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FDA Approves AJOVY as First CGRP Antagonist for Pediatric Migraine Prevention

FDA ApprovalDrug Approval
  • Teva Pharmaceutical Industries received FDA approval for AJOVY as the first CGRP antagonist indicated for preventive treatment of episodic migraine in children and adolescents aged 6-17 weighing at least 45 kilograms.
  • This approval expands Teva's neuroscience portfolio and provides a new treatment pathway for a pediatric population with previously limited preventive migraine options.
  • The approval supports Teva's Pivot to Growth strategy and contributed to the company raising its 2025 revenue guidance to $16.8-17.2 billion.
  • Analysts project Teva will reach $17.9 billion in revenue and $1.5 billion in earnings by 2028, representing a 2.6% annual revenue growth rate.

Iovance Biotherapeutics Cuts Workforce by Nearly 20% Following Slow Amtagvi Cell Therapy Launch

Drug Approval
  • Iovance Biotherapeutics is reducing its workforce by less than 20% in a strategic restructuring following disappointing commercial performance of its TIL cell therapy Amtagvi.
  • The company slashed its 2024 revenue forecast by 40% in May, from $450-475 million to $250-300 million, citing challenging launch dynamics for the melanoma treatment.
  • Amtagvi, the first marketed tumor-infiltrating lymphocyte therapy priced at $515,000, represents a breakthrough in cellular medicine but faces adoption challenges due to complex manufacturing and logistics.
  • The restructuring aims to extend Iovance's cash runway while the company pursues potential approvals in Europe, UK, and Canada, plus late-stage lung cancer trials.

NICE Recommends First NHS Treatment for Rare Eye Disease LHON, Ending Access Disparity in England

Drug ApprovalRare Disease
  • NICE has recommended idebenone (Raxone) as the first NHS treatment for Leber's hereditary optic neuropathy (LHON) in England, benefiting an estimated 250 eligible patients aged 12 and over.
  • The approval ends a disparity in UK access, as the drug has been available in Scotland, Wales, and Northern Ireland for several years, with NHS England set to provide treatment within three months.
  • Clinical evidence from RHODOS, LEROS, and PAROS trials demonstrates that idebenone can improve vision and reduce eyesight deterioration by restoring cellular energy production in affected eye cells.
  • LHON primarily affects young men and boys, causing rapid progression from painless central vision blurring to potential blindness in both eyes within months, with devastating impacts on independence and employment prospects.

Valneva Overcomes FDA Safety Pause for IXCHIQ Chikungunya Vaccine, Strengthens Market Position

Drug Approval
  • The FDA temporarily paused IXCHIQ vaccine use in adults aged 60 and older in May 2025 after 17 serious adverse events globally, but lifted restrictions by August 2025 following safety review and updated labeling.
  • Valneva demonstrated regulatory agility by collaborating with authorities to refine safety data, securing approvals in Brazil and maintaining European market access through EMA approval.
  • The company achieved strong financial performance with Q1 2025 product sales surging 50.3% to €48.6 million while reducing operating cash burn by 67% in 2024.
  • Valneva's diversified pipeline includes VLA15 Lyme disease vaccine in Phase 3 trials with Pfizer partnership and S4V2 Shigella vaccine with FDA Fast Track designation.

Protagonist Therapeutics Files First NDA for Icotrokinra in Psoriasis, Advances Rusfertide for Polycythemia Vera

Drug ApprovalRare Disease
  • Protagonist Therapeutics submitted its first New Drug Application (NDA) for icotrokinra to treat moderate to severe plaque psoriasis in adults and adolescents 12 years and older in July 2025.
  • The company presented positive Phase 3 VERIFY trial data for rusfertide in polycythemia vera at ASCO's plenary session, with a U.S. NDA filing expected in Q4 2025.
  • Protagonist reported $673 million in cash and equivalents as of June 30, 2025, providing runway through at least the end of 2028, despite a Q2 net loss of $34.8 million.
  • The company is advancing early-stage assets including PN-881, a first-in-class oral IL-17 peptide antagonist, and PN-477, a GLP-1/GIP/GCG triple agonist for obesity treatment.

FDA Grants First Accelerated Approval for H3 K27M-Mutated Diffuse Midline Glioma with Dordaviprone

Drug ApprovalOrphan DrugRare Disease
  • The FDA has granted accelerated approval to dordaviprone (Modeyso) for adult and pediatric patients aged 1 year and older with H3 K27M-mutated diffuse midline glioma following disease progression after prior therapy.
  • This represents the first FDA approval of a systemic therapy specifically for H3 K27M-mutant diffuse midline glioma, addressing a significant unmet medical need.
  • Clinical trials demonstrated a 22% overall response rate with a median duration of response of 10.3 months among 50 patients with recurrent disease.
  • The protease activator received multiple FDA designations including Priority Review, Orphan Drug, Rare Pediatric Disease, and Fast Track status.

Study of Pemetrexed+Platinum Chemotherapy With or Without Pembrolizumab (MK-3475) in Participants With First Line Metastatic Nonsquamous Non-small Cell Lung Cancer (MK-3475-189/KEYNOTE-189)

NCT02578680CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 1/15/2016

A Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib in Participants With Non-Cystic Fibrosis Bronchiectasis

NCT04594369CompletedPhase 3
Insmed Incorporated
Posted 12/1/2020

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)

NCT05580562RecruitingPhase 3
Chimerix
Posted 1/23/2023

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

NCT05139810CompletedPhase 3
Ionis Pharmaceuticals, Inc.
Posted 12/3/2021

ONC201 in Pediatric H3 K27M Gliomas

NCT03416530TerminatedPhase 1
Chimerix
Posted 1/25/2018

Oral ONC201 in Adult Recurrent Glioblastoma

NCT02525692TerminatedPhase 2
Chimerix
Posted 1/1/2016

Beamion LUNG-2: A Study to Test Whether Zongertinib (BI 1810631) Helps People With Advanced Non-small Cell Lung Cancer With HER2 Mutations Compared With Standard Treatment

