News

The FDA has accepted Allergan Aesthetics' supplemental Biologics License Application for BOTOX Cosmetic to treat masseter muscle prominence, a submission that could make it the first neurotoxin approved for this indication in the US.

Oblenio Bio has dosed the first patients in a phase 1a trial of tri-specific T-cell engager LBL-051 for refractory autoimmune disease.

New clinical updates this week highlight progress across rare disease gene therapy, food allergy immunotherapy, and immuno-oncology, with developers advancing treatments for high unmet need through regulatory milestones and early clinical development.

In an interview with BioPharm International, Stephanie Fradette, head of Biogen’s Rare Neurology Development Unit, discussed FDA Breakthrough Therapy designation for salanersen and early Phase 1b data evaluating the therapy in individuals with spinal muscular atrophy who had suboptimal responses following gene therapy.

Laurent Levy, founder and CEO of Nanobiotix, discusses why physics-based therapeutics could complement traditional biology-driven drug development and explains how the company's NanoPrimer platform is designed to improve delivery of complex medicines.

Rocket Pharmaceuticals has reported a positive safety update for RP-A501 from the first 3 patients treated under a modified phase 2 protocol in Danon disease.

FDA has granted fast track designation to Aravax's PVX108, a peptide immunotherapy for peanut allergy, ahead of phase 2 data.

Opus Genetics has enrolled the last patient in its registrational Phase 3 trial of OPGx-LCA5, an AAV8 gene therapy for LCA5-associated inherited retinal disease, keeping the program on track for a potential first-in-disease approval.

China's NMPA has approved an IND for a Phase Ib/II trial combining Leads Biolabs' PD-L1/4-1BB bispecific antibody opamtistomig with standard regimens in metastatic colorectal cancer, extending the drug's reach into one of oncology's most immunotherapy-resistant tumor types.

Three major regulatory milestones highlight this week's biopharmaceutical news, from a biosimilar advancing through FDA and EMA review to a new first-line breast cancer approval in Europe and fresh Phase 3 cardiovascular data that could reshape inflammatory disease drug development.

Planning the analytical strategy for product release should be started as early as possible to ensure product quality and regulatory success.

Novo Nordisk's ziltivekimab failed to reduce cardiovascular events in the phase 3 ZEUS trial despite confirmed IL-6 target engagement.

The EC has approved AstraZeneca and Daiichi Sankyo’s datopotamab deruxtecan ADC for 1st-line metastatic TNBC, which showed a 5.0-month overall survival benefit over chemotherapy.

Polpharma Biologics and Fresenius Kabi announced that the FDA and EMA have accepted for review PB016, a proposed biosimilar to Takeda's Entyvio (vedolizumab), with Fresenius Kabi holding exclusive global commercialization rights outside the Middle East and North Africa.

We answer readers’ top questions on limit of blank, limit of detection, limit of quantitation, acceptance criteria, and equivalence testing in analytical method validation.

This episode of The BioPharm Brief explores how next-generation biologics are reshaping oncology and autoimmune disease, from a first-line antibody-drug conjugate filing and a novel ADC entering the clinic to a major investment in in vivo CAR-T technology.

As quality control labs move toward more connected, data-driven operations, digital technologies must be adopted in a way that upholds patient safety.

CMC teams can align with Q5A(R2)’s updated guidance to shorten testing timelines and strengthen assurance of participant safety.

An FDA warning letter to Purolea Cosmetics Lab shows that AI-assisted drafting can support documentation work only when the resulting document is subject to quality-system controls.

Kelun-Biotech's sac-TMT has gained NMPA acceptance for a new first-line indication application in advanced triple-negative breast cancer.

Collaboration between suppliers and developers, as well as viral vector manufacturing simplification, are becoming critical to CGT's commercial future, says Brandon Pence of FUJIFILM Biosciences.

Iksuda Therapeutics has received FDA clearance of its IND application for IKS04, a CA242-directed antibody-drug conjugate paired with a novel dosing approach designed to improve solid tumor penetration, enabling a Phase 1 trial in gastrointestinal cancers.

FDA has granted priority review to Johnson & Johnson's Rybrevant Faspro for head and neck cancer, backed by 42% ORR in the OrigAMI-4 trial.

Johnson & Johnson has entered a collaboration and equity investment agreement with Sail Biomedicines to advance in vivo CAR-T therapies for immune-mediated diseases, securing an exclusive option to acquire the company for $2.58 billion.

Today's episode explores how FDA expedited pathways continue to shape advanced therapy development, from new RMAT designations for cell and gene therapies to an advisory committee review that underscores the agency's evolving expectations for clinical evidence.

Aprea Therapeutics CEO Dr Oren Gilad discusses ASCO 2026 data for WEE1 inhibitor APR-1051 and the company's precision oncology strategy while at BIO 2026.

Allogene's cema-cel has received FDA RMAT and fast track designations for first-line consolidation therapy in high-risk large B-cell lymphoma.

The FDA has granted Regenerative Medicine Advanced Therapy designation to Ocugen's OCU410, a one-time AAV gene therapy for geographic atrophy secondary to dry age-related macular degeneration, based on Phase 2 data showing a statistically significant reduction in lesion growth.