A new once-daily attention deficit hyperactive disorder (ADHD) drug developed by prodrug specialist Kempharm Inc. has won FDA approval under the brand name Azstarys. The medicine combines serdexmethylphenidate, Kempharm's prodrug of the CNS stimulant dexmethylphenidate, with immediate-release d-MPH, the core medicine in the now off-patent product Focalin, a refined formulation of Ritalin.
The separate “pass-through” payment Medicare provides for new, high-cost Part B drugs that are part of certain hospital procedures in the U.S. may be an incentive for hospitals to use those drugs rather than less expensive alternatives, according to a new Government Accountability Office (GAO) report.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Adverum, Argenx, Astrazeneca, Beigene, Biocryst, Biogen, Eisai, Eyenovia, Fibrogen, Merck, Molecular Targeting Technologies, Oyster Point, RDIF, Sorrento, Strongbridge.
Molybdenum cofactor deficiency (MoCD) type A, an ultra-rare metabolic disorder causing intractable seizures, brain injury and death, now has a world-first treatment in Nulibry (fosdenopterin), a new I.V. therapy developed by Bridgebio Pharma Inc. subsidiary Origin Biosciences Inc. The agency's priority review, supported by its orphan, breakthrough and rare pediatric disease programs, also yielded a priority review voucher (PRV) for Origin.
In divvying up U.S. spending on orphan vs. nonorphan indications for drugs approved for both, a new study could fuel future debates and inform policy on orphan drug incentives. The study, led by a team of University of Michigan and Boston University researchers, found that 21% of the total dollars spent in 2018 in the U.S. on the 15 top-selling partial orphan drugs went to the treatment of rare diseases, while more than 70% went to the treatment of common diseases.
In a move that caught analysts and investors by surprise, the FDA rejected Athenex Inc.’s NDA for Oraxol, its oral version of paclitaxel, for use in metastatic breast cancer, citing safety risks and questioning the reliability of the clinical data supporting the application.
The cost of newly FDA approved Pepaxto (melphalan flufenamide) from Oncopeptides AB is designed to put the therapy in the middle of the range compared to other multiple myeloma therapies, CEO Marty Duvall told BioWorld. “There are products well entrenched in that space,” he added.
With the FDA’s granting of emergency use authorization (EUA) to Johnson & Johnson (J&J), there is now a third vaccine – and the first requiring only a single shot – against COVID-19 for adult Americans. Though it packs less of an efficacy punch, the EUA allows J&J’s Ad26.COV2.S to join mRNA vaccines from Moderna Inc. and Pfizer Inc./Biontech SE as protection against the virus.
RSS
