MedPath

Tagged News

FDA Grants Orphan Drug Designation to Xinnate's TCP-25 for Epidermolysis Bullosa Treatment

Rare Disease
  • The US FDA has granted Orphan Drug Designation to Xinnate's TCP-25, an immunomodulatory peptide developed for treating the rare skin disease Epidermolysis Bullosa.
  • TCP-25 demonstrates dual-action capabilities by targeting both inflammation and bacterial infection in wounds, potentially offering more effective healing for EB patients.
  • The designation provides Xinnate with benefits including tax credits, FDA fee exemptions, and seven years of market exclusivity following approval, supporting the company's plans for Phase 2 trials in 2025.

Catalyst Pharmaceuticals Secures Patent Settlement with Teva, Extending Firdapse Market Exclusivity Until 2035

Orphan DrugRare Disease
  • Catalyst Pharmaceuticals and its licensor SERB S.A. reached a settlement agreement with Teva Pharmaceuticals that prevents Teva from marketing a generic version of Firdapse until February 25, 2035.
  • The settlement resolves patent litigation over Firdapse 10 mg tablets, which treats Lambert-Eaton myasthenic syndrome (LEMS), a rare neuromuscular disorder.
  • Patent disputes with remaining defendants Hetero and Lupin continue, while the agreement must undergo review by the Federal Trade Commission and Department of Justice.
  • The settlement strengthens Catalyst's market position for its flagship orphan drug, following previous regulatory challenges that established important precedents for orphan drug exclusivity.

Ionis Reports Positive Phase 3 Results for Olezarsen in Familial Chylomicronemia Syndrome

Rare DiseaseOrphan Drug
  • Ionis Pharmaceuticals' olezarsen met primary and key secondary endpoints in Phase 3 BALANCE trial for familial chylomicronemia syndrome (FCS), demonstrating significant triglyceride reduction.
  • The antisense therapy showed a favorable safety profile with no major adverse events reported, potentially offering a new treatment option for this rare genetic disorder.
  • Ionis plans to submit regulatory applications in the coming months, positioning olezarsen to potentially become the first approved therapy specifically targeting FCS.

First-of-its-kind Gene Editing Treatment Saves Baby with Rare Genetic Disorder

Rare Disease
  • Doctors at Children's Hospital of Philadelphia successfully treated a baby with CPS1 deficiency using a custom-designed CRISPR base editing therapy, marking a groundbreaking advancement in personalized genetic medicine.
  • The experimental treatment corrected a specific genetic mutation in KJ Muldoon's DNA, allowing his liver to properly process ammonia and preventing toxic buildup that threatens brain development and survival.
  • Researchers believe this pioneering approach could transform treatment for millions with rare genetic disorders, demonstrating that personalized gene therapies can be developed relatively quickly and at costs comparable to conventional treatments.

GRI Bio Advances Phase 2a Trial of GRI-0621 for Idiopathic Pulmonary Fibrosis with Promising Interim Safety Data

Rare Disease
  • GRI Bio has completed enrollment for the interim analysis of its Phase 2a trial evaluating GRI-0621 in idiopathic pulmonary fibrosis patients, with 24 of 36 planned patients randomized.
  • Early safety data from the first 12 patients shows GRI-0621 is well-tolerated with no significant changes in lipid profiles, consistent with previous studies of related compounds.
  • The company has secured $13.9 million in funding since early 2024, extending its cash runway through the interim data readout expected in Q1 2025, with topline results anticipated in Q2 2025.

Biomarker Modulation and the Inhibition of NKT1 Cells by Oral GRI-0621 in Patients With IPF

NCT06331624CompletedPhase 2
GRI Bio Operations, Inc.
Posted 4/24/2024

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 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 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 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

FDA Grants Orphan Drug and Rare Pediatric Disease Designations to ATSN-201 for X-Linked Retinoschisis

Rare Disease
  • Atsena Therapeutics' gene therapy ATSN-201 has received both Orphan Drug and Rare Pediatric Disease designations from the FDA for X-linked retinoschisis, a rare genetic disorder causing vision loss primarily in males.
  • ATSN-201 utilizes a novel spreading capsid (AAV.SPR) to deliver functional copies of the RS1 gene to retinal photoreceptors while minimizing risks of foveal detachment, potentially addressing the root cause of the disease.
  • Preliminary data from the Phase 1/2 LIGHTHOUSE trial has shown promising results, with two out of three patients experiencing extensive schisis resolution beginning at 8 weeks post-treatment.

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

NCT05878860RecruitingPhase 1
Atsena Therapeutics Inc.
Posted 8/22/2023

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.

Gene Therapy Pioneer Jim Wilson Departs UPenn to Launch Two New Companies

Rare Disease
  • Gene therapy pioneer Jim Wilson is leaving the University of Pennsylvania after 30 years to found two new companies, Gemma Biotherapeutics and Franklin Biolabs, spinning out the university's Gene Therapy Program.
  • Gemma Bio will focus on rare disease research and has licensed three clinical-stage gene therapy candidates from Passage Bio for pediatric conditions including GM1 gangliosidosis and Krabbe disease.
  • The move aims to accelerate gene therapy development and patient access during a biotech funding downturn, with Wilson's team having contributed to three FDA-approved AAV-based gene therapies and over 40 active programs in development.

Pfizer Discontinues Duchenne Gene Therapy Development Following Phase 3 Trial Failure

Rare Disease
  • Pfizer has officially terminated development of its experimental gene therapy fordadistrogene movaparvovec for Duchenne muscular dystrophy after disappointing Phase 3 results revealed in June.
  • The company will lay off 150 employees at its Sanford, North Carolina facility and take a $230 million impairment charge, with an additional $400 million charge expected for a related manufacturing facility.
  • The decision eliminates the near-term possibility of a second gene therapy option for Duchenne patients, leaving Sarepta Therapeutics' Elevidys as the only approved gene therapy for this progressive and fatal condition.
  • Development was previously complicated by safety concerns, including patient deaths in clinical trials that led to temporary FDA holds and protocol modifications.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.