Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Adaptimmune, Astrazeneca, Biomarin, Blueprint, Galapagos, Gilead, Heron, Insmed, Kyowa Kirin, Miragen, Myokardia, Novartis, Oncoceutics, Sarepta, Vistagen.
Since the stock-jolting phase III blowup in March of Milestone Pharmaceuticals Inc.’s etripamil for paroxysmal supraventricular tachycardia (PSVT), questions have lingered regarding the short-acting channel blocker’s regulatory path forward – but no longer. Shares of Montreal-based Milestone (NASDAQ:MIST), which in the spring plummeted to an all-time low of $1.70, made up for the loss and then some, closing at $8.91, up $5.57, or 167%, on word that the FDA has agreed to terms whereby an NDA for the nasal spray may be submitted without launching another phase III study.
Although PDUFA VI still has two years of life left to it, PDUFA VII is already in the birthing process, with the use of real-world data (RWD), AI, and a coming surge of novel cell and gene therapies looking to be prominent features of the next five-year user fee agreement. Politics likely will play a role as well.
The FDA has lifted all the clinical holds it placed on seladelpar from Cymabay Therapeutics Inc. for INDs in nonalcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC) and primary sclerosing cholangitis, giving the company new hope.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Adial, Cymabay, Elsalys, Inotrem, Milestone, Neumentum, Vita.
The U.S. government has charged two citizens of China with cybercrime in connection with purported hacking of research into vaccines for the SARS-CoV-2 virus, but more than one speaker on a July 22 webinar said scientists involved in basic life science research at universities fail to appreciate the need for cybersecurity, a problem they may take with them to the private sector.
The FDA has approved Xywav (calcium, magnesium, potassium and sodium oxybates; JZP-258), an oxybate product for treating both cataplexy and excessive daytime sleepiness in narcolepsy patients ages 7 and older, from Jazz Pharmaceuticals plc.
Interludes of classical music. Little kids talking in the background. Unmuted mics as panelists multitask. Gurgles of “underwater” sound. Periods of silence as speakers forget to unmute. And then the technical problems – lots of them. Such are the challenges of addressing a virtual FDA advisory committee in the time of COVID-19.
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aurinia, AGTC, Jazz, MC2, Redhill.
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