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FDA Budget Cuts Linked to Drug Approval Delays as Small Biotech Faces PDUFA Postponement

Drug ApprovalRare Disease
• Recent government efficiency cuts at the FDA appear to be causing delays in drug approval timelines, with 66% of healthcare professionals surveyed expressing concern about potential PDUFA date postponements.
• Stealth BioTherapeutics' treatment for rare Barth syndrome, elamipretide, has experienced an unexplained decision delay, with no typical safety or manufacturing concerns cited as reasons for the postponement.
• Industry experts warn that while widespread disruption isn't yet evident, continued FDA resource constraints could significantly impact clinical trial oversight and create approval backlogs lasting years.

FDA Sets Q3 2025 Approval Dates for Three Breakthrough Therapies Targeting Rare Diseases

Drug ApprovalRare DiseaseMonoclonal AntibodyOrphan Drug
  • The FDA has established PDUFA target action dates for three promising therapies in Q3 2025: sepiapterin for phenylketonuria (July 29), apitegromab for spinal muscular atrophy (September 22), and paltusotine for acromegaly (September 25).
  • PTC Therapeutics' sepiapterin demonstrated a 63% reduction in blood phenylalanine levels in PKU patients, with 84% achieving therapeutic control and over 97% able to liberalize their protein-restricted diets.
  • Scholar Rock's apitegromab showed statistically significant motor function improvements in SMA patients already receiving standard care, while Crinetics' paltusotine achieved IGF-1 normalization in 83% of acromegaly patients switching from injectable treatments.
  • These approvals could provide significant therapeutic advances for patients with rare genetic disorders who currently have limited treatment options or face challenges with existing therapies.

Sakar Healthcare Secures Second EU Marketing Authorization for Colorectal Cancer Treatment

OncologyDrug Approval
  • Sakar Healthcare received its second Marketing Authorization from the European Union for an oncology injection product targeting colorectal cancer treatment.
  • The approval follows the company's first EU marketing authorization received in April 2025 for treating breast cancer, non-small cell lung cancer, and prostate cancer.
  • The company has several additional oncology products under EU registration and expects to receive marketing authorizations within the coming months.
  • Sakar's strategic partnership with Accord Healthcare provides access to commercial infrastructure across 45 countries in the EU and UK markets.

Peanut Allergy Treatment Market Poised for 16% Growth as New Therapies Enter Pipeline

Drug Approval
  • The peanut allergy treatment market is projected to grow at a 16% CAGR from 2020-2034, driven by current epinephrine usage, anticipated XOLAIR adoption in adults, PALFORZIA expansion to toddlers, and the expected entry of Viaskin Peanut patches.
  • DBV Technologies' Viaskin Peanut patch leads the pipeline as a promising non-invasive epicutaneous immunotherapy option, with Phase III VITESSE trial results expected in Q4 2025 and potential BLA submission in the first half of 2026.
  • The market reached USD 400 million in 2024 across seven major markets, with approximately 9.8 million diagnosed prevalent cases, though only 50% of affected individuals in the US received treatment, highlighting significant care gaps.
  • Stallergenes Greer launched PALFORZIA for toddlers aged 1-3 years in February 2025, while the FDA approved OMLYCLO as the first interchangeable biosimilar for XOLAIR in March 2025.

Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Children 4-7 Years of Age

NCT05741476Active, Not RecruitingPhase 3
DBV Technologies
Posted 2/21/2023

Cuban Drug Jusvinza Advances Through Phase III Trial for Rheumatoid Arthritis Treatment

Drug Approval
  • Jusvinza, developed by Cuba's Center for Genetic Engineering and Biotechnology, is currently undergoing a Phase III clinical trial for rheumatoid arthritis treatment following conditional registration in September 2023.
  • The placebo-controlled, double-blind trial includes patients with moderate rheumatoid arthritis and aims to corroborate the drug's efficacy, with initial results showing good safety profile and only mild, reversible adverse events.
  • Clinical studies are expanding to investigate Jusvinza's potential in other autoimmune diseases including ankylosing spondylitis, systemic lupus erythematosus, and inflammatory bowel diseases.
  • The drug has demonstrated effectiveness in controlling hyperinflammation and has shown benefits in real-world studies of severe rheumatoid arthritis patients, with definitive registration expected next year.

