Tagged News
PharmaEssentia's Ropeginterferon Shows Promising Results in Phase 3 Trial for Essential Thrombocythemia
- PharmaEssentia will present positive Phase 3 SURPASS-ET trial results showing ropeginterferon alfa-2b-njft achieved significantly higher durable clinical response rates compared to anagrelide (42.9% vs. 6.0%; p=0.0001) in essential thrombocythemia patients.
- The trial demonstrated not only effective blood count control but also a measurable reduction in JAK2 mutation allele burden over 12 months, addressing an underlying disease mechanism that current treatments fail to target.
- Ropeginterferon alfa-2b-njft (marketed as BESREMi® for polycythemia vera) could potentially offer a new second-line treatment option for essential thrombocythemia, a rare blood disorder characterized by excessive platelet production.
Highlighted Clinical Trials:
PharmaEssentia
Posted 8/25/2020
CHOP Researchers Develop Breakthrough AAV Vectors for Brain Gene Therapy at Lower Doses
- Researchers at Children's Hospital of Philadelphia have engineered new adeno-associated viral (AAV) vectors that can target brain cells at significantly lower doses than current therapies, potentially improving safety and reducing costs.
- Two novel capsids were identified: AAV-Ep+ for Batten disease treatment and AAV-DB-3 for Huntington's disease, both showing robust efficacy in preclinical models and human neurons derived from stem cells.
- The breakthrough could transform treatment for neurodegenerative disorders by enabling one-time precision therapies that require lower doses while maintaining therapeutic efficacy, with potential applications for other inherited disorders.
FDA Upgrades Xinnate's TCP-25 Trial for Epidermolysis Bullosa to Registrational Status
- Following a successful Type C meeting with the FDA, Xinnate's upcoming STEP trial for TCP-25 in Epidermolysis Bullosa patients has been upgraded to serve as a registrational trial.
- TCP-25, an immunomodulatory peptide with dual-action capabilities targeting both inflammation and bacterial infection, previously received Orphan Drug Designation from the FDA for EB treatment.
- The STEP trial, planned to begin in 2025, is designed as an international multicenter, randomized, double-blind, placebo-controlled study to assess TCP-25's efficacy and safety in dystrophic and junctional EB patients.
Real-World Data Confirms Trikafta's Long-Term Efficacy as Gold Standard in Cystic Fibrosis Treatment
- New real-world evidence presented at ATS 2025 validates Trikafta (elexacaftor/tezacaftor/ivacaftor) as an effective long-term therapy for cystic fibrosis patients with at least one F508del mutation.
- The study by Sutharsan and colleagues provides valuable insights into laboratory markers while demonstrating sustained clinical benefits across various outcomes in this patient population.
- These findings further establish Trikafta's position as the gold standard treatment in long-term cystic fibrosis care, reinforcing its therapeutic value for patients.
FDA Grants Orphan Drug Designation to Repair Biotechnologies' mRNA Therapy for Rare Cholesterol Disorder
- The FDA has granted Orphan Drug Designation to REP-0003, a novel mRNA therapy developed by Repair Biotechnologies for treating homozygous familial hypercholesterolemia (HoFH), a rare condition causing accelerated atherosclerosis.
- REP-0003 works by selectively clearing harmful excess free cholesterol inside cells while preserving essential cholesterol, showing promising results in preclinical studies including plaque regression and improved liver function.
- The designation provides Repair Biotechnologies with seven years of market exclusivity upon approval, tax credits for clinical trials, waived FDA fees, and potential fast-track pathways as they prepare for first human trials in 2026.
Prime Medicine Reports Breakthrough Clinical Data for First Prime Editing Therapy in Chronic Granulomatous Disease
- Prime Medicine's PM359, the first Prime Editing therapy administered to humans, demonstrated rapid restoration of NADPH oxidase activity in a patient with Chronic Granulomatous Disease, exceeding therapeutic thresholds.
- A single infusion of PM359 achieved 66% DHR positivity by Day 30, significantly above the 20% threshold believed to be potentially curative, with faster engraftment than existing gene editing technologies.
- The therapy showed an encouraging safety profile with no serious adverse events related to PM359, marking a significant milestone for Prime Editing technology as a potential one-time curative treatment for genetic diseases.
Ruxoprubart Shows Promising Phase II Results as Novel Monotherapy for Paroxysmal Nocturnal Hemoglobinuria
- NovelMed's Ruxoprubart demonstrated significant efficacy in a Phase II trial for treatment-naïve PNH patients, meeting all primary endpoints including complete transfusion avoidance and increased hemoglobin levels.
- The drug's selective inhibition of the Alternative Pathway without affecting the Classical Pathway offers a potentially safer profile than existing treatments, which often carry Black Box warnings for infection risk.
- With FDA Orphan Drug Designation already secured and plans to file for Breakthrough Therapy Designation, Ruxoprubart is positioned as a potential best-in-class therapy for PNH and other complement-mediated disorders.
EMA Designates Allopurinol as First Orphan Drug for Marfan Syndrome
- The European Medicines Agency has designated allopurinol as the first orphan drug for Marfan syndrome, a rare connective tissue disease affecting approximately 7 in 100,000 people in the European Union.
- Researchers from the University of Barcelona, IDIBAPS, and CIBERER have demonstrated allopurinol's potential to halt and prevent aortic aneurysms in animal models, with international clinical trials in patients planned for the future.
- This repurposing of allopurinol, currently used for gout treatment, represents a significant advancement for Marfan syndrome patients who currently have no curative options beyond limited palliative treatments and high-risk surgical interventions.
Taiwan Approves Chugai's PiaSky as First Subcutaneous Treatment for Paroxysmal Nocturnal Hemoglobinuria
- Taiwan FDA has granted orphan drug approval for PiaSky, making it the first subcutaneous treatment for paroxysmal nocturnal hemoglobinuria (PNH) available in Taiwan for patients 13 years and older.
- PiaSky, developed with Chugai's proprietary Recycling Antibody technology, allows for convenient subcutaneous administration every 4 weeks, significantly reducing treatment burden compared to existing biweekly intravenous options.
- The approval was based on positive results from three Phase III clinical trials, including COMMODORE 2, which demonstrated efficacy in transfusion avoidance and control of hemolysis compared to eculizumab.
AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis
- SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
- The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
- Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.