Newly approved gene therapies targeting sickle cell disease will be the first focus of the U.S. Centers for Medicare & Medicaid Services’ (CMS) Cell and Gene Therapy Access Model, the agency said Jan. 30.
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
Access to advanced therapies proved to be a major talking point at a conference in London, following the U.S. approval of Bluebird Bio Inc.’s Zynteglo (betibeglogene autotemcel) cell-based gene therapy for beta thalassemia and its $2.8 million price tag. Regulators in Europe backed Zynteglo in 2019 but Bluebird opted to withdraw the therapy in 2021 after deciding that the complex thicket of pricing bodies in Europe was too difficult to negotiate.
Bluebird Bio Inc. isn’t giving out much of the information on the margins for the cost of its newly approved cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia. The numbers will, the company said, come into better focus when another Bluebird drug is approved and launched.
The U.S. FDA has approved the first cell-based gene therapy for treating adult and pediatric patients with beta-thalassemia requiring frequent red blood cell transfusions. The $2.8 million wholesale acquisition cost for the one-time I.V. infusion will make it one of the most expensive drugs in the U.S.
The Cellular, Tissue and Gene Therapies Advisory Committee scrutinized Bluebird Bio Inc.’s gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.
Wall Street took in stride mixed FDA briefing documents with regard to the upcoming adcom review of Bluebird Bio Inc.’s two gene therapy prospects, and shares of the company (NASDAQ:BLUE) closed at $3.61, up 63 cents, or 21%.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
There was no slowing of biopharma innovation in 2021, even as industry directed significant resources to, while feeling the impact of, the ongoing COVID-19 pandemic. The year saw big wins for developers of DNA vaccines and biosimilars, while CAR T expanded its reach and a drug target once considered undruggable was finally conquered. And as 2021 gives way to 2022, other potentially game-changing technologies and therapeutics are waiting in the wings.
