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FDA Approves First CGRP Antagonist for Pediatric Migraine Prevention as Ajovy Expands to Children

Drug Approval
  • The FDA has approved Ajovy (fremanezumab-vfrm) for preventive treatment of episodic migraine in children and adolescents aged 6-17 years weighing at least 45 kg, making it the first CGRP antagonist approved for pediatric migraine prevention.
  • The approval addresses a significant treatment gap for pediatric migraine, which affects approximately 1 in 10 children and adolescents in the U.S. and often leads to missed school days and impaired academic performance.
  • Ajovy is administered as a 225 mg subcutaneous injection once monthly and can be given in-office or at home, with the pediatric safety profile generally consistent with adult trials.
  • The approval was supported by phase 3 data from the SPACE trial involving 237 pediatric patients, which demonstrated significant reductions in monthly migraine days compared to placebo.

A Study to Test if Fremanezumab is Effective in Preventing Episodic Migraine in Patients 6 to 17 Years of Age

NCT04458857CompletedPhase 3
Teva Branded Pharmaceutical Products R&D, Inc.
Posted 7/15/2020

Fremanezumab Compassionate Use Program for Pediatric Patients

NCT03539393available
Teva Branded Pharmaceutical Products R&D, Inc.

A Study to Confirm Safety and Efficacy of Lecanemab in Participants With Early Alzheimer's Disease

NCT03887455Active, Not RecruitingPhase 3
Eisai Inc.
Posted 3/27/2019

Dual-sided Hypoglossal neRvE stimulAtion for the treatMent of Obstructive Sleep Apnea (DREAM)

NCT03868618Active, Not RecruitingNot Applicable
Nyxoah S.A.
Posted 10/14/2020

SERB Pharmaceuticals to Acquire Y-mAbs Therapeutics for $412 Million, Expanding Rare Oncology Portfolio

Monoclonal AntibodyRare DiseaseDrug Approval
  • SERB Pharmaceuticals announced an all-cash acquisition of Y-mAbs Therapeutics for $8.60 per share, representing a 105% premium and $412 million equity value.
  • The acquisition includes DANYELZA (naxitamab-gqgk), the first FDA-approved treatment for relapsed or refractory high-risk neuroblastoma in pediatric and adult patients.
  • DANYELZA expands SERB's rare oncology portfolio and provides outpatient treatment options for neuroblastoma patients who have shown partial response, minor response, or stable disease to prior therapy.
  • The transaction is expected to close by Q4 2025, with SERB leveraging its global footprint to expand DANYELZA's reach to new markets.

Contract Manufacturing Industry Report Reveals Impact of Global Conflicts and Inflation on Pharmaceutical Production

Drug Approval
  • The 15th edition of the CMO industry analysis uses FDA NDA approvals as the primary performance indicator for contract manufacturing organizations.
  • This year's report specifically examines how inflation and increasing global conflict have impacted pharmaceutical manufacturers and their outsourcing strategies.
  • The comprehensive analysis covers outsourcing trends for New Molecular Entities, special product categories including Accelerated Approval and Orphan designations, and CDMO performance metrics.
  • Key findings include record-high biosimilar approvals, increased outsourcing of special product categories, and concentration of dose contracts among a few large CMOs.

Axogen Raises 2025 Revenue Growth Target to 17% as Avance Nerve Graft BLA Approval Approaches

Drug Approval
  • Axogen reported strong Q2 2025 results with revenue of $56.7 million, representing 18.3% growth compared to the same period last year.
  • The company has raised its 2025 revenue growth guidance to at least 17%, or $219 million for the full year, driven by anticipated regulatory approval.
  • The FDA has assigned a September 5, 2025 goal date for the Biologics License Application (BLA) of Avance Nerve Graft, with key regulatory milestones completed in Q2.
  • Axogen expects to maintain gross margins of 73-75% for the year and achieve net cash flow positive status despite one-time costs related to the anticipated BLA approval.

Health Canada Approves Dyanavel XR Extended-Release Amphetamine for ADHD Treatment in Adults and Children

Drug Approval
  • Health Canada has approved Dyanavel XR, an extended-release amphetamine formulation available as both tablets and oral suspension, for treating ADHD in adults 18 years and older and children aged 6-12 years.
  • The medication utilizes novel LiquiXR extended-release delivery technology and represents the first and only extended-release oral suspension and chewable tablet amphetamine approved in Canada.
  • Dyanavel XR demonstrates significant onset of action at one hour post-dose with a 13-hour duration of therapy, addressing the needs of ADHD patients who affect approximately 3-5% of adults and 5-9% of children and adolescents.
  • The approval expands Kye Pharmaceuticals' ADHD portfolio in Canada, providing healthcare professionals with additional treatment options for optimizing ADHD management in both pediatric and adult populations.

