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Spain Dramatically Reduces Timeframe Between EU Drug Approval and Reimbursement Decisions

Drug Approval
  • Spain has significantly shortened the waiting period between European drug authorization and national financing decisions, improving patient access to new medications.
  • Spanish health authorities emphasize that pharmaceutical companies can further accelerate the reimbursement process by submitting more comprehensive and well-prepared applications.
  • The streamlined approval pathway represents Spain's commitment to modernizing its healthcare system while balancing innovation access with fiscal sustainability.

Tempus and Verastem Partner to Develop Companion Diagnostic for First FDA-Approved KRAS-Mutant LGSOC Treatment

Precision MedicineDrug ApprovalOncologyTargeted Therapy
  • Tempus AI and Verastem Oncology have announced a collaboration to develop a companion diagnostic test for the recently FDA-approved combination therapy of avutometinib and defactinib for KRAS-mutated recurrent low-grade serous ovarian cancer.
  • The partnership builds on Tempus' confirmatory testing in Verastem's Phase 2 RAMP-201 clinical trial, with their FDA-approved xT CDx assay now being used as an investigational assay in the global Phase 3 RAMP-301 trial.
  • Low-grade serous ovarian cancer (LGSOC) is a rare form affecting younger women, accounting for 6-10% of serous ovarian cancers, with historically poor response to chemotherapy and limited treatment options.

FDA Approves Teal Wand: First At-Home HPV Self-Collection Device for Cervical Cancer Screening

Drug Approval
  • Teal Health's Wand device has received FDA approval as the first prescription device for at-home self-collection of vaginal samples for HPV-based cervical cancer screening.
  • The pivotal SELF-CERV study published in JAMA Network Open demonstrated 95% positive agreement for high-risk HPV detection and 96% sensitivity for precancerous lesions, matching clinician-collected samples.
  • With an estimated 21 million women under- or unscreened for cervical cancer in the US, the Teal Wand aims to increase screening rates by offering a preferred at-home alternative without compromising clinical accuracy.

New Guidelines and Therapies Reshape Acne Treatment Approach in 2025

Drug Approval
  • The American Academy of Dermatology's 2024 updated acne guidelines recommend limiting systemic antibiotic use and emphasize treating all four pillars of acne pathogenesis simultaneously.
  • Several new FDA-approved acne medications since 2019 have expanded treatment options, including Winlevi (clascoterone), the only topical therapy effective for excessive sebum production.
  • Dermatology experts now recommend simpler treatment regimens with fewer products that address multiple pathogenic factors, with a growing focus on early intervention to prevent scarring.

FDA's Approval of MRD as End Point Transforms Multiple Myeloma Research and Treatment

Drug ApprovalMonoclonal AntibodyOncology
  • The FDA's Oncology Drugs Advisory Committee unanimously approved minimal residual disease (MRD) as an end point for accelerated approval of multiple myeloma therapies in April 2024, potentially reducing trial timelines from 10-15 years to just 3 years.
  • Pharmaceutical companies have rapidly adapted by implementing MRD as a coprimary end point in new trials and amending existing protocols, with the CEPHEUS trial being the first major study to read out with MRD as a coprimary endpoint.
  • Researchers are now exploring MRD applications beyond drug approval, including using MRD status to guide treatment decisions, developing improved blood-based detection technologies, and expanding the approach to other hematologic malignancies.

Daratumumab, VELCADE (Bortezomib), Lenalidomide and Dexamethasone Compared to VELCADE, Lenalidomide and Dexamethasone in Subjects With Previously Untreated Multiple Myeloma

NCT03710603Active, Not RecruitingPhase 3
Stichting European Myeloma Network
Posted 12/14/2018

A Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) With VELCADE, Lenalidomide, and Dexamethasone (VRd) in Participants With Untreated Multiple Myeloma and for Whom Hematopoietic Stem Cell Transplant is Not Planned as Initial Therapy

