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Drug Pipeline
Gene Therapy for Hemophilia Is on the Brink of FDA Approval
November 17, 2022
CSL Behring's gene therapy, called EntranaDez, may be approved later this month. BioMarin, Pfizer, Bayer and Freeline Therapeutics also have gene therapies for hemophilia in development.
Removing the CD47 ‘Do Not Eat Me’ Sign
November 03, 2022
Gilead’s magrolimab is the leading CD47 immune checkpoint inhibitor in development.
Drugs on the Fast Track as Treatments for PTSD
October 10, 2022
Several drugs with novel mechanisms of action have been put on the pathway for speedier approval decisions by the FDA. Phase 2 and 3 trials are underway.
Gene Therapies for Hemophilia. Possibly Curative, Likely Ultraexpensive
September 08, 2022
Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.
For Plaque Psoriasis Treatment, Abundance Gets More Company
August 12, 2022
Nonsteroidal topical agents and TYK2 inhibitors may join an already full armamentarium.
Zynteglo May Herald “New Dawn” of Many Seven-Figure Gene Therapies
August 05, 2022
An OptumRx drug pipeline report highlights Zynteglo, a curative gene therapy treatment for beta-thalassemia, and Skysona, a gene therapy for a cerebral adrenoleukodystrophy. FDA approval decision are imminent for both. Price tags of $2 million are expected.
Review Paper Argues for Broader Use of Riabni, a Rituxan Biosimilar
June 03, 2022
The review, partly funded by Amgen, the maker of Riabni, makes the case for using the biosimilar to Rituxan for all indications of the originator.
Mark Cuban Says He Has the Prescription for High, Opaque Drug Pricing
April 12, 2022
Cuban, an investor on “Shark Tank” and owner of the NBA’s Dallas Mavericks, has launched a namesake online pharmacy that says it will have transparent pricing and will bypass pharmacy benefit managers.
Multiple Sclerosis - Some Drugs New, Some Tried-and-True
April 10, 2022
The race is on to be the first-approved Bruton tyrosine kinase inhibitor. Meanwhile, trials are underway to test statins and metformin as treatments for multiple sclerosis.
Fourth Shot is Recommended for the Immunocompromised
March 10, 2022
Getting boosted is now the rule, not the exception, when it comes to COVID-19 vaccination recommendations. The CDC now recommends that everyone, ages 12 and older, get a dose of an mRNA COVID-19 vaccine, either Pfizer’s or Moderna’s. For people who have gotten the two-shot Pfizer or Moderna series, the booster is their third jab. For those who got the single-dose Johnson & Johnson vaccine, it is the second.