Sarilumab Demonstrates Significant Quality of Life Improvements in Polymyalgia Rheumatica Phase 3 Trial

Monoclonal AntibodyDrug Approval
  • A phase 3 randomized controlled trial of 118 patients showed sarilumab 200 mg every two weeks with 14-week glucocorticoid taper significantly improved quality of life measures compared to placebo with 52-week taper in relapsing polymyalgia rheumatica.
  • Patients receiving sarilumab demonstrated clinically meaningful improvements in SF-36 Physical Component Summary scores (7.65 vs 2.87, p=0.020) and Mental Component Summary scores (3.04 vs -1.71, p=0.030) at week 52.
  • More than half of sarilumab-treated patients reached normative values in several SF-36 domains, an outcome not observed in the placebo group, supporting its use when glucocorticoid monotherapy proves inadequate.

Syndax Reports Strong Q2 2025 Results with Revuforj Revenue Growth and Pending FDA Approval for NPM1 AML

Monoclonal AntibodyDrug Approval
  • Syndax Pharmaceuticals reported $28.6 million in Revuforj net revenue for Q2 2025, representing a 43% increase over the first quarter despite approximately one-third of patients pausing treatment for stem cell transplants.
  • The FDA granted Priority Review to Syndax's supplemental New Drug Application for Revuforj in relapsed/refractory mutant NPM1 acute myeloid leukemia, with a PDUFA action date of October 25, 2025.
  • Niktimvo generated $36.2 million in net revenue during its first full quarter of launch, with Syndax recording $9.4 million in collaboration revenue from its 50% profit-sharing agreement with Incyte.
  • The company maintains $517.9 million in cash and investments, which management expects will fund operations to profitability alongside growing product revenues from both approved therapies.

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564RecruitingPhase 2
Incyte Corporation
Posted 10/11/2024

MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06132256RecruitingPhase 2
Syndax Pharmaceuticals
Posted 12/11/2023

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

NCT06585774RecruitingPhase 3
Incyte Corporation
Posted 1/21/2025

Real-World Study Confirms Safety and Efficacy of Aflibercept Biosimilar Pavblu in 6,313 Patients

Real World EvidenceDrug Approval
  • A large real-world study of 7,773 eyes from 6,313 patients treated with Pavblu (aflibercept-ayyh) showed stable vision outcomes with no unexpected safety concerns across 255 locations nationwide.
  • The biosimilar demonstrated comparable efficacy to reference aflibercept in a phase 3 trial of 579 patients with neovascular age-related macular degeneration, with 95.6% maintaining vision at week 52.
  • Only 0.01% of patients experienced serious eye infections (endophthalmitis) and 0.09% had minor inflammation, confirming the biosimilar's safety profile matches the reference product.
  • Pavblu secured national insurance coverage for approximately 80% of patients as of May 2025, potentially improving access to anti-VEGF therapy while reducing healthcare costs.

A Study to Understand Effectiveness and Safety of ABP 938 Compared to Aflibercept (Eylea®) in Patients Suffering With Neovascular Age-related Macular Degeneration [Neovascular (Wet) AMD]

NCT04270747CompletedPhase 3
Amgen
Posted 6/22/2020

TG Therapeutics Reports 91% Revenue Surge Driven by BRIUMVI Multiple Sclerosis Treatment

Drug Approval
  • TG Therapeutics reported a remarkable 91% year-over-year revenue increase in Q2 2025, primarily driven by strong sales of its multiple sclerosis treatment BRIUMVI.
  • The company raised its 2025 BRIUMVI U.S. net revenue target to $570-575 million and increased global revenue projections to approximately $585 million.
  • BRIUMVI has gained regulatory approval in the EU, UK, Switzerland, and Australia, with management citing strong physician confidence and uptake.
  • CEO Michael S. Weiss highlighted the continued momentum across all business aspects, positioning BRIUMVI to redefine multiple sclerosis treatment expectations.

Agios Pharmaceuticals Faces FDA Delay for Pyrukynd Thalassemia Approval Amid Safety Concerns

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA has extended the review period for Agios Pharmaceuticals' Pyrukynd (mitapivat) in thalassemia, pushing the PDUFA date from September 7, 2025 to December 7, 2025 due to a required Risk Evaluation and Mitigation Strategy (REMS) submission.
  • The delay stems from hepatocellular injury risks requiring stringent monitoring protocols, though Agios emphasizes no new negative safety or efficacy data prompted the extension.
  • Despite the setback, Pyrukynd demonstrated statistically significant improvements in hemoglobin levels and reduced transfusion needs in Phase 3 ENERGIZE trials, targeting a $200-300 million peak annual revenue opportunity in the U.S.
  • The company maintains a strong financial position with $1.3 billion in cash through 2027, while the stock has shown volatility with a 25.78% decline but remains up 16.03% year-to-date.

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