MedPath

Tagged News

Novartis Secures Pan-Canadian Agreement for Leqvio Access in Familial Hypercholesterolemia

Drug ApprovalRare Disease
  • Novartis successfully concluded negotiations with the pan-Canadian Pharmaceutical Alliance for public reimbursement of Leqvio (inclisiran) to treat adults with heterozygous familial hypercholesterolemia.
  • The agreement represents a significant step toward addressing treatment gaps for patients with this genetic condition who face elevated cardiovascular risk from high LDL cholesterol levels.
  • While pCPA negotiations are complete, public reimbursement through provincial and territorial formularies has not yet been secured, requiring further collaboration with drug programs.
  • Since commercialization in 2022, Leqvio has treated more than 4,500 Canadians and 290,000 patients worldwide following Health Canada approval in 2021.

Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol

NCT03399370CompletedPhase 3
The Medicines Company
Posted 12/21/2017

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH)

NCT03397121CompletedPhase 3
The Medicines Company
Posted 11/28/2017

Inclisiran for Subjects With ASCVD or ASCVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol

NCT03400800CompletedPhase 3
The Medicines Company
Posted 11/1/2017

FDA Grants Orphan Drug Designation to Crinetics' Atumelnant for Congenital Adrenal Hyperplasia Treatment

Rare Disease
  • The FDA has granted orphan drug designation to atumelnant, Crinetics Pharmaceuticals' novel oral ACTH receptor antagonist for treating classic congenital adrenal hyperplasia.
  • Phase 2 TouCAHn trial results showed up to 80% mean reduction in androstenedione levels and meaningful improvements in clinical symptoms including resumption of menses.
  • The company plans to initiate Phase 3 CALM-CAH study in adults and Phase 2/3 BALANCE-CAH study in pediatrics in the second half of 2025.
  • Orphan drug status provides seven years of market exclusivity, FDA fee exemptions, and financial incentives for clinical development if approved.

Andelyn Biosciences Partners with Amplo Biotechnology to Advance AAV Gene Therapies for Neuromuscular Junction Disorders

Rare Disease
  • Andelyn Biosciences has partnered with Amplo Biotechnology to provide scalable manufacturing of clinical-grade AAV material using Andelyn's suspension AAV Curator® platform for neuromuscular junction disorders.
  • The collaboration aims to advance Amplo's AAV gene therapy programs toward clinical evaluation, with a focus on safety and efficacy for upcoming trials targeting genetic NMJ diseases.
  • Neuromuscular junction disorders typically result in muscle weakness and can be life-threatening, with genetic forms usually diagnosed in early childhood but potentially manifesting in adolescence or adulthood.
  • Amplo's lead program, AMP-101, is advancing toward a first-in-human trial in Dok7 congenital myasthenic syndrome, built on research from the University of Tokyo and Oxford.

Savara's Inhaled Molgramostim Shows 9.8% Lung Function Improvement in Phase 3 Autoimmune PAP Trial

Rare Disease
  • Savara's Phase 3 IMPALA-2 trial demonstrated that inhaled molgramostim significantly improved lung function by 9.8% at 24 weeks compared to 3.8% with placebo in autoimmune pulmonary alveolar proteinosis patients.
  • The treatment showed sustained benefits through 48 weeks with 11.6% improvement in gas transfer, while also enhancing quality of life and exercise capacity in the largest clinical trial conducted in this rare disease.
  • Results from the 43-site global study will be published in the New England Journal of Medicine, marking a potential breakthrough for patients with this chronic rare lung condition.

Keros Therapeutics Receives FDA Orphan Drug Designation for KER-065 in Duchenne Muscular Dystrophy

Rare Disease
  • Keros Therapeutics announced that the U.S. FDA granted Orphan Drug designation to KER-065 for the treatment of Duchenne muscular dystrophy on August 20, 2025.
  • The designation provides potential benefits including tax credits for clinical testing, waiver of FDA application fees, and seven years of market exclusivity if approved.
  • KER-065 is a novel ligand trap designed to inhibit myostatin and activin A to increase muscle regeneration and strength in patients with neuromuscular diseases.
  • The company plans to advance KER-065 into a Phase 2 clinical trial for DMD patients, addressing a significant unmet medical need in this rare disease population.

