New phase III data on Takeda Pharmaceutical Co. Ltd.'s TAK-620 (maribavir) for the treatment of transplant recipients with tough-to-treat cytomegalovirus (CMV) infections met the trial's primary endpoint, setting the company up to file an NDA for the oral antiviral in the first half of next year.
Shares of immunotherapy developer Enlivex Therapeutics Ltd. (NASDAQ:ENLV) climbed as much as 69% Dec. 3 after interim data showed the apparent benefits of its lead candidate, Allocetra, for severe and critical COVID-19 patients.
Rare disease-focused Ovid Therapeutics Inc. has paused development of its investigational Angelman syndrome therapy, OV-101, after initial phase III results showed no difference between the drug and a placebo.
Investors in Minerva Neurosciences Inc. on Dec. 1 gave a chilly reception to reported FDA feedback on the company's experimental treatment for the negative symptoms in schizophrenia, roluperidone. Though the drug failed to deliver statistically significant differences vs. placebo in a phase III study this year, CEO Remy Luthringer outlined a potential path to NDA filing for the candidate using a modified intent-to-treat analysis of the trial and evidence from other ongoing evaluations.
Moderna Inc., late on Nov. 30, said it has asked the FDA to bless emergency use of its COVID-19 vaccine mRNA-1273 after phase III data confirmed it to be 94.1% effective in preventing symptomatic cases of the disease and 100% effective in preventing severe cases. The Cambridge, Mass.-based company said efficacy was "consistent across age, race and ethnicity and gender demographics."
Rhythm Pharmaceuticals Inc., a company developing medicines for rare genetic disorders of obesity, has won FDA approval for its first product, Imcivree (setmelanotide), following a priority review. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger. The approval covers the treatment of three types of ultrarare early onset obesity tied to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) and leptin receptor (LEPR) deficiencies, when confirmed by genetic testing.
Moderna Inc., late on Nov. 30, said it has asked the FDA to bless emergency use of its COVID-19 vaccine mRNA-1273 after phase III data confirmed it to be 94.1% effective in preventing symptomatic cases of the disease and 100% effective in preventing severe cases. The Cambridge, Mass.-based company said efficacy was "consistent across age, race and ethnicity and gender demographics."
Just ahead of the U.S. celebration of Thanksgiving, the FDA granted an accelerated approval for Danyelza (naxitamab), a new therapy for certain adults and children with relapsed or refractory high-risk neuroblastoma. The antibody, which Y-mabs Therapeutics Inc. initially sublicensed via a deal with Mabvax Therapeutics Inc. in 2018, is approved for use in combination with granulocyte macrophage colony-stimulating factor. The regulator had assigned the BLA a Nov. 30 PDUFA date.<
An antibody cocktail developed by Regeneron Pharmaceuticals Inc. has received emergency use authorization (EUA) from the FDA for the treatment of mild to moderate COVID-19. Monoclonal antibodies (Mabs) such as Regeneron's, called casirivimab and imdevimab, "have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load," the company said.
Citing a lack of evidence that it improves survival, the need for ventilation or time to clinical improvement, the World Health Organization (WHO) has advised doctors against using Gilead Sciences Inc.’s antiviral Veklury (remdesivir) to treat COVID-19.
