iStock, Bo Shen The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed. Incyte on Thursday successfully expanded the label of its cancer therapy Zynyz, opening up its use as a first-line therapy for patients with squamous cell carcinoma of the anal canal. Zynyz, first approved in 2023 for metastatic, recurrent or locally advanced Merkel cell carcinoma, is an

Courtesy of Sarepta Therapeutics Since Elevidys’ first approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up. Sarepta Therapeutics touted “statistically significant and clinically meaningful” functional improvements in kids aged 8 to 9 years with Duchenne muscular dystrophy treated with the company’s gene therapy Elevidys. Th

THOUSAND OAKS, Calif.--(BUSINESS WIRE)-- Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, announced the appointment of James Huang and Nachi Subramanian to its Board of Directors, effective following the completion the Company’s previously announced $16 million offering that was announced on May 15, 2025. The o

iStock, Dmitrii_Guzhanin The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency. BioMarin is acquiring rare disease specialist Inozyme Pharma for $270 million to build out its portfolio of enzyme therapies, the biotech announced on Friday. BioMarin will pay $4 for each outstanding share of Inozyme. The companies expect to complete the transaction in the third quarter, pending customary closing conditions such as anti-trust

Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected in Early 2026 with Potential Launch in 2027; Additional Clinical Programs to Expand to Patients of all Ages Potential First-in-Disease Treatment for ENPP1 Deficiency Conference Call and Webcast Scheduled Today at 8:45 a.m. ET SAN RAFAEL, Calif. and BOSTON, May 16, 2025 /PRNewswire/ -- BioMarin Phar

AbbVie’s Emrelis (telisotuzumab vedotin-tllv) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat a subset of lung cancer patients. The drug has been authorised to treat locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) in adults who exhibit high overexpression of the c-Met protein and have previously received systemic therapy. An estimated 226,650 people will be diagnosed with lung cancer in the US this year, with NSCLC accou

Novo Nordisk and Septerna have announced a new partnership worth $2.2bn to develop oral small molecule drugs for type 2 diabetes, obesity and other cardiometabolic diseases. The exclusive global collaboration and licence agreement combines Novo’s capabilities in cardiometabolic diseases with Septerna’s Native Complex Platform, which aims to “unlock the full potential” of G protein-coupled receptor (GPCR) therapies. GPCRs represent the largest and most varied family of cell membrane receptors in

- Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) - Safety profile consistent with prior studies of ELEVIDYS and real-world experience CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 16, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported new results from Study 9001-103. Also known as ENDEAVOR, Study 9001-103 is a multi-cohor

Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (

SAN FRANCISCO and SUZHOU, China, May 15, 2025 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncologic, autoimmune, cardiovascular and metabolic, ophthalmologic and other major diseases, announces that the first participant has been successfully dosed in GLORY-3, a Phase 3 clinical study of mazdutide (Innovent R&D Code: IBI362), a dual glucagon