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Two cell-based gene therapies to treat sickle cell disease — Casgevy (exagamglogene autotemcel or exa-cel) and Lyfgenia (lovotibeglogene autotemcel or lovo-cel) — received FDA approval Friday, marking the first treatments to provide potential life-long elimination of severe disease symptoms. Read More
As action dates for two groundbreaking sickle cell gene therapies loom, U.S. Senator Bill Cassidy, MD (R-LA), is raising questions about the cost, and how these extremely expensive, but life-changing, treatments will be paid for and allocated. Read More
Drug containers contaminated with wood, cellulose, brass and steel as well as multiple corrective and preventive actions (CAPA) to address mold are among the reasons the FDA listed in its untitled letter to Novartis Pharmaceuticals over its biological product Kymriah (tisagenlecleucel). Read More
In an expansion of its investigation into suicidal ideation associated with GLP-1 agonists, the European Medicines Agency (EMA) wants manufacturers to answer some undisclosed questions about these drugs, while the popularity of these drugs also continues to expand. Read More
Aldeyra Therapeutics on Monday announced receipt of an FDA complete response letter (CRL) for the NDA of reproxalap, for the treatment of dry eye disease, asking the company to conduct an additional trial to show the efficacy of the drug. Read More
Continued efforts to obtain approval for a drug to treat a rare neuromuscular disorder appear in this month’s Research Roundup along with an oral anticoagulant, and treatments for peanut allergy in children and migraine. Read More
Nearly half of rare oncology drug approvals include postmarketing requirements (PMR) for drug-drug interactions (DDI), a particular concern for cancer patients who are frequently being treated for co-morbidities and palliative care. Read More
In this edition of Quick Notes, we review recent FDA drug approvals of combination drug Truqap/Faslodex for breast cancer, DefenCath for hemodialysis patients, non-small cell lung cancer treatment Augtyro, a 38th approval for Keytruda, and an approval for Xtandi for prostate cancer. Read More
Merck appears destined for another disappointment with its investigative cough suppressant gefapixant, after an FDA advisory committee almost universally panned the drug’s phase 3 data, citing an almost complete overlap of patient-reported outcomes, a 57 percent placebo response rate, and a 28 percent dropout rate related to taste disturbance, the drug’s biggest side effect. Read More