MedPath

Tagged News

FDA Approves Hundreds of Drugs Without Adequate Evidence of Effectiveness, Investigation Reveals

Drug Approval
  • A two-year investigation found that 73% of the 429 drugs approved by the FDA from 2013-2022 failed to meet the agency's four foundational standards for proving effectiveness.
  • Cancer drugs showed particularly concerning approval patterns, with only 2.4% of 123 cancer treatments meeting all four scientific criteria and 81% approved based on preliminary surrogate outcomes rather than survival data.
  • The investigation documented cases like Elmiron, which remained on market for nearly three decades despite causing blindness and having no proven efficacy, highlighting systemic failures in post-market confirmation studies.

Formycon's Ranivisio Becomes First Lucentis Biosimilar Approved in Brazil

Drug Approval
  • Brazil's regulatory authority ANVISA has granted marketing authorization for Ranivisio (FYB201), marking the first approved Lucentis biosimilar in the country with launch expected in Q4 2025.
  • Formycon has established a commercialization partnership with Brazilian pharmaceutical company Biomm to distribute the ranibizumab biosimilar in Brazil's BRL 374 million annual anti-VEGF therapy market.
  • The approval initiates a phased Latin American rollout, with marketing authorizations already secured in Peru, El Salvador, Honduras, and the Dominican Republic, and further launches planned through early 2027.

FDA Denies Approval for Elamipretide, Forcing Stealth BioTherapeutics to Halt Production of Rare Disease Treatment

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA has denied approval for Elamipretide, a treatment for Barth Syndrome that significantly improved quality of life for patients including a 17-year-old Mississippi teen who gained the ability to walk stairs and participate in normal activities.
  • Stealth BioTherapeutics will close operations after investing $100 million in the drug's development, with investors withdrawing support following the FDA's rejection.
  • Patients with Barth Syndrome, including Christopher Pena and his cousin Jacob Wilson, will lose access to the medication by the end of August, potentially leading to health deterioration.
  • Families are advocating for intervention from new FDA Commissioner Marty Makary and other officials, arguing the denial is due to regulatory red tape rather than safety or efficacy concerns.

Daiichi Sankyo's New CEO Charts Course for ADC Expansion Beyond Enhertu Success

OncologyDrug Approval
  • Hiroyuki Okuzawa assumed leadership of Daiichi Sankyo two months ago, inheriting a company strengthened by three major pharmaceutical licensing deals worth up to $27 billion based on ADC technology.
  • Enhertu demonstrated potential as frontline therapy for advanced breast cancer at ASCO 2025, continuing the drug's track record of clinical success since 2022.
  • The company plans to have five ADCs approved across more than 30 tumor types by 2030, targeting treatment of nearly 400,000 cancer patients annually.
  • Okuzawa faces the challenge of identifying next-generation growth drivers while navigating development setbacks and potential trade policy impacts on global supply chains.

FDA Approves First Drug-Free Wearable Device for Pediatric Nasal Congestion Treatment

AIDrug Approval
  • The FDA has approved an expanded pediatric indication for the SONU Band, making it the first FDA-approved, drug-free solution for nasal congestion and allergies in children aged 12 years and older.
  • The AI-enabled wearable device uses personalized acoustic vibrational energy to reduce nasal swelling and open passages, providing relief in 15 minutes or less without medications or steroids.
  • The device received FDA De Novo authorization, establishing a new regulatory pathway for this novel medical technology that addresses significant unmet needs for safe pediatric congestion treatment alternatives.

Vadadustat Phase 3 Data Reveals Geographic Differences in CKD Anemia Treatment Outcomes

Drug Approval
  • The Journal of the American Society of Nephrology published pre-specified analyses from vadadustat's global phase 3 program, comparing U.S. and non-U.S. patient outcomes in chronic kidney disease-related anemia treatment.
  • Among dialysis-dependent CKD patients, vadadustat showed similar safety and efficacy to darbepoetin alfa both within and outside the United States.
  • For non-dialysis-dependent CKD patients in the U.S. subgroup, vadadustat demonstrated higher cardiovascular risk compared to darbepoetin alfa when used outside the United States.
  • The analyses highlight how regional differences in patient characteristics, hemoglobin targets, and healthcare practices can influence treatment outcomes in global clinical trials.

