In an unusual move, three members of the Nov. 6 FDA advisory committee that voted against recommending approval of Biogen Inc.’s high profile Alzheimer’s disease candidate, aducanumab, have doubled down on their argument in an editorial published in JAMA on March 31.
The first BCMA-targeted CAR T therapy, idecabtagene vicleucel, cleared FDA approval for use in adults with multiple myeloma (MM) who have received four or more prior lines of therapy. Developed by partners Bluebird Bio Inc. and Bristol Myers Squibb Co., the drug, branded Abecma, is also the first CAR T drug indicated for MM. It is designed for use as a one-time infusion, with a recommended dose range of 300 to 460 x 106 CAR-positive T cells. The personalized therapy will be produced at BMS’ cellular manufacturing facility in Summit, N.J. Bluebird developed the lentiviral vector used in Abecma.
Barely five and a half years since the company was founded, Kiniksa Pharmaceuticals Ltd. has moved its first drug over the finish line, winning FDA approval for interleukin-1 blocker (IL-1) rilonacept to treat recurrent pericarditis and reduce the risk of recurrence in adults and children 12 and older.
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.
Investing in biopharma has never been for the faint of heart. So headline figures unveiled from a clinical development success report during the BIO CEO & Investor Conference Feb. 17, putting the average likelihood of a drug entering phase I development ultimately achieving approval at 7.9% and the average drug development timeline at 10.5 years, appear largely unsurprising. But the addition of machine learning capabilities to the mix helped identify those factors that have the greatest impact on predictive outcome.
As expected, G1 Therapeutics Inc.’s Cosela (trilaciclib) won FDA approval for use in extensive-stage small-cell lung cancer patients undergoing chemotherapy, becoming the first proactively administered myelopreservation therapy to hit the market.
Regeneron Pharmaceuticals Inc.’s angiopoietin-like 3 (ANGPTL3)-targeting antibody, Evkeeza (evinacumab), won FDA approval for use in reducing LDL cholesterol in patients with homozygous familial hypercholesterolemia.
While it’s too late to save the contingent value rights connected with the acquisition of Celgene Inc., Bristol Myers Squibb Co.’s CD19-targeted CAR T therapy, lisocabtagene maraleucel, for treating certain types of relapsed or refractory large B-cell lymphoma patients who have received at least two prior therapies, won FDA approval.
Executives of TG Therapeutics Inc. have promised a big year in 2021 and the company got off to strong start with accelerated FDA approval of umbralisib in marginal zone lymphoma and follicular lymphoma. Branded Ukoniq, the drug is the first oral, once-daily inhibitor of PI3K delta and CK1 epsilon and TG’s first commercial product. Pricing for Ukoniq has not yet been disclosed.
Concert Pharmaceuticals Inc. is scratching further work on CTP-692 after the deuterated form of D-serine missed the primary endpoint in a phase II study in schizophrenia. The news sent shares of Concert (NASDAQ:CNCE) falling 28% Feb. 1 and shifts focus to the firm’s sole remaining clinical-stage program in the competitive alopecia areata space.