NCT06151574RecruitingPhase 3
Boehringer Ingelheim
Posted 8/4/2022

Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

NCT04562766Active, Not RecruitingPhase 3
Principia Biopharma, a Sanofi Company
Posted 12/14/2020

Adjuvant PRGN-2012 in Adult Patients With Recurrent Respiratory Papillomatosis

NCT04724980Active, Not RecruitingPhase 1
Precigen, Inc
Posted 3/16/2021

Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas

NCT03134131No Longer Available
Chimerix

Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas

NCT05392374No Longer Available
Chimerix

Safety, Efficacy, and Pharmacokinetic Behavior of Leuprolide Mesylate (LMIS 25 mg) in Subjects With Prostate Cancer

NCT03261999CompletedPhase 3
Foresee Pharmaceuticals Co., Ltd.
Posted 9/26/2017

Beamion LUNG-1: A Study to Test Different Doses of Zongertinib in People With Different Types of Advanced Cancer (Solid Tumours With Changes in the HER2 Gene)

NCT04886804RecruitingPhase 1
Boehringer Ingelheim
Posted 7/2/2021

ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

NCT03295396TerminatedPhase 2
Chimerix
Posted 10/30/2017

Insmed Reports Strong Q2 2025 Results with ARIKAYCE Growth and Brensocatib Approval Pending

Drug Approval
  • Insmed achieved $107.4 million in ARIKAYCE revenue for Q2 2025, representing 19% growth compared to the same quarter in 2024, with growth across all geographic regions.
  • The company's brensocatib NDA for bronchiectasis remains on track with a PDUFA target action date of August 12, 2025, following FDA Priority Review designation.
  • TPIP program advances with positive Phase 2b PAH study results that surpassed expectations, with Phase 3 studies planned for PH-ILD in H2 2025 and PAH in early 2026.
  • The company maintains 2025 global ARIKAYCE revenue guidance of $405-425 million, reflecting double-digit growth, while holding $1.9 billion in cash and marketable securities.

FDA Approves Tocilizumab-anoh Biosimilar for Cytokine Release Syndrome Treatment

Monoclonal AntibodyDrug Approval
  • The FDA approved tocilizumab-anoh (Avtozma) IV formulation on August 6, 2025, for treating cytokine release syndrome in patients aged 2 years and older.
  • This approval achieves full indication alignment between the biosimilar and its reference drug tocilizumab (Actemra), expanding access to high-quality biologics.
  • Cytokine release syndrome is a potentially life-threatening condition causing widespread inflammation and organ damage through excessive cytokine release.
  • The IV formulation is expected to be available in the United States on August 31, 2025, following a patent settlement agreement with Genentech.

A Study to Compare Efficacy and Safety of CT-P47 and RoActemra in Patients With Rheumatoid Arthritis

NCT05489224CompletedPhase 3
Celltrion
Posted 9/14/2022

Lantheus Secures FDA Acceptance for Enhanced PSMA PET Imaging Agent with 50% Increased Batch Size

Drug Approval
  • Lantheus Holdings announced FDA acceptance of a New Drug Application for an enhanced formulation of its PSMA PET imaging agent piflufolastat F 18, with a PDUFA date set for March 6, 2026.
  • The new formulation is designed to increase batch size by approximately 50% and enhance supply resilience, potentially expanding patient access to PSMA PET imaging for prostate cancer diagnosis.
  • The enhanced formulation builds on the success of PYLARIFY, which has demonstrated an 86% median true-positive rate in patients with recurrent prostate cancer and has been used in over 500,000 scans across the United States.
  • Prostate cancer represents a significant clinical burden as the second most frequently diagnosed cancer in U.S. men, with nearly 315,000 new cases and over 35,000 deaths projected for 2025.

Study of 18F-DCFPyL PET/CT Imaging in Patients With Prostate Cancer

NCT02981368CompletedPhase 2
Progenics Pharmaceuticals, Inc.
Posted 11/1/2016

Study of 18F-DCFPyL PET/CT Imaging in Patients With Suspected Recurrence of Prostate Cancer

NCT03739684CompletedPhase 3
Progenics Pharmaceuticals, Inc.
Posted 11/27/2018

A Study of HLD-0915 in Patients With Metastatic Castration Resistant Prostate Cancer (mCRPC)

NCT06800313RecruitingPhase 1
Halda Therapeutics OpCo, Inc.
Posted 2/24/2025

A Study Evaluating Efficacy and Safety of Gepotidacin Compared With Ceftriaxone Plus Azithromycin in the Treatment of Uncomplicated Urogenital Gonorrhea

NCT04010539CompletedPhase 3
GlaxoSmithKline
Posted 10/21/2019

Bracco Imaging Receives NMPA Approval for SonoVue in Female Infertility Assessment in China

Drug Approval
  • Bracco Imaging's ultrasound contrast agent SonoVue has received approval from China's National Medical Products Administration for use in hysterosalpingo contrast sonography (HyCoSy) to assess female infertility.
  • The non-invasive procedure offers a radiation-free alternative to traditional X-ray hysterosalpingography for detecting fallopian tube blockages and uterine abnormalities that contribute to 25-35% of infertility cases in Chinese women.
  • Clinical evidence from 24 studies involving 1,358 women demonstrates HyCoSy with SonoVue achieves 93% sensitivity, 90% specificity, and 96% accuracy in assessing fallopian tube patency.
  • This approval expands access to advanced diagnostic tools supporting China's Healthy China 2030 initiative and represents a significant advancement in reproductive health services.

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