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FDA Approves Multiple New Cancer Therapies Including Bayer's Hyrnuo for NSCLC and Amgen's Imdelltra for Small Cell Lung Cancer

Drug ApprovalOncology
  • The FDA has approved Bayer's Hyrnuo (sevabertinib), an oral tyrosine kinase inhibitor for locally advanced or metastatic non-squamous non-small cell lung cancer in adults with HER2-activating mutations.
  • Amgen received full FDA approval for Imdelltra (tarlatamab-dlle) to treat extensive stage small cell lung cancer with disease progression after platinum-based chemotherapy, converting from accelerated approval status.
  • AstraZeneca's Koselugo (selumetinib) gained FDA approval for adult patients with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas based on Phase III trial results.
  • Additional approvals include Regeneron's Eylea HD for macular edema and Accord BioPharma's biosimilars Osvytri and Jubereq for bone-related conditions.

FDA Approves Accord BioPharma's Denosumab Biosimilars OSVYRTI and JUBEREQ for Bone Disorders

Drug ApprovalOncology
  • The FDA has approved OSVYRTI (denosumab-desu) and JUBEREQ (denosumab-desu), biosimilars to Amgen's Prolia and Xgeva respectively, marking Accord BioPharma's fourth and fifth biosimilar approvals.
  • OSVYRTI is indicated for treating osteoporosis in postmenopausal women and men, glucocorticoid-induced osteoporosis, and bone loss from cancer hormone therapy, while JUBEREQ prevents skeletal-related events in cancer patients.
  • The approvals were based on Phase I and Phase III trials demonstrating comparable pharmacokinetics, pharmacodynamics, safety and efficacy to the reference products, with commercial launch planned for 2026.
  • These biosimilars target a significant market opportunity, as Prolia generated over $4.374 billion in global sales in 2024 while Xgeva reached $2.225 billion in revenues.

Denosumab Biosimilar Injection in Post Menopausal Women With Osteoporosis

NCT05419427RecruitingPhase 3
Lambda Therapeutic Research Ltd.
Posted 11/11/2021

Novartis Raises Peak Sales Targets for Cancer Drugs Kisqali and Scemblix, Forecasts 5-6% Annual Growth Through 2030

OncologyDrug Approval
  • Novartis increased peak sales forecasts for breast cancer drug Kisqali to at least $10 billion from $8 billion and leukemia therapy Scemblix to at least $4 billion from $3 billion.
  • The Swiss drugmaker projects 5-6% annual sales growth through 2030 on a currency-adjusted basis, driven by these key oncology assets despite patent expiries for other products.
  • The company has invested $30 billion in acquisitions and licensing deals this year, including a planned $12 billion acquisition of Avidity Biosciences to strengthen its pipeline.
  • Novartis targets a core operating income margin of at least 40% by 2029, factoring in a 1-2 percentage point impact from the Avidity acquisition expected to close in first half 2026.

FDA Approves AbbVie and Genmab's Epkinly for Relapsed or Refractory Follicular Lymphoma

Drug ApprovalOncology
  • AbbVie and Genmab have received FDA approval for Epkinly as a treatment option for patients with relapsed or refractory follicular lymphoma.
  • The approval adds a new targeted immunotherapy to the lymphoma treatment landscape and expands Epkinly's reach following prior approvals in other B-cell malignancies.
  • This development marks a significant advancement in the treatment of follicular lymphoma, a type of non-Hodgkin's lymphoma.
  • The decision provides patients with an additional therapeutic option in the challenging relapsed or refractory setting.

FDA Grants Full Approval to Amgen's Imdelltra for Extensive Stage Small Cell Lung Cancer

Drug Approval
  • The FDA has granted full approval to Amgen's Imdelltra (tarlatamab-dlle) for treating adult patients with extensive stage small cell lung cancer that has progressed after platinum-based chemotherapy.
  • The Phase 3 DeLLphi-304 study demonstrated that Imdelltra reduced the risk of death by 40% and extended median overall survival by more than five months compared to standard chemotherapy (13.6 vs. 8.3 months).
  • The National Comprehensive Cancer Network has updated its guidelines to include tarlatamab as the only Category 1 preferred treatment option for this patient population.
  • Imdelltra is a first-in-class bispecific T-cell engager that targets DLL3 protein, which is expressed on the surface of SCLC cells in approximately 85-96% of patients.

