MedPath

Tagged News

Philippines FDA Expands VAT-Free Medicine List to Include New Cancer, Diabetes, and Cardiovascular Treatments

Drug Approval
  • The Philippines FDA has expanded its VAT-exempt medicine list to include additional treatments for cancer, diabetes, hypertension, and other conditions under President Marcos's directive to reduce medicine costs.
  • New cancer medications include Tegafur combination capsules and established treatments like Bortezomib, Docetaxel, Lenvatinib, Lenalidomide, and Paclitaxel.
  • Diabetes treatments now include Metformin-Teneligliptin combinations and Saxagliptin-Dapagliflozin tablets, while cardiovascular medicines feature new combination therapies.
  • The expansion covers 10 additional medications across multiple therapeutic areas including mental health treatments with Lamotrigine formulations.

Revolutionary Gene Therapy and BBB-Penetrating Treatments Transform Hunter Syndrome Treatment Landscape

Rare DiseaseDrug Approval
  • RGX-121, a one-time gene therapy using AAV vector technology, is expected to receive regulatory approval in the second half of 2025, potentially offering lifelong benefits from a single infusion for Hunter Syndrome patients.
  • Tividenofusp alfa and Verenafusp alfa represent breakthrough therapies designed to cross the blood-brain barrier, addressing both CNS and somatic symptoms that current treatments like Elaprase cannot reach.
  • The Hunter Syndrome treatment paradigm is shifting from weekly enzyme replacement therapy costing over $500,000 annually to potentially curative single-dose treatments targeting neurological decline.
  • IZCARGO, launched in Japan in 2021, currently stands as the only approved therapy addressing both somatic and CNS symptoms by crossing the blood-brain barrier, though availability remains limited to the Japanese market.

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324WithdrawnPhase 1
Homology Medicines, Inc
Posted 9/8/2022

FDA Approves Moderna's Next-Generation COVID-19 Vaccine mRNA-1283 with Enhanced Efficacy

Drug Approval
  • The FDA has approved Moderna's new COVID-19 vaccine mRNA-1283 (mNEXSPIKE), marking the first authorization under updated federal guidelines prioritizing vaccination for adults 65 and older.
  • In a Phase 3 trial of over 11,000 participants, mRNA-1283 demonstrated 9.3% higher relative vaccine efficacy compared to Moderna's original vaccine, with 13.5% increased efficacy in adults 65 and older.
  • The vaccine showed a comparable safety profile with fewer local side effects and will be available for the 2025-2026 respiratory virus season in the U.S.
  • COVID-19 vaccination remains available for children 6 months and older through shared clinical decision-making, reflecting ongoing federal adjustments to immunization recommendations.

FDA Accepts Kura Oncology's NDA for Ziftomenib in NPM1-Mutant AML with Priority Review

Drug Approval
  • The FDA has accepted Kura Oncology's new drug application for ziftomenib, an oral menin inhibitor for treating relapsed or refractory acute myeloid leukemia with NPM1 mutations.
  • The agency granted priority review with a decision expected by November 30, 2025, potentially making ziftomenib the first approved menin inhibitor for this indication.
  • The NDA was based on positive results from the phase II KOMET-001 study, which met its primary endpoint of complete remission with statistical significance.
  • Kura Oncology's stock surged 15.5% following the FDA acceptance, reflecting investor confidence in the drug's approval prospects.

FDA Grants Phathom Pharmaceuticals 10-Year Market Exclusivity for VOQUEZNA Through 2032

Drug Approval
  • The FDA has approved Phathom Pharmaceuticals' Citizen Petition and will correct the Orange Book to recognize 10 years of New Chemical Entity exclusivity for VOQUEZNA (vonoprazan) tablets, extending through May 3, 2032.
  • VOQUEZNA is a first-in-class potassium-competitive acid blocker (PCAB) currently marketed for treating heartburn associated with Non-Erosive GERD, healing and maintenance of Erosive GERD, and H. pylori infection in adults.
  • The regulatory exclusivity provides Phathom with significant market protection for their gastrointestinal disease treatment portfolio, including VOQUEZNA TRIPLE PAK and DUAL PAK formulations.
  • This FDA decision strengthens Phathom's competitive position in the acid-blocking therapy market and supports their commercialization strategy for vonoprazan-based treatments.

