Massachusetts-based bluebird bio BLUE is a biotechnology company focused on developing gene therapies for severe genetic diseases and cancer.
bluebird’s performance has been impressive after the company reported several positive news over the past two months.
In September, the company received an accelerated FDA approval for Syskona (elivaldogene autotemcel), also known as eli-cel, as a treatment for patients younger than 18 years with early, active cerebral adrenoleukodystrophy (CALD).
The company also announced that the clinical hold previously imposed by the FDA on the eli-cel development program in August 2021 has now been lifted on Sep 15, 2022, before the review of the Syskona biologics license application (BLA).
However, we remind investors that the continued approval of Syskona for CALD may depend upon verification and description of clinical benefit in a confirmatory study.
Thus, as a condition of Syskona’s accelerated approