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Blueprint Medicines has enough pivotal data in hand to seek a new indication for Ayvakit (avapritinib) as a treatment for nonadvanced systemic mastocytosis and plans to file a supplemental new drug application later this year. Read More
The FDA has approved Bluebird Bio’s Zynteglo (betibeglogene autotemcel) for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Read More
With the announcement of a failed phase 3 breast cancer trial hot on the heels of a negative phase 2, Sanofi has decided to pull the plug on amcenestrant, an investigational oral selective estrogen receptor degrader (SERD). Read More
Piling more criticism on the already-embattled Emergent Biosolutions, the FDA has issued a warning letter citing significant sterility issues at the company’s Camden facility in Baltimore, Md., and stating that Emergent’s management oversight and control over the manufacture of drugs is inadequate. Read More
Drug and biologics manufacturers submitting electronic individual case safety reports (ICSR) and ICSR attachments to the FDA Adverse Event Reporting System database should follow a new technical guide the agency released yesterday. Read More
Merck and Orna Therapeutics announced that they are joining in a new partnership to advance development of the Cambridge, Mass., startup company’s RNA and lipid nanoparticle delivery platform to treat cancer, genetic disorders and infectious diseases. Read More