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Roche's PiaSky Approved in EU as First Monthly Self-Administered Treatment for Paroxysmal Nocturnal Haemoglobinuria

Monoclonal AntibodyRare DiseaseDrug Approval
  • The European Commission has approved PiaSky (crovalimab), the first monthly subcutaneous treatment for paroxysmal nocturnal haemoglobinuria (PNH), offering patients the option to self-administer following training.
  • PiaSky utilizes innovative recycling antibody technology developed by Chugai Pharmaceutical, allowing it to bind to C5 protein multiple times and remain effective longer with a small volume of medicine.
  • Clinical trials demonstrated PiaSky's non-inferiority to eculizumab, the current standard of care, while potentially reducing treatment burden through less frequent administration and eliminating the need for regular clinic visits.

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT04861259Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/22/2021

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

NCT04434092Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/8/2020

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

NCT04432584Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 9/30/2020

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT04958265Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/17/2021

Federal Circuit Reverses Patent Invalidation for Allergan's IBS-D Drug Viberzi in Landmark Double Patenting Case

Drug Approval
  • The Federal Circuit reversed a district court's invalidation of Allergan's patent claims for eluxadoline (Viberzi®), used to treat irritable bowel syndrome with diarrhea, clarifying key principles in obviousness-type double patenting doctrine.
  • The court held that a first-filed, first-issued patent cannot be invalidated by later-filed, later-issued patents from the same family simply because they expire earlier due to Patent Term Adjustment differences.
  • The decision also reversed findings on written description requirements, ruling that patent specifications need not explicitly label every component as "optional" if a skilled person would understand the invention as disclosed.
  • This ruling provides important protections for patentees who receive Patent Term Adjustment on first-filed applications, preventing invalidation based solely on expiration date comparisons with related patents.

PTAB Invalidates Multiple Regeneron EYLEA Patents as Biosimilar Competition Intensifies

Drug Approval
  • The Patent Trial and Appeal Board (PTAB) has issued multiple final written decisions finding Regeneron's aflibercept-dosing patents for EYLEA unpatentable, with Regeneron voluntarily dismissing several Federal Circuit appeals.
  • Samsung Bioepis's EYLEA biosimilar Opuviz received FDA approval as an interchangeable biosimilar on May 20, 2024, but faces a preliminary injunction preventing commercial launch.
  • The patent invalidations affect key intellectual property protecting EYLEA, which generated $5.89 billion in sales for Regeneron in 2023, signaling potential increased biosimilar competition.

IASO Bio Secures FDA Approval for BCMA CAR-T Therapy in Multiple Autoimmune Diseases

CAR-TDrug Approval
  • IASO Bio received FDA IND approval for Equecabtagene Autoleucel (Eque-cel) to treat multiple sclerosis, marking the second autoimmune indication approved in 2024.
  • The fully human anti-BCMA CAR-T therapy has now secured five total IND approvals across China and the US for various autoimmune diseases including lupus and myasthenia gravis.
  • Multiple sclerosis affects approximately 3.07 million patients worldwide with 400,000 in the US, representing a significant unmet medical need in neuroinflammatory diseases.
  • The therapy targets BCMA-expressing B cells and plasma cells to reduce pathogenic autoantibodies and suppress autoimmune inflammation across multiple disease indications.

NHS England Approves First CRISPR Gene-Editing Therapy for Beta Thalassaemia Treatment

Gene EditingDrug ApprovalRare Disease
  • NHS England has approved Casgevy, the first CRISPR gene-editing therapy, for treating severe beta thalassaemia in patients aged 12 and older, making it among the first healthcare systems globally to offer this treatment.
  • Clinical trials demonstrated that 93% of beta thalassaemia patients did not require blood transfusions for at least a year after receiving the one-time gene therapy, offering potential lifetime cure for a condition that typically requires transfusions every 3-5 weeks.
  • The therapy will be available at seven specialist NHS centres within weeks, with an estimated 460 patients in England potentially eligible for the treatment that uses Nobel Prize-winning CRISPR technology to edit bone marrow stem cells.

FDA Accepts sBLA for Susvimo in Diabetic Eye Diseases Following Promising Phase 3 Results

Drug Approval
• The FDA has accepted Roche's supplemental Biologics License Application for Susvimo to treat diabetic macular edema and diabetic retinopathy, based on successful Phase 3 trial results.
• Susvimo, a refillable ocular implant that continuously delivers ranibizumab, demonstrated non-inferior visual gains compared to monthly injections in DME patients, with 95% requiring no additional treatment during the study period.
• The treatment recently returned to the US market for wet AMD after addressing quality issues, potentially offering a paradigm shift in diabetic eye disease management by reducing the need for frequent injections.

