Argenx NV’s Vyvgart (efgartigimod), approved late Friday by the FDA for treating generalized myasthenia gravis, became the first FcRn antagonist to cross the finish line. But the best news may be the drug’s broad label, which company executives highlighted during an investor call.
Calliditas Therapeutics AB expects its oral version of budesonide to be available early in the first quarter of 2022, following FDA approval for treating patients with progressive kidney disease primary immunoglobulin A nephropathy (IgAN). Branded Tarpeyo, the delayed-release capsules were cleared for use in reducing proteinuria in adults with primary IgAN at risk of rapid disease progression, usually determined as a protein-to-creatinine ration (UPCR) of 1.5 g/g or less.
Biomarin Pharmaceutical Inc.’s data supporting the use of Voxzogo (vosoritide) in children with the most common form of dwarfism proved compelling for the FDA, which cleared the modified C-type natriuretic peptide as the first treatment for the rare genetic disease affecting bone growth.
A European approval for Alzheimer’s drug aducanumab by year-end looks even more in doubt. A week after being called in before the EMA’s Committee for Medicinal Products for Human Use (CHMP) for an oral explanation of the trial data, Biogen Inc. said it received a “negative trend vote” on the marketing authorization application. A formal opinion by the CHMP is expected at its December meeting, but analysts are not optimistic.
Shares of Alkermes plc slipped 15% Nov. 9 on news that longtime partner Janssen Pharmaceutica NV plans to partially terminate two license agreements related to know-how royalties on U.S. sales of paliperidone products using Alkermes’ nanoparticulate technology. While the move is expected to result in only a modest near-term impact on the Dublin-based firm’s bottom line, it took both company management and investors by surprise.
Clade Therapeutics Inc., which launched with an $87 million series A round, may have what sounds like an ambitious goal: to create scalable, off-the-shelf stem cell-based medicines that can be as accessible to patients as antibody therapies are today. But the startup, backed by more than two decades of advances in the area of induced pluripotent stem cells, is within sight of developing a cell therapy to take into clinical testing.
Shares of Angion Biomedica Corp. tumbled more than 50% Oct. 27 on disappointing top-line data from its phase III study testing ANG-3777’s ability to improve organ function in patients receiving deceased donor kidney transplants. The company’s management, however, remained cautiously optimistic that the safety results and signals of biological activity could bode well for an exploratory phase II study testing the drug in acute kidney injury associated with cardiac surgery involving cardiopulmonary bypass, which is expected to read out later this quarter.
As largely expected, the FDA on Oct. 20 authorized the use of booster doses for COVID-19 vaccines from Moderna Inc. and Johnson & Johnson, in line with last week’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) meetings. It also cleared the way for “mix-and-match” boosters, allowing eligible people to receive boosters from any of the FDA-approved vaccines, regardless of which vaccine they received originally.
In its first big pharma deal since it was founded around a cell programming technology in 2009, Immusoft Corp. signed Takeda Pharmaceutical Co. Ltd. to a research collaboration and license option targeting rare inherited metabolic disorders. The agreement brings an undisclosed up-front fee and research funding to Immusoft, which is also eligible to earn more than $900 million if all options are exercised and all milestones hit.
