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GSK Receives FDA Orphan Drug Designation for Autosomal Dominant Polycystic Kidney Disease Treatment

Monoclonal AntibodyRare DiseaseFDA Approval
  • GSK received FDA Orphan Drug Designation for its investigational treatment of autosomal dominant polycystic kidney disease (ADPKD), a rare genetic disorder affecting approximately 600,000 people in the U.S.
  • The designation provides critical incentives including tax credits, reduced regulatory fees, and seven years of market exclusivity upon approval for GSK's monoclonal antibody candidate currently in Phase 1 trials.
  • GSK's kidney disease strategy extends beyond ADPKD through a £50 million, five-year partnership with University of Cambridge focusing on immune-related kidney diseases using AI-driven precision therapies.
  • The company's diversified approach includes Atacicept, a B-cell inhibitor for IgA nephropathy that recently completed Phase III trials, positioning GSK to capture market share in the growing kidney disease therapeutics sector.

FDA Recommends Enhanced Brain Monitoring for Alzheimer's Patients on Leqembi Following Six Early Deaths

Monoclonal Antibody
  • The FDA is requiring additional brain scans between the second and third Leqembi infusions after identifying six deaths early in treatment related to brain swelling complications.
  • The enhanced monitoring aims to detect amyloid-related imaging abnormalities with edema (ARIA-E), characterized by brain swelling or fluid buildup, before serious complications occur.
  • Leqembi, developed by Eisai and Biogen, was approved in 2023 as the first therapy targeting an underlying cause of Alzheimer's disease and has shown a 31% reduction in clinical decline.
  • The new monitoring requirements are more stringent than the current protocol, which recommends MRI scans before the fifth, seventh, and fourteenth infusions.

Gastric Cancer Pipeline Shows Robust Growth with 220+ Therapies in Development Across 200+ Companies

Monoclonal AntibodyOncology
  • DelveInsight's 2025 pipeline report reveals over 200 companies developing 220+ therapeutic candidates for gastric cancer treatment across all clinical stages.
  • Recent clinical developments include Bold Therapeutics' Phase 1b/2a study of BOLD-100 with FOLFOX chemotherapy and Jazz Pharmaceuticals' zanidatamab combination trials.
  • Leading pipeline candidates include tislelizumab (BeiGene), catumaxomab (Linton Pharm), and tivumecirnon (RAPT Therapeutics) targeting various mechanisms from PD-1 inhibition to regulatory T cell migration.
  • The pipeline encompasses diverse therapeutic approaches including monoclonal antibodies, small molecules, and combination therapies targeting novel mechanisms like Claudin 18.2 and DKK1 signaling.

MS Pharma Opens First Biologics Manufacturing Facility in Middle East with $50 Million Saudi Arabia Investment

Monoclonal Antibody
  • MS Pharma has inaugurated the Middle East's first biologics manufacturing facility in Saudi Arabia, receiving GMP approval from SFDA and meeting EMA and FDA standards.
  • The $50 million facility specializes in monoclonal antibodies and complex peptides, featuring the region's first in-house bioanalytical testing laboratories.
  • The strategic hub enables global pharmaceutical partnerships for market access to MENA, EU, and US markets through localized and contract manufacturing solutions.
  • The facility aligns with Saudi Arabia's Vision 2030 localization goals, supporting technology transfer and high-skilled employment in the region.

Mount Sinai Researchers Discover Potent Monoclonal Antibodies Against Mpox with Complete Protection in Animal Models

Monoclonal Antibody
  • Researchers at Mount Sinai identified three powerful monoclonal antibodies targeting the A35 viral protein that completely prevented death and protected rodents from severe mpox disease in laboratory studies.
  • The antibodies bind to a highly conserved region across the entire poxvirus family, making them resistant to viral mutations and potentially effective against multiple orthopoxviruses.
  • Humans previously infected with mpox carry high levels of these protective antibodies, with their presence associated with milder symptoms and reduced hospitalization rates.
  • The discovery addresses a critical unmet medical need, as no approved drugs currently exist to treat mpox despite ongoing global outbreaks and WHO emergency declarations.

