Roctavian (valoctocogene roxaparvovec), the gene therpay for hemophilia A, gets nods as holding the most promise among the drugs in the pipeline.
Roctavian (valoctocogene roxaparvovec), the gene therpay for hemophilia A, gets nods as holding the mRoctavian (valoctocogene roxaparvovec), the gene therpay for hemophilia A, gets nods as holding the most promise among the drugs in the pipeline.ost promise among the drugs in the pipeline.
Payers, providers, pharmaceutical companies all keep an eye on the “drug pipeline,” industry shorthand for drugs in mid- and late-stage development. FDA approval is a finish line and 14 new drugs have crossed that threshold so far this year, including the Leqembi (lecanemab), a new Alzheimer’s drug, in January, and just last week, Qalsody (tofersen), a drug for a small minority of people with amyotrophic lateral sclerosis who have certain gene changes.
The 2023 Annual Managed Healthcare Executive Pharmacy Surveyasked respondents to rate which drugs “hold the most promise” among several in the much-heeded pipeline. Valoctocogene roxaparvovec, a gene therapy for hemophilia A, garnered the most support (33%). The FDA is currently expected to announce whether it has approved on June 30. BioMarin Pharmaceutical Inc. is planning to market valoctocogene roxaparvovec under the brand name Roctavian.
After Roctavian, 27% of the 311 respondents ranked zuranolone, a treatment for major depressive disorder and postpartum depression, as holding the most promise, followed by the vaccines for respiratory syncytial virus (17%), tirzepatide, marketed as Mounjaro, for weight loss (16%) and resmetirom (6%), a new drug for nonalcoholic steatohepatitis, or NASH.
The survey also asked respondents to which gene therapies will have the most significant impact. Zynteglo (betibeglogene autotemcel) for beta thalassemia garnered the most selections, followed by Hemgenix (etranacogene dezaparvovec) for hemophilia B, Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy and Adstiladrin (nadofaragene firadenovec) for bladder cancer.
The online survey was conducted Feb. 14-28. The respondents were spread fairly evenly across the various sectors of the U.S. healthcare industry, with 20% self-identifying as working for hospitals, 22% for physician practices, 19% for pharmacy benefit managers, 16% for payer organizations, 13% for drug and device manufacturers and the rest in a variety of sectors.