A day ahead of its PDUFA date, Mepsevii (vestronidase alfa-vjbk, previously UX-003) won FDA approval to treat children and adults with the ultra rare inherited metabolic condition mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. The nod gave developer Ultragenyx Pharmaceutical Inc. its first commercial product and represented a first approval in the indication. Read More

Sangamo Therapeutics Inc.'s chief medical officer Edward Conner said the first patient to be treated with SB-913 "was clear that he wasn't just looking for help for himself, but he wanted to help future generations of MPS patients as well," and the in vivo genome-editing therapy could do just that for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. Read More

As far as gene editing technologies go, clustered regularly interspaced short palindromic repeats, or CRISPR, tends to suck up all the oxygen in the room. Read More

Astrazeneca plc has won FDA approval to market Fasenra (benralizumab) as an add-on maintenance therapy for severe, eosinophilic asthma patients, age 12 years and older. Injected every eight weeks after a run-in period, the monoclonal antibody helped trial patients achieve a reduction in their annual asthma exacerbation rates (AAER) of up to 51 percent vs. placebo. Read More

DUBLIN – U.K. start-up Critical Pressure Ltd. raised £10 million (US$13.2 million) in a series A round to move a selective inhibitor of dimethylarginine dimethylaminohydrolase 1 (DDAH1) into clinical development in intensive care patients with severe sepsis. Read More

Therapeutic administration of interleukin-10 (IL-10) into the cerebrospinal fluid through intrathecal gene therapy caused fatal meningitis in swine, an unexpected toxicity that had not been seen in multiple rodent experiments. Read More

BOGOTA, Colombia – Efforts to streamline and speed up drug approvals in Colombia are proving effective, with a new committee focused on new molecules and biologics moving applications forward at a rapid pace. Read More

Biogen Inc., of Cambridge, Mass., entered a partnership for French diagnostic firm Theradiag SA to provide its Lisa Tracker kits for monitoring Flixabi, an infliximab biosimilar. Financial terms were not disclosed. Biogen has marketed Flixabi in France since 2016. Read More

Sage Therapeutics Inc., of Cambridge, Mass., said it priced an underwritten public offering of 3.52 million shares of its common stock at $85.00 each. The gross proceeds are expected to be approximately $300 million. The company has also granted the underwriters a 30-day option to purchase up to an additional 529,411 shares of its common stock. Read More

The FDA released a draft guidance on assessing user fees under the BsUFA II agreement approved by Congress earlier this year. The draft discusses the agency's implementation of the agreement and certain intended policy and procedural changes. For instance, the guidance explains the new fee structure under BsUFA II and the types of fees biosimilar sponsors will pay. It also describes when the various fees are incurred, how they are to be submitted, what happens when a sponsor fails to pay the fees, and how requests for reconsideration or an appeal are to be filed. Notice of the draft is slated for publication in Thursday's Federal Register and will be followed by a 60-day comment period. Read More

Cynata Therapeutics Ltd., of Melbourne, Australia, said that the eighth participant has been dosed in its first trial of the mesenchymal stem cell product CYP-001 for the treatment of steroid-resistant graft-versus-host-disease, marking the half-way point for recruitment into the study. An independent data safety monitoring board review will be triggered once the patient reaches day 28 of the study. Read More