With the FDA approval of Abbvie Inc.’s Qulipta (atogepant) to prevent episodic migraine in adults, the oral calcitonin gene-related peptide (CGRP) receptor antagonist became the first specifically developed for preventing migraine. Read More
Amicus Therapeutics Inc.’s plan to spin off its gene therapy work by way of the combination with blank check firm Arya Sciences Acquisition Corp. IV “allows us to be laser-focused on maximizing the opportunity for Galafold [migalastat]” while setting up launch preparations for AT-GAA, said Bradley Campbell, chief operating officer. Read More
Astrazeneca plc’s Alexion rare diseases unit has taken an option to buy all remaining equity in Caelum Biosciences Inc., adding a rare disease drug to its pipeline in a deal worth $500 million. At the center of the deal is CAEL-101, a potentially first-in-class monoclonal antibody designed to tackle the toxic amyloid deposits that build up in organs of people with the rare disease light chain amyloidosis. Read More
Expansion Therapeutics Inc., a company developing new drugs for severe RNA-mediated diseases based on the work of its scientific founder, Scripps Research chemistry professor Matthew Disney, has raised $80 million in a series B financing to identify and advance oral small-molecule candidates for the potential treatment of myotonic dystrophy type 1, amyotrophic lateral sclerosis and tauopathies. Read More
Japan’s Ministry of Health, Labour and Welfare approved Takeda Pharmaceutical Co. Ltd.’s Alofisel (darvadstrocel) to treat complex perianal fistulas in patients with non-active or mildly active luminal Crohn’s disease. This marks the first allogeneic stem cell therapy to be greenlighted in the country. Read More
As expected, Mirum Pharmaceuticals Inc. won FDA clearance for Livmarli (maralixibat), a minimally absorbed ileal bile acid transporter inhibitor, for cholestatic pruritus in patients with the rare liver disease Alagille syndrome, 1 year of age and older. Read More
With new therapeutics under FDA review for migraine, Alzheimer’s disease and seizures, and several rare disease gene therapies in development, stocks included in BioWorld’s Neurological Diseases Index are tracking 6.09% ahead for the year. Read More
LONDON – Industry-sponsored clinical research in the U.K. took a big hit in the pandemic and is still struggling to catch up – and to capitalize on innovative trial designs and delivery approaches that were so successful in speeding up COVID-19 studies. Read More
When the White House COVID-19 Response Team announced its booster program in August, it justified the broad use of a third vaccine dose with antibody studies and real-world data from Israel. Read More
As part of its real-world evidence (RWE) program, the U.S. FDA is issuing a draft guidance on using electronic health records and medical claims data in clinical trials to support a drug’s safety and effectiveness. Read More
The FDA unveiled a draft guidance addressing the responsibilities of clinical investigators for safety reporting for investigational drugs and devices, a novel attempt to combine the disparate adverse event reporting timelines for investigational drugs and devices. Read More
Thanks to a drop in the cost of conducting U.S. FDA reviews, it’s getting even cheaper to use a priority review voucher (PRV) to shave four months off the agency’s standard 10-month drug review. Read More
New hires and promotions in the biopharma industry, including: Ajax, Aura, Curemark, Incyte, Mind Medicine, Neurosense, Pharmaessentia, Phosplatin, Sporos, Terns. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Altimmune, Artios, Dicerna, Ena Respiratory, First Wave, Graybug, Mind Medicine, Oncoinvent, Pfizer, Sanofi, Seagen, Translate, Valneva. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aim Immunotech, Astrazeneca, Eisai, Gilead, Humanigen, I-Mab, Iterum, Lipocine, MRM, Pfizer, Regeneron, Revelation, Sanofi, Viiv, Zogenix. Read More
