNH, Hemoglobinuria, Quality of life, Conditional sentence, Pharmacotherapy, KRMD, Med Systems Software, SEC, Federal Food, Drug, and Cosmetic Act, Paroxysmal nocturnal hemoglobinuria, Medical device

EMPAVELI and Aspaveli are approved to treat adults with a chronic disease called paroxysmal nocturnal hemoglobinuria (PNH), which affects approximately 15,000 patients with PNH worldwide.

Key Points: 
  • EMPAVELI and Aspaveli are approved to treat adults with a chronic disease called paroxysmal nocturnal hemoglobinuria (PNH), which affects approximately 15,000 patients with PNH worldwide.
  • The FreedomEdge was used in EMPAVELIs PNH pivotal studies and is now commercially available.
  • This FDA clearance is another example of our strategy to expand the use of our self-administered Freedom pump platforms to further drug therapies and optimize the infusion experience."
  • KORU Medical Systems develops, manufactures, and commercializes innovative and easy-to-use specialty infusion solutions that improve quality of life for patients around the world.

Aspaveli®/Empaveli™ (pegcetacoplan) demonstrated sustained normalisation of clinical measures in a broad PNH patient population

Retrieved on: 
Monday, December 13, 2021
Patient, Hemoglobinuria, Society, Disease, Standard of care, PRINCE, Nasdaq, LDH, Swedish Orphan Biovitrum, PNH, ASH, Pharmaceutical industry

STOCKHOLM, Dec. 13, 2021 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced today new data demonstrating that Aspaveli®/Empaveli™ (pegcetacoplan) provides consistent, sustained normalisation of clinical measures across patients with paroxysmal nocturnal haemoglobinuria (PNH) who are treatment-naïve and patients with baseline haemoglobin levels greater than or equal to 10.0 g/dL. The data were presented at the American Society of Hematology Annual Meeting (ASH) taking place 11 – 14 December 2021. 

Key Points: 
  • The data were presented at the American Society of Hematology Annual Meeting (ASH) taking place 11 14 December 2021.
  • "Pegcetacoplan has the potential to elevate the standard of care for adults with PNH regardless of prior treatment or baseline haemoglobin levels."
  • The PRINCE study (NCT04085601) was a randomised, multi-centre, open-label, controlledphase 3 study in 53 treatment-nave adults with paroxysmal nocturnal haemoglobinuria (PNH).
  • The PEGASUS study (NCT03500549) was a multi-centre, randomised, head-to-headphase 3 study in 80 adults with paroxysmal nocturnal haemoglobinuria (PNH).

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).

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).