Nuravax will test AV-1959R in a 160-adult phase 2 trial to see whether the amyloid vaccine can prevent Alzheimer disease before symptoms begin.
|Articles|January 17, 2019
FDA Issues Draft Guidance on Rare Diseases
Author(s)BioPharm International Editors
The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.
Advertisement
On Jan. 17, 2019, FDA announced that it issued a revised draft
The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015. The revised guidance covers a range of topics such as natural history studies, use of biomarkers, and methods for demonstrating effectiveness and safety.
Advertisement
Related to this article
Alexion’s ravulizumab won accelerated approval to reduce proteinuria in adults with IgA nephropathy after the Phase 3 I CAN trial showed a 43.4% placebo-adjusted reduction at Week 34.
Cemiplimab's proposed EU regimen could cut Libtayo infusion visits from every 3 weeks to every 6 weeks, trimming infusion time by up to half.
The FDA approved Roche's satralizumab (Enspryng), an IL-6 receptor-targeting monoclonal antibody, for adults with thyroid eye disease based on the Phase 3 SatraGO-1 and SatraGO-2 trials, giving patients the first at-home subcutaneous treatment option.
AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.
Rohit Tripathi, vice president of industry strategy, consumer packaged goods, and manufacturing at RELEX Solutions, explains where cold-chain planning still breaks down and why biopharma supply chain leaders should use AI to act on data sooner, with humans making the final call.
Nathan Edwards of Arya Consulting moderates a discussion with Elisabeth Gardiner of Tevard Biosciences and Donny Xu of the RTW Institute about rare disease treatment development and navigating the FDA’s Rare Pediatric Disease Priority Review Voucher Program.
The FDA grants accelerated approval to Ultomiris for IgA nephropathy, Alcon acquires Neurotech and its MacTel cell therapy for $550 million, and BioNTech and Bristol Myers Squibb add Trodelvy to a phase 3 trial in triple-negative breast cancer.
Advertisement
Advertisement
Trending on BioPharm International
1
BioNTech, BMS to Pair Pumitamig With Trodelvy in Phase 3 First-Line TNBC Trial
2
Zylidac Bio to Scale Prolific's Optogenetic Cell Line Platform to GMP
3
FDA Grants Accelerated Approval to Ultomiris as First C5 Inhibitor for IgA Nephropathy
4
Sanofi Begins Construction of EU-Backed Insulin Plant in Frankfurt
5