ToolGen Files Patent Infringement Lawsuit Against Vertex Over CRISPR Gene Therapy Casgevy

Drug Approval
  • Korean biotech company ToolGen has filed patent infringement lawsuits against Vertex Pharmaceuticals in the UK and Netherlands over the world's first approved CRISPR gene therapy, Casgevy.
  • ToolGen claims Vertex's $2.2 million therapy was developed using its proprietary CRISPR RNP technology without proper licensing recognition.
  • The lawsuit targets Casgevy, which treats sickle cell disease and beta-thalassemia and has received marketing authorization in the UK, EU, and US.
  • ToolGen seeks a global licensing agreement rather than restricting patient access to the breakthrough therapy.

Cemiplimab Shows Significant Survival Benefit in NSCLC Patients with Brain Metastases and High PD-L1 Expression

Monoclonal AntibodyDrug ApprovalOncology
  • First-line cemiplimab (Libtayo) monotherapy demonstrated a remarkable survival advantage over chemotherapy in advanced NSCLC patients with PD-L1 ≥50% and brain metastases, with median OS of 52.4 vs 20.7 months.
  • The EMPOWER-Lung 1 trial's 5-year follow-up data confirms cemiplimab's long-term clinical benefits across all PD-L1 expression levels, with the greatest benefit observed in patients with PD-L1 ≥90%.
  • Patients receiving cemiplimab experienced fewer grade ≥3 adverse events compared to chemotherapy (18.3% vs 39.9%), while also reporting improved quality of life measures including better emotional functioning and reduced fatigue.

Study of REGN 2810 Compared to Platinum-Based Chemotherapies in Participants With Metastatic Non-Small Cell Lung Cancer (NSCLC)

NCT03088540CompletedPhase 3
Regeneron Pharmaceuticals
Posted 5/29/2017

Combinations of Cemiplimab (Anti-PD-1 Antibody) and Platinum-based Doublet Chemotherapy in Patients With Lung Cancer

NCT03409614CompletedPhase 3
Regeneron Pharmaceuticals
Posted 3/6/2018

A Study of CS1001 in Subjects With Stage IV Non-Small Cell Lung Cancer

NCT03789604Active, Not RecruitingPhase 3
CStone Pharmaceuticals
Posted 12/13/2018

Ipsen Secures Dual CHMP Approvals for Rare Liver Disease Treatments Following Regulatory Strategy Shift

Drug ApprovalRare DiseaseOrphan Drug
  • Ipsen received CHMP approval for odevixibat under the new brand name Kayfanda for Alagille syndrome, marking the second approval for the same drug after rebranding due to orphan status complications.
  • The company simultaneously secured CHMP recommendation for Iqirvo (elafibranor) as a treatment for primary biliary cholangitis, representing a rare dual approval achievement.
  • Odevixibat demonstrated statistically significant improvements in scratching severity in the ASSERT trial, the world's first phase 3 study completed in Alagille syndrome patients.
  • Both approvals address significant unmet medical needs in rare cholestatic liver diseases, with analysts estimating the PBC market alone could exceed $1.5 billion annually.

Chugai's Vabysmo Becomes First Approved Treatment for Angioid Streaks in Japan

Drug ApprovalRare Disease
  • Chugai Pharmaceutical has received Japanese regulatory approval for Vabysmo as the first-ever treatment for choroidal neovascularization associated with angioid streaks, a rare eye disease that can lead to vision loss.
  • The approval follows positive Phase III NIHONBASHI study results showing statistically significant visual acuity improvement of +5.8 letters at week 12 and a reduction in central retinal thickness of -106.4 μm.
  • Vabysmo, a bispecific antibody targeting both VEGF-A and Ang-2 pathways, was generally well-tolerated with no new safety concerns identified during the clinical trial.

FDA Approves 25 First-Time Generic Drugs in First Half of 2025, Expanding Access to Critical Therapies

FDA ApprovalDrug ApprovalOncology
  • The FDA approved 25 first-time generic drugs in the first six months of 2025, including generics for critical medications treating HIV, cancer, and neurological conditions.
  • Notable approvals include generic versions of Xarelto (rivaroxaban) for blood clot prevention, Complera for HIV treatment, and Fycompa (perampanel) for seizure management.
  • These generic approvals span diverse therapeutic areas from oncology to ophthalmology, potentially reducing healthcare costs while maintaining equivalent clinical benefits to brand-name products.

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