Aytu BioPharma Secures Exclusive Rights to First-in-Class Antidepressant EXXUA for $22B US Market

Drug Approval
  • Aytu BioPharma has secured exclusive US commercialization rights for EXXUA (gepirone), the first FDA-approved selective serotonin 5HT1a receptor agonist for major depressive disorder in adults.
  • EXXUA demonstrated significant improvement in depression symptoms across clinical trials involving over 5,000 patients, with sexual side effects comparable to placebo rather than typical antidepressants.
  • The company plans to launch EXXUA in Q4 2025 to compete in the over $22 billion US prescription MDD market, targeting 21 million affected Americans.
  • The transaction was financed by healthcare-focused institutional investors including Nantahala Capital Management and Stonepine Capital Management.

Aytu BioPharma Raises $16.6M to Launch First-in-Class Depression Treatment EXXUA

Drug Approval
  • Aytu BioPharma completed an upsized public offering raising $16.6 million to commercialize EXXUA, the first FDA-approved selective serotonin 5HT1a receptor agonist for major depressive disorder.
  • EXXUA represents a breakthrough in depression treatment as it demonstrates comparable sexual side effects to placebo, addressing a major limitation of current antidepressants.
  • The company plans to launch EXXUA in Q4 2025 to compete in the $22 billion U.S. prescription major depressive disorder market.
  • Clinical trials involving over 5,000 patients showed significant improvement in depression symptoms with EXXUA's novel mechanism of action.

Nemluvio Shows Sustained Long-Term Efficacy in Atopic Dermatitis with Two-Year Extension Data

Monoclonal AntibodyDrug Approval
  • New two-year data from the ARCADIA long-term extension study demonstrate that Nemluvio (nemolizumab) maintains sustained and increased improvements in itch, skin lesions, sleep, and quality of life in patients with moderate-to-severe atopic dermatitis.
  • At week 104, more than 85% of evaluable patients achieved a 75% reduction in the Eczema Area and Severity Index (EASI), with approximately 85% achieving at least four-point improvement in itch scores.
  • The study reinforces Nemluvio's rapid onset of action at week 4 and demonstrates a consistent safety profile with no new safety signals identified during prolonged treatment up to two years.

Efficacy & Safety of Nemolizumab in Subjects With Moderate-to-Severe Atopic Dermatitis

NCT03989349CompletedPhase 3
Galderma R&D
Posted 6/30/2019

A Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04501679CompletedPhase 3
Galderma R&D
Posted 8/11/2020

Study to Assess the Efficacy and Safety of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04501666CompletedPhase 3
Galderma R&D
Posted 9/11/2020

A Long-term Study of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04204616Active, Not RecruitingPhase 3
Galderma R&D
Posted 1/11/2021

Long-term Safety and Efficacy of Nemolizumab With Moderate-to-severe Atopic Dermatitis

NCT03989206Active, Not RecruitingPhase 3
Galderma R&D
Posted 12/30/2019

Efficacy and Safety of Nemolizumab in Subjects With Moderate-to-Severe Atopic Dermatitis

NCT03985943CompletedPhase 3
Galderma R&D
Posted 6/27/2019

Foldax Receives World's First Approval for Polymer Heart Valve in India

Drug Approval
  • Foldax's TRIA Mitral Valve becomes the first commercial polymer heart valve approved anywhere in the world, receiving regulatory clearance from India's Central Drugs Standard Control Organization.
  • The valve is made from proprietary LifePolymer material engineered to be calcium-resistant and biocompatible, addressing durability limitations of traditional tissue valves and anticoagulation requirements of mechanical valves.
  • Clinical research demonstrates favorable safety, stable hemodynamics, and meaningful improvements in patient quality of life, with particular benefits for younger patients and women of childbearing age.
  • The approval marks a potential paradigm shift in heart valve therapy, with Dolphin Life Science India LLP set to manufacture the device locally.

Adolescent Obesity Medication Prescriptions Surge 301% Despite Persistent Access Barriers

Drug Approval
  • Obesity medication prescriptions among US adolescents aged 12-17 years increased by 301.7% between 2020 and 2023, driven primarily by FDA approvals of semaglutide and phentermine-topiramate for pediatric use.
  • Despite the dramatic increase, only 0.5% of adolescents with obesity received medication prescriptions in 2023, highlighting significant treatment gaps in this population.
  • Substantial disparities emerged in prescribing patterns, with Black adolescents receiving medications at lower rates than White adolescents despite having higher rates of severe obesity.
  • Access barriers including high out-of-pocket costs, insurance coverage limitations, and provider knowledge gaps continue to limit widespread adoption of evidence-based obesity treatments.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.