Study to Evaluate Vadadustat for the Correction of Anemia in Participants With Non-dialysis-dependent Chronic Kidney Disease

NCT02648347CompletedPhase 3
Akebia Therapeutics
Posted 12/1/2015

Efficacy and Safety Study to Evaluate Vadadustat for the Maintenance Treatment of Anemia in Participants With Dialysis-dependent Chronic Kidney Disease (DD-CKD)

NCT02892149CompletedPhase 3
Akebia Therapeutics
Posted 8/1/2016

Efficacy and Safety Study to Evaluate Vadadustat for the Maintenance Treatment of Anemia in Participants With Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD)

NCT02680574CompletedPhase 3
Akebia Therapeutics
Posted 2/1/2016

Efficacy and Safety Study to Evaluate Vadadustat for the Correction or Maintenance Treatment of Anemia in Participants With Incident Dialysis-dependent Chronic Kidney Disease (DD-CKD)

NCT02865850CompletedPhase 3
Akebia Therapeutics
Posted 7/1/2016

Fresenius Kabi Launches First Generic Iodixanol Contrast Agent in Canada, Addressing Critical Supply Shortages

Drug Approval
  • Fresenius Kabi Canada launched Iodixanol Injection 270 and 320, marking the first generic injectable iodinated contrast media available in the Canadian market.
  • Health Canada granted Notice of Compliance approval on December 17, 2024, based on pharmaceutical equivalence to reference drug Visipaque®.
  • The launch addresses critical supply shortages that previously forced healthcare agencies to ration contrast media and reduce dosages for patients.
  • Iodixanol injection was classified as a tier 3 drug shortage in June 2022, indicating significant potential impact on Canada's healthcare system.

Moderna Agrees to Placebo-Controlled Trial for New COVID-19 Vaccine Following FDA Approval

Drug Approval
  • Moderna has agreed to conduct a placebo-controlled trial of its newly approved second-generation COVID-19 vaccine mNexspike, as announced by HHS Secretary Robert F. Kennedy Jr.
  • The FDA required this future placebo-controlled study as a condition for approving the new vaccine, specifically targeting adults ages 50 to 64 without high-risk conditions.
  • The new vaccine received limited approval for adults 65 and older and anyone 12 and over with at least one risk factor for severe disease, marking a narrower approval than previous COVID-19 vaccines.
  • This requirement represents an unusual regulatory approach that has raised ethical concerns about exposing participants to preventable illness by administering placebo instead of existing vaccines.

FDA Approves Darolutamide for Metastatic Castration-Sensitive Prostate Cancer Based on ARANOTE Trial Results

Drug Approval
  • The FDA has approved darolutamide (Nubeqa) for treating patients with metastatic castration-sensitive prostate cancer, expanding its approved indications beyond the previously approved combination with docetaxel.
  • The approval was based on the phase 3 ARANOTE trial, which demonstrated that darolutamide significantly improved radiographic progression-free survival compared to placebo when combined with androgen deprivation therapy.
  • In the 669-patient study, median radiographic progression-free survival was not reached with darolutamide versus 25.0 months with placebo, representing a 46% reduction in disease progression risk.
  • The safety profile remained consistent with previous darolutamide studies, with hypertension and anemia being the most common grade 3/4 adverse events observed in the treatment arm.