A Study to Evaluate Efficacy, Safety & Pharmacokinetics of the Port Delivery System (PDS) With Ranibizumab in Participants With Diabetic Macular Edema (DME) Compared With Intravitreal Ranibizumab; A Subtudy to Evaluate the Safety of Re-Implanting the PDS With Ranibizumab in Participants With DME

NCT04108156RecruitingPhase 3
Hoffmann-La Roche
Posted 9/30/2019

A Multicenter, Randomized Study in Participants With Diabetic Retinopathy Without Center-involved Diabetic Macular Edema To Evaluate the Efficacy, Safety, and Pharmacokinetics of Ranibizumab Delivered Via the Port Delivery System Relative to the Comparator Arm

NCT04503551Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 8/10/2020

Global Breast Cancer Therapeutics Market Expected to Reach $78.61 Billion by 2033

OncologyTargeted TherapyMonoclonal AntibodyAIPrecision MedicineDrug Approval
• The global breast cancer therapeutics market, valued at $32.93 billion in 2023, is projected to reach $78.61 billion by 2033, growing at a CAGR of 9.09% over the next decade.
• North America dominates the market with a 38.61% revenue share, driven by high breast cancer prevalence and presence of key pharmaceutical companies including Pfizer, Roche, and Novartis.
• Targeted therapies hold the largest market segment at 64.85%, with hormone receptor-positive treatments accounting for 66.97% of the market as precision medicine approaches gain traction.

FDA Advisory Panel Unanimously Recommends Approval of Lilly's Alzheimer's Drug Donanemab

Drug Approval
  • An FDA advisory committee voted 11-0 to recommend approval of Eli Lilly's donanemab for early Alzheimer's disease, concluding that benefits outweigh risks despite safety concerns.
  • Clinical studies demonstrated that donanemab significantly reduced amyloid plaque buildup in the brain and plasma phosphorylated tau levels, key biomarkers of Alzheimer's disease.
  • If approved later this year, donanemab would become the second drug on the market to treat Alzheimer's disease, with Lilly expecting blockbuster sales potential.
  • The unanimous recommendation positions donanemab for a market showdown with existing competitor Leqembi following anticipated FDA approval.

InnoCare Advances Dual Regulatory Milestones with Tafasitamab BLA Acceptance and BCL2 Inhibitor ICP-248 Clinical Trial Approval in China

Monoclonal AntibodyDrug Approval
  • China's NMPA has accepted and granted priority review to InnoCare's biologics license application for tafasitamab plus lenalidomide combination therapy for relapsed or refractory diffuse large B-cell lymphoma patients ineligible for stem cell transplant.
  • The company received separate approval to initiate clinical trials of its novel BCL2 inhibitor ICP-248 in combination with azacitidine for acute myeloid leukemia treatment in China.
  • DLBCL represents the most common non-Hodgkin lymphoma type globally, accounting for 45.8% of all NHL cases in China, highlighting significant unmet medical need.
  • These regulatory advances strengthen InnoCare's hemato-oncology pipeline with complementary mechanisms targeting CD19 and BCL2 pathways across different blood cancer indications.

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

Three RSV Vaccines Now Available for Older Adults as FDA Approves Moderna's mRESVIA

Drug Approval
  • The FDA has approved three RSV vaccines for adults 60 years and older, with Moderna's mRESVIA joining GSK's Arexvy and Pfizer's Abrysvo in the market as of May 2024.
  • Clinical trials demonstrate high efficacy rates across all three vaccines, with Arexvy showing 82.6% effectiveness, Abrysvo achieving 88.9% efficacy, and mRESVIA demonstrating 83.7% protection against RSV-associated lower respiratory tract disease.
  • The vaccines employ different approaches, with Abrysvo being bivalent targeting both RSV A and B strains, Arexvy containing an adjuvant for enhanced immune response, and mRESVIA utilizing mRNA technology.
  • Pharmacists are positioned to play a crucial role in RSV vaccination efforts, given their accessibility and expertise, as nine in ten Americans live within five miles of a pharmacy.

A Study to Evaluate the Safety and Efficacy of mRNA-1345 Vaccine Targeting Respiratory Syncytial Virus (RSV) in Adults ≥60 Years of Age

NCT05127434Active, Not RecruitingPhase 2
ModernaTX, Inc.
Posted 11/17/2021

Study to Evaluate the Efficacy, Immunogenicity, and Safety of RSVpreF in Adults.

NCT05035212RecruitingPhase 3
Pfizer
Posted 8/31/2021

Efficacy Study of GSK's Investigational Respiratory Syncytial Virus (RSV) Vaccine in Adults Aged 60 Years and Above

NCT04886596CompletedPhase 3
GlaxoSmithKline
Posted 5/25/2021
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