Study Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer

NCT05740566Active, Not RecruitingPhase 3
Amgen
Posted 5/31/2023

SCYNEXIS Completes BREXAFEMME NDA Transfer to GSK, Enabling Antifungal Drug Relaunch

Drug Approval
  • SCYNEXIS has completed the transfer of BREXAFEMME's New Drug Application to GSK, allowing the pharmaceutical giant to initiate FDA discussions for the antifungal drug's relaunch.
  • BREXAFEMME (ibrexafungerp) is FDA-approved for treating vulvovaginal candidiasis and reducing recurrent infections, representing the first in SCYNEXIS's novel "fungerps" antifungal platform.
  • Following relaunch, SCYNEXIS stands to receive up to $145.5 million in annual net sales milestones plus low to mid single-digit royalties from GSK.
  • The transfer positions GSK to reintroduce BREXAFEMME for vulvovaginal candidiasis and refractory vulvovaginal candidiasis in the U.S. market through regulatory engagement with the FDA.

Arrowhead Pharmaceuticals Secures First FDA Approval with Redemplo for Rare Genetic Disorder

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved Redemplo (plozasiran), Arrowhead Pharmaceuticals' first commercial drug, for treating familial chylomicronemia syndrome in adults as an adjunct to diet.
  • The approval represents a transformational milestone for the RNAi biotech company, which has been developing RNA interference medicines for over two decades without a marketed product.
  • Redemplo demonstrated superior efficacy in Phase 3 trials with 70% triglyceride reduction versus 59% for competitor Tryngolza, while launching at $60,000 annually compared to Tryngolza's $595,000 price.
  • The drug targets apoC-III protein production using Arrowhead's proprietary TRiM platform and is administered subcutaneously once every three months for approximately 6,500 FCS patients in the U.S.

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

NCT05089084Active, Not RecruitingPhase 3
Arrowhead Pharmaceuticals
Posted 1/11/2022

FDA Approves First siRNA Therapy Plozasiran for Rare Metabolic Disorder Familial Chylomicronemia Syndrome

Drug ApprovalRare DiseaseOrphan Drug
  • The FDA approved Redemplo (plozasiran), the first small interfering RNA therapy for familial chylomicronemia syndrome, a rare metabolic disorder with triglyceride levels 10-100 times above normal.
  • In the phase 3 PALISADE trial, plozasiran achieved an 80% median reduction in triglycerides compared to 17% with placebo at 10 months when administered subcutaneously every 3 months.
  • The therapy targets apolipoprotein C-III to slow triglyceride breakdown and received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA.
  • Plozasiran is expected to be available in the US before year-end, offering hope for patients with this historically overlooked condition that carries high pancreatitis risk.

Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)

NCT05089084Active, Not RecruitingPhase 3
Arrowhead Pharmaceuticals
Posted 1/11/2022

Health Canada Approves NILEMDO for LDL-Cholesterol Reduction in Cardiovascular Disease Patients

Drug Approval
  • Health Canada has approved NILEMDO (bempedoic acid) for reducing LDL-cholesterol in Canadians at risk of cardiovascular disease, with commercial launch expected in Q2 2026.
  • The approval represents a significant milestone for Esperion's global strategy to expand access to non-statin cholesterol-lowering therapies for patients with unmet medical needs.
  • Heart disease affects approximately 2.6 million Canadian adults and is the second leading cause of death in Canada, highlighting the critical need for additional treatment options.
  • NILEMDO is part of Esperion's portfolio of FDA-approved oral, once-daily, non-statin medicines supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial.

FDA Approves First Antithrombin Concentrate for Pediatric Patients with Hereditary Antithrombin Deficiency

Drug ApprovalRare Disease
  • The FDA has approved an expanded indication for Grifols' antithrombin III (THROMBATE III) to include pediatric patients with hereditary antithrombin deficiency, making it the first and only antithrombin concentrate approved for both adults and children with this rare blood clotting disorder.
  • The approval was based on extrapolated adult clinical data demonstrating that antithrombin concentrate can be safely and effectively used in pediatric patients with hereditary antithrombin deficiency.
  • This expansion addresses a significant unmet medical need, as hereditary antithrombin deficiency has one of the highest thrombotic risks among inherited thrombophilias, with 85% of patients experiencing at least one thrombotic episode by age 50.
  • The approval provides clinicians with added confidence to appropriately use antithrombin concentrate in children, helping to close a long-standing treatment gap for pediatric patients with this rare disorder.

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