FDA Expands Repatha Indication to Include Adults at High Risk for Cardiovascular Events Without Prior Disease Diagnosis

Monoclonal AntibodyDrug Approval
  • The FDA has expanded Repatha's (evolocumab) indication to include adults at increased risk for major adverse cardiovascular events due to uncontrolled LDL cholesterol, removing the requirement for prior cardiovascular disease diagnosis.
  • Real-world data from over 1.3 million patients showed a 1.4% incidence of major adverse cardiovascular events within one year, rising to 6.9% at four years among at-risk patients.
  • Repatha, a PCSK9 inhibitor, has demonstrated 54% to 77% LDL cholesterol reduction compared to placebo in clinical trials spanning 15 years of research across 50 studies involving more than 57,000 patients.

VYVGART Achieves Primary Endpoint in Phase 3 Trial for AChR-Ab Seronegative Myasthenia Gravis

Monoclonal Antibody
  • argenx's VYVGART met its primary endpoint in the ADAPT SERON study with a statistically significant improvement in MG-ADL scores (p-value=0.0068) for AChR-Ab seronegative generalized myasthenia gravis patients.
  • The study represents the first global Phase 3 trial to demonstrate clinically meaningful improvements across all three seronegative subtypes: MuSK+, LRP4+, and triple seronegative patients.
  • argenx plans to submit a supplemental Biologics License Application to the FDA by end of 2025 to expand VYVGART's label to include all AChR-Ab seronegative gMG patients.
  • The treatment was well tolerated with no new safety concerns identified, consistent with VYVGART's established safety profile in other indications.

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Episodic Migraine

NCT07018700RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

NCT05070858Active, Not RecruitingPhase 3
Regeneron Pharmaceuticals
Posted 12/14/2021

A Clinical Trial to Evaluate Efficacy and Safety of Xeomin® Injections for Preventing Chronic Migraine

NCT07018713RecruitingPhase 3
Merz Therapeutics GmbH
Posted 8/21/2025

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

NCT06298552Active, Not RecruitingPhase 3
argenx
Posted 4/16/2024

PBAC Rejects PBS Listing for Alzheimer's Drug Donanemab Despite Clinical Need

Drug ApprovalMonoclonal Antibody
  • The Pharmaceutical Benefits Advisory Committee (PBAC) rejected Lilly's application for PBS listing of donanemab (Kisunla), the first new Alzheimer's treatment registered in Australia in 25 years.
  • While clinical trials showed donanemab can delay early Alzheimer's disease progression by approximately six weeks, PBAC found the benefits too small and uncertain to justify the treatment burden.
  • Patients can still access the drug privately at $4,700 per infusion every four weeks for 18 months, creating significant equity concerns for those unable to afford the treatment.
  • The decision highlights ongoing challenges in balancing clinical innovation with healthcare system sustainability for Australia's aging population.

Harbour BioMed Appoints Yajie Li as Chief Medical Officer to Advance Antibody Therapeutics Pipeline

Monoclonal AntibodyOncology
  • Harbour BioMed has appointed Yajie Li as Chief Medical Officer to lead clinical development and regulatory strategy for the company's immunology and oncology programs.
  • Li brings over a decade of pharmaceutical experience from multinational corporations including Merck and Johnson & Johnson, plus nine years at China's National Medical Products Administration.
  • The appointment strengthens Harbour BioMed's capabilities as it advances its pipeline using proprietary antibody technologies including Harbour Mice, HBICE, and HBICA platforms.
  • Li will evaluate early-stage assets based on unmet medical needs and scientific differentiation while reporting directly to CEO Dr. Jingsong Wang.

Invivyd Raises $57.5 Million to Advance Monoclonal Antibody Pipeline Targeting RSV, Measles, and Long COVID

Monoclonal Antibody
  • Invivyd completed a $57.5 million public offering in August 2025 to accelerate development of monoclonal antibody candidates for respiratory syncytial virus, measles, and Long COVID treatments.
  • The company aims to identify a best-in-class RSV monoclonal antibody candidate by Q3 2025, targeting a $3 billion global market opportunity by 2027.
  • Invivyd's SPEAR Study Group launched in July 2025 represents entry into the $10 billion+ Long COVID treatment market through collaboration with leading researchers.
  • The capital raise is paired with a $30 million non-dilutive term loan from Silicon Valley Bank, providing financial flexibility while minimizing equity dilution.

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