Invion's INV043 Receives FDA Orphan Drug Designation for Anal Cancer Treatment

OncologyRare Disease
  • Invion Limited has secured FDA Orphan Drug Designation for INV043, its lead cancer drug candidate targeting anal cancer treatment.
  • The designation provides seven years of exclusive marketing rights in the US, financial incentives including tax credits, and potential for accelerated approval pathways.
  • Preclinical data demonstrated approximately 80% tumor control in mouse models when INV043 was combined with immune checkpoint inhibitors.
  • Invion plans to conduct clinical trials in collaboration with Peter MacCallum Cancer Centre for anogenital cancers including anal, vulvar, and penile cancers.

āshibio Licenses Vantictumab for Rare Bone Disease ADO2 in Strategic Pipeline Expansion

Monoclonal AntibodyRare Disease
  • āshibio has secured an exclusive global licensing agreement with Mereo BioPharma for vantictumab, a first-in-class monoclonal antibody targeting autosomal dominant osteopetrosis type 2 (ADO2).
  • ADO2 affects 1 in 20,000 births and causes dense, brittle bones leading to multiple fractures, poor healing, and nerve compression, with no currently approved therapies available.
  • Vantictumab inhibits Wnt signaling pathways and has demonstrated favorable safety profiles in previous oncology trials, with biomarker data supporting its activity on osteoclast function.
  • The licensing agreement grants āshibio worldwide development and commercialization rights excluding Europe, where Mereo retains commercial rights.

jCyte Initiates Phase 2 Trial of Famzeretcel Cell Therapy for Retinitis Pigmentosa

Rare Disease
  • jCyte has dosed the first patients in the JC02-88 phase 2 trial evaluating famzeretcel, an allogeneic retinal progenitor cell therapy for retinitis pigmentosa.
  • The study tests a single injection of 8.8 million cells, representing a 50% dose increase from previous trials, with patients monitored for safety and vision changes over 6 months.
  • Retinitis pigmentosa affects approximately 2 million people worldwide and 100,000 in the United States, with patients typically becoming legally blind by middle age.
  • The therapy requires sufficient living cone photoreceptors to achieve visual function restoration through its neurotrophic mechanism.

Atropos Health and Novartis Partner to Deploy AI for Faster Rare Disease Diagnosis in PNH Patients

AIRare DiseaseReal World EvidencePrecision Medicine
  • Atropos Health has partnered with Novartis to develop AI models that identify undiagnosed patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder affecting 10-20 people per million worldwide.
  • The collaboration addresses critical diagnostic delays in PNH, where many patients wait over a year for diagnosis and some wait more than five years due to the disease's rarity and varied symptoms.
  • The AI model, trained on real-world data from the Atropos Evidence Network, is now available for integration into health systems to accelerate appropriate diagnosis at the point of care.
  • The partnership aims to reduce time from initial symptoms to testing, diagnosis and treatment by implementing patient-finding models across health system members of the Atropos Evidence Network.

Opus Genetics Receives FDA Clearance for BEST1 Gene Therapy Trial in Rare Inherited Retinal Disease

Rare Disease
  • Opus Genetics received FDA clearance for its IND application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease.
  • The company plans to initiate a Phase 1/2 multi-center, open-label trial in the second half of 2025 to evaluate safety and preliminary efficacy.
  • BEST1-related IRDs currently have no approved treatments, representing a significant unmet medical need for patients facing progressive vision loss.
  • The therapy uses AAV-based gene delivery to provide functional BEST1 gene copies directly to retinal pigment epithelium cells.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.