NCT03652064Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 11/6/2018

A Study of Subcutaneous Daratumumab Versus Active Monitoring in Participants With High-Risk Smoldering Multiple Myeloma

NCT03301220Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 11/7/2017

A Study of Daratumumab, Carfilzomib, Lenalidomide, and Dexamethasone in Patients With Newly-Diagnosed Multiple Myeloma

NCT04268498Active, Not RecruitingPhase 2
University of Miami
Posted 2/11/2020

Ionis Reports Positive Phase 3 Results for Olezarsen in Hypertriglyceridemia

Drug Approval
  • Olezarsen demonstrated significant triglyceride reductions of 61% and 58% at 6 months with 80mg and 50mg monthly doses respectively in patients with moderate hypertriglyceridemia.
  • The Phase 3 Essence study met all primary and secondary endpoints, with most participants achieving triglyceride levels within normal range despite already being on standard lipid-lowering therapies.
  • Following recent FDA approval of olezarsen (Tryngolza) for familial chylomicronemia syndrome, these results support potential expanded indication for severe hypertriglyceridemia pending upcoming CORE and CORE2 trial data.

A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia

NCT05610280CompletedPhase 3
Ionis Pharmaceuticals, Inc.
Posted 11/21/2022

Ensifentrine Shows Promise as Novel Monotherapy and Add-On Treatment for COPD

Drug Approval
  • New data from the Phase 3 ENHANCE trials demonstrates that ensifentrine, a dual phosphodiesterase 3 and 4 inhibitor, significantly improves lung function and reduces exacerbations in COPD patients.
  • Ensifentrine monotherapy showed meaningful improvements in patient-reported outcomes including dyspnea, COPD symptoms, and health-related quality of life compared to placebo in patients not on long-acting medications.
  • As the first nebulized non-steroidal therapy with a novel dual mechanism of action, ensifentrine represents an important addition to the COPD treatment arsenal, offering benefits both as first-line and add-on therapy.

A Phase 3 Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04542057CompletedPhase 3
Verona Pharma plc
Posted 9/22/2020

A Phase 3 Clinical Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

NCT04535986CompletedPhase 3
Verona Pharma plc
Posted 9/29/2020

Dose Ranging Study of RPL554 in Chronic Obstructive Pulmonary Disease (COPD) Patients

NCT03443414CompletedPhase 2
Verona Pharma plc
Posted 6/1/2017

FDA Expands Jivi Approval for Hemophilia A Treatment in Children Ages 7-12

Drug Approval
  • The FDA has approved Bayer's Jivi (antihemophilic factor recombinant, PEGylated-aucl) for pediatric patients aged 7 to 12 years with hemophilia A, expanding treatment options for this age group.
  • The approval was based on data from the Alfa-PROTECT and PROTECT Kids studies, which demonstrated the safety and efficacy of Jivi in pediatric patients with severe hemophilia A.
  • Jivi was previously approved in 2018 for adults and adolescents aged 12 and older, and can be used for on-demand treatment, perioperative management, and routine prophylaxis to reduce bleeding episodes.

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

NCT01775618CompletedPhase 3
Bayer
Posted 5/29/2013

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT05147662CompletedPhase 3
Bayer
Posted 3/23/2022

AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis

Orphan DrugDrug ApprovalRare Disease
  • 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.

FDA Approves 18 New Personalized Medicines in 2024, Marking Significant Shift in Treatment Paradigm

Drug ApprovalOncologyRare DiseaseAIReal World EvidencePrecision Medicine
  • The FDA approved 18 new personalized medicines in 2024, representing 38% of all newly approved therapeutic molecular entities across multiple treatment areas including cancer and Alzheimer's disease.
  • Six new gene and cell-based therapies for rare genetic diseases and cancers were authorized, alongside expanded indications for 11 diagnostic testing systems and the first-ever expanded indication for an approved gene therapy.
  • Personalized medicines now constitute at least 25% of drug approvals for the past decade, a substantial increase from less than 10% ten years ago, demonstrating the healthcare system's shift away from one-size-fits-all approaches.

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