Lenti-D (elivaldogene autotemcel or eli-cel) is an investigational gene therapy being developed by bluebird bio for the treatment of childhood cerebral adrenoleukodystrophy (CALD). The FDA advisory committee reviewed it in June 2022 and unanimously approved drug/therapy 15-0. The FDA’s scheduled decision date on the therapy is September 16, 2022.
Read moreGene Therapy Update: Beti-cel
This week a decision should be made by the FDA on a new gene therapy, betibeglogene autotemcel (beti-cel). Beti-cel is a one-time autologous product designed to treat transfusion-dependent beta thalassemia.
Read moreCell and Gene Therapies: Navigating a Challenging Landscape
Cell and gene therapy will most certainly continue to shift paradigms for manufacturers, patients, healthcare providers and pharmacies for many years. These are groundbreaking technologies which offer hope to millions of individuals that previously had no viable options for treatment or cure.
Read moreAre You Ready? Preparing for Gene and Cell Therapies Coming Down the Pipeline
Gene and cell therapies can be life changing but come with expensive price tags. As more therapies are FDA approved, payers are looking for options to control the spiraling cost of specialty pharmacy and to project future financial risk related to new therapies coming to market.
Read moreGene Therapy for Hemophilia A: Valrox may be the world’s most expensive one-time treatment
Should it be approved, BioMarin is considering pricing Valrox, a gene therapy drug aimed at hemophilia A, at $2 million to $3 million for the single treatment.
Read moreZolgensma and Gene Therapy: What is the Cost?
Gene therapy in healthcare has become a reality, and it is set to move from a trickle to a stream in the next few years. This article discusses Zolgensma and other emerging gene therapies in the context of how they may affect a health plan’s costs.
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