Six of six critically ill COVID-19 patients survived after being treated as compassionate care cases with Capricor Therapeutics Inc.’s lead candidate, an off-the-shelf cardiac cell therapy. The success prompted the FDA to review the data and approve the company’s expanded access protocol for treating as many as 20 more COVID-19 patients.
Saudi Arabia, which last year made its first appearance on the U.S. Trade Representative’s (USTR) Priority Watch List, is back on the list this year and is being singled out for an out-of-cycle review due to what the USTR calls its “unfair commercial use” and “unauthorized disclosure” of proprietary data submitted for drug approvals.
A pair of good-news items from Chimerix Inc. pushed the Durham, N.C.-based company’s stock (NASDAQ:CMRX) to $2.15, closing up 64 cents, or 42%, higher as backers reacted to near-term NDA plans for smallpox countermeasure brincidofovir (BCV) and the start of a phase II/III trial with dociparstat sodium (DSTAT) in COVID-19 patients with acute lung injury (ALI).
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Algernon, Amylyx, ANI, Azurrx, Beyondspring, Capricor, Chimerix, Erytech, Merck, Neuraptive, Novartis, Ortho Dermatologics, Remegen.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Adaptimmune, Bioeclipse, Moderna, Moleculin, Pharmamar, Retrotope, Roche, Sanofi, Shionogi, Zambon.
A once-daily add-on therapy for Parkinson's disease (PD) used in Europe for years has now gained clearance in the U.S. with FDA approval of Ongentys (opicapone). The drug, an improvement upon generics in its class, will be sold by Neurocrine Biosciences Inc. The medicine, a catechol-O-methyltransferase (COMT) inhibitor first approved by the EMA in 2016, extends the half-life of levodopa, increasing doses of which are required to achieve motor control as PD progresses. Portugal-based Bial-Portela & Ca SA, from which Neurocrine licensed North American rights to the drug, will receive a $20 million award from its partner. Shares of San Diego-based Neurocrine (NASDAQ:NBIX), a neuroscience company focused on treating movement disorders, rose 3.5% following the approval, closing at $101.67 on April 27.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Carmell, Carsgen, Cytodyn, Diffusion, Eiger, Green Valley, Halozyme, Janssen, Mateon, Neurocrine, Polyneuron, Takeda.
The U.S. Centers for Medicare and Medicaid Services (CMS) and the Office of the National Coordinator for Health Information Technology (ONC) said that they will offer enforcement discretion for their respective final rules for electronic health records (EHRs), a nod to the COVID-19 pandemic.