Darolutamide in Addition to Standard Androgen Deprivation Therapy and Docetaxel in Metastatic Hormone-Sensitive Prostate Cancer

NCT02799602CompletedPhase 3
Bayer
Posted 11/30/2016

A Phase 3 Single-Arm Study of UGN-102 for Treatment of Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

NCT05243550Active, Not RecruitingPhase 3
UroGen Pharma Ltd.
Posted 3/1/2022

Adding Mitomycin to BCG as Adjuvant Intravesical Therapy for High-risk Non-Muscle-invasive Bladder Cancer

NCT02948543Active, Not RecruitingPhase 3
University of Sydney
Posted 7/1/2013

A Clinical Study of KTX-2001 in Subjects With Metastatic Castration-Resistant Prostate Cancer (STRIKE-001)

NCT07103018Not Yet RecruitingNot Applicable
K36 Therapeutics, Inc.
Posted 9/1/2025

Study of DS-1062a Versus Docetaxel in Previously Treated Advanced or Metastatic Non-small Cell Lung Cancer With or Without Actionable Genomic Alterations (TROPION-LUNG01)

NCT04656652Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/21/2020

Darolutamide in Addition to ADT Versus ADT in Metastatic Hormone-sensitive Prostate Cancer

NCT04736199Active, Not RecruitingPhase 3
Bayer
Posted 2/23/2021

A Phase 3 Study of UGN-102 for Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer

NCT04688931TerminatedPhase 3
UroGen Pharma Ltd.
Posted 2/19/2021

A Study Comparing Obinutuzumab and BGB-3111 Versus Obinutuzumab Alone in Treating R/R Follicular Lymphoma

NCT03332017CompletedPhase 2
BeiGene
Posted 11/14/2017

Efficacy and Safety Study of Darolutamide (ODM-201) in Men With High-risk Non-metastatic Castration-resistant Prostate Cancer

NCT02200614CompletedPhase 3
Bayer
Posted 9/12/2014

A Study of AB-106 in Subjects With Advanced NSCLC Harboring ROS1 Fusion Gene

NCT04395677Active, Not RecruitingPhase 2
AnHeart Therapeutics Inc.
Posted 7/7/2020

Study of Pembrolizumab Given Prior to Surgery and in Combination With Radiotherapy Given Post-surgery for Advanced Head and Neck Squamous Cell Carcinoma (MK-3475-689)

NCT03765918Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 12/17/2018

A Phase 3 Study to Assess Efficacy and Safety of Tafasitamab Plus Lenalidomide and Rituximab Compared to Placebo Plus Lenalidomide and Rituximab in Patients With Relapsed/Refractory (R/R) Follicular Lymphoma or Marginal Zone Lymphoma.

NCT04680052Active, Not RecruitingPhase 3
Incyte Corporation
Posted 4/15/2021

A Study to Compare Darolutamide Given With Androgen Deprivation Therapy (ADT) With ADT in Men With Hormone Sensitive Prostate Cancer and Raise of Prostate Specific Antigen (PSA) Levels After Local Therapies

NCT05794906RecruitingPhase 3
Bayer
Posted 4/3/2023

A Phase III Study for Patients With Metastatic Hormone-naïve Prostate Cancer

NCT01957436Active, Not RecruitingPhase 3
UNICANCER
Posted 11/13/2013

Taletrectinib Phase 2 Global Study in ROS1 Positive NSCLC

NCT04919811RecruitingPhase 2
Nuvation Bio Inc.
Posted 9/1/2021

Study of DS-1062a in Advanced or Metastatic Non-small Cell Lung Cancer With Actionable Genomic Alterations (TROPION-Lung05)

NCT04484142Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 3/30/2021

A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma

NCT05651932RecruitingPhase 1
K36 Therapeutics, Inc.
Posted 2/22/2023

Biocon Receives CDSCO Approval for Liraglutide Diabetes Drug in India

Drug Approval
  • Biocon has received regulatory approval from India's Central Drugs Standard Control Organisation (CDSCO) for its diabetes drug liraglutide, marking a significant milestone for the company's pharmaceutical portfolio.
  • The approval enables Biocon to market and distribute liraglutide in the Indian market, expanding treatment options for diabetes patients in the country.
  • Following the regulatory clearance, Biocon shares have come into focus among investors, reflecting market confidence in the company's drug development capabilities.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.