PNH

Onco360 Has Been Selected as the Sole National Specialty Pharmacy Partner for Voydeya® (danicopan)

Retrieved on: 
Monday, April 8, 2024
Immune system, PNH, Specialty pharmacy, Patient, Ravulizumab, FDA, Eculizumab, Paroxysmal nocturnal hemoglobinuria, EVH, Blood, Alexion Pharmaceuticals

LOUISVILLE, Ky., April 08, 2024 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a national pharmacy partner by Alexion Pharmaceuticals for Voydeya® (Danicopan) as a first class oral, Factor D inhibitor.

Key Points: 
  • LOUISVILLE, Ky., April 08, 2024 (GLOBE NEWSWIRE) -- Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a national pharmacy partner by Alexion Pharmaceuticals for Voydeya® (Danicopan) as a first class oral, Factor D inhibitor.
  • It is FDA-approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in combination with C5 inhibitor, Ultomiris® (Ravulizumab) or Soliris® (Eculizumab) for patients that experience clinically significant extravascular hemolysis (EVH).1 Voydeya® is an oral, Factor D inhibitor that acts in the alternative complement pathway of the immune system, providing control of red blood cell destruction within and outside the blood vessels.
  • “Onco360 appreciates the opportunity to partner further with the team at Alexion and become the sole national specialty pharmacy provider for Voydeya®”, said Benito Fernandez, Chief Commercial Officer.
  • “We are proud to add this first in class oral Factor D inhibitor treatment option for PNH patients.”

Deepening the Partnership: Dawn Health Introduces Ekiva for Enhanced Disease Management of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
Monday, April 8, 2024
Digital health, Disease, Caregiver, Partnership, Novartis, PNH, Patient, Conditional sentence, Multiple sclerosis, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

This latest Ekiva product launch signifies not only a continuation of the innovative and collaborative efforts between Dawn Health and Novartis, but also illustrates the customizability and efficiency of the modular Dawn platform.

Key Points: 
  • This latest Ekiva product launch signifies not only a continuation of the innovative and collaborative efforts between Dawn Health and Novartis, but also illustrates the customizability and efficiency of the modular Dawn platform.
  • Ekiva PNH is tailored to the specific needs of the PNH patient population, offering enhanced functionalities for supporting bespoke care and setting a new standard in patient personalization.
  • Ekiva addresses these challenges by providing a comprehensive digital platform that empowers a patient reducing the uncertainties associated with disease management.
  • With the launch of Ekiva PNH, Dawn Health and Novartis reaffirm their position at the forefront of digital innovation in healthcare, setting a new standard in the management of rare chronic diseases.

Deepening the Partnership: Dawn Health Introduces Ekiva for Enhanced Disease Management of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
Monday, April 8, 2024
Digital health, Disease, Caregiver, Partnership, Novartis, PNH, Patient, Conditional sentence, Multiple sclerosis, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

This latest Ekiva product launch signifies not only a continuation of the innovative and collaborative efforts between Dawn Health and Novartis, but also illustrates the customizability and efficiency of the modular Dawn platform.

Key Points: 
  • This latest Ekiva product launch signifies not only a continuation of the innovative and collaborative efforts between Dawn Health and Novartis, but also illustrates the customizability and efficiency of the modular Dawn platform.
  • Ekiva PNH is tailored to the specific needs of the PNH patient population, offering enhanced functionalities for supporting bespoke care and setting a new standard in patient personalization.
  • Ekiva addresses these challenges by providing a comprehensive digital platform that empowers a patient reducing the uncertainties associated with disease management.
  • With the launch of Ekiva PNH, Dawn Health and Novartis reaffirm their position at the forefront of digital innovation in healthcare, setting a new standard in the management of rare chronic diseases.

VOYDEYA™ approved in the US as add-on therapy to ravulizumab or eculizumab for treatment of extravascular hemolysis in adults with the rare disease PNH

Retrieved on: 
Monday, April 1, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Factor D, Arthralgia, Chest pain, Confusion, Neuropsychological test, Infection, Eculizumab, Risk, Headache, ALPHA, PNH, C5, Patient, Orphan, Mitigation, Vaccine, Light, Paroxysmal nocturnal hemoglobinuria, EVH, Bacteria, University, Chills, MD, Breakthrough therapy, Cough, European Medicines Agency, Fatigue, Nausea, Rash, Fever, Priority, Safety, Immune system, REMS, Division, Vomiting, Hematology, Patient safety, Medicine

VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.

Key Points: 
  • VOYDEYA has also been granted Orphan Drug Designation in the US, European Union (EU) and Japan for the treatment of PNH.
  • You must complete or update meningococcal vaccine(s) and streptococcus vaccine(s) at least 2 weeks before your first dose of VOYDEYA.
  • If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting VOYDEYA.
  • Carry it with you at all times during treatment and for 1 week after your last VOYDEYA dose.

Omeros Corporation Reports Fourth Quarter and Year-End 2023 Financial Results

Retrieved on: 
Monday, April 1, 2024
Mental Health, Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Science, Oncology, PIN, PNH, Patient, Sale, CUD, Graft-versus-host disease, Investment, Clinical trial, Hemolysis, Calcineurin, Emory University, ASH, Risk, L-DOPA, Thumbay Hospital, Behavior informatics, Trial of the century, Myelodysplastic syndrome, Cancer, Safety, Transplant, LID, DRI, BLA, Corporation, Paroxysmal nocturnal hemoglobinuria, Tardive dyskinesia, Parkinson's disease, Publication, DSM-IV codes, Retirement, FDA, PDE7, LDH, Hematology, Mortality, Hematopoietic stem cell transplantation, Bone marrow failure, Abstract, Society, Pharmacology, Pharmaceutical industry

Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.

Key Points: 
  • Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.
  • Total operating expenses for the fourth quarter of 2023 were $39.8 million compared to $48.2 million for the third quarter of 2023.
  • Interest expense during the fourth quarter of 2023 was $7.1 million compared to $7.9 million during the prior quarter.
  • During the fourth quarter of 2023, we earned $3.4 million in interest and other income compared to $4.4 million in the third quarter.

Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024
PEGASUS, Quarter, EMA, Committee, ALS, APLS, Recurrence, AAO, ASH, Note, Physician, FDA, Marketing, Data, Treasury, Safety, CAD, Society, VALIANT, Cold agglutinin disease, CHMP, European Medicines Agency, Administration, Patient, Public expenditure, Webcast, GALE, PRINCE, PNH, ASN, Research, Travel, American Academy, Pharmaceutical industry

Cost of sales was $19.9 million for the fourth quarter of 2023, compared to $2.9 million for the fourth quarter of 2022, respectively.

Key Points: 
  • Cost of sales was $19.9 million for the fourth quarter of 2023, compared to $2.9 million for the fourth quarter of 2022, respectively.
  • R&D expenses were $69.3 million for the fourth quarter of 2023, compared to $99.4 million for the fourth quarter of 2023.
  • G&A expenses were $141.7 million for the fourth quarter of 2023, compared to $84.4 million for the fourth quarter of 2022.
  • Apellis will host a conference call and webcast to discuss its fourth quarter and year end 2023 financial results and business highlights today, February 27, 2024, at 8:30 a.m.

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

Apellis Announces Preliminary Fourth Quarter and Full Year 2023 U.S. Net Product Revenues

Retrieved on: 
Monday, January 8, 2024
Conference, Geographic atrophy, Injector, PNH, APLS, Webcast, Patient, Medicine, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

WALTHAM, Mass., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced preliminary U.S. net product revenues of approximately $138 million for the fourth quarter and approximately $366 million for the full year 2023 for SYFOVRE® (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration and for EMPAVELI® (pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Key Points: 
  • WALTHAM, Mass., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced preliminary U.S. net product revenues of approximately $138 million for the fourth quarter and approximately $366 million for the full year 2023 for SYFOVRE® (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration and for EMPAVELI® (pegcetacoplan) for adults with paroxysmal nocturnal hemoglobinuria (PNH).
  • Approximately $114 million and $275 million expected in preliminary U.S. net product revenues in the fourth quarter and full year 2023, respectively.i
    Approximately 62,000 doses (commercial and sample vials) distributed to physician practices in the fourth quarter; approximately 160,000 total doses have been distributed in 2023.
  • “Additionally, the high compliance observed with EMPAVELI speaks to the important impact of this medicine on patients’ lives.
  • The event will be available via a live webcast from the “Events and Presentations” page of the “Investors and Media” section of the company’s website .

USAN Approves Generic Name “Ruxoprubart” for NM8074, an Antibody Therapy focused on Complement-Mediated Diseases

Retrieved on: 
Monday, January 8, 2024
Clinical trial, Marketing, Anemia, PNH, USAN, Alternative complement pathway, Name, United States Adopted Name, Paroxysmal nocturnal hemoglobinuria, Patient, Safety, Publication, Clinical research associate, PRBC, AP, Vaccine

Phase I Clinical Trial results in healthy subjects demonstrated the safety and well-tolerance of NM8074.

Key Points: 
  • Phase I Clinical Trial results in healthy subjects demonstrated the safety and well-tolerance of NM8074.
  • Across all cohorts, total Alternative Pathway (AP) inhibition was achieved, with the duration of AP inhibition showing a dose-dependent response.
  • The USAN Council has approved the nonproprietary name "Ruxoprubart" (pronounced Ruk” soe proo’ bart) for NM8074, designating it as a first-in-class Alternative Pathway selective investigational monoclonal antibody.
  • Ruxoprubart, a potent and highly selective investigational drug candidate, targets the complement protein Bb, playing a crucial role in benefiting several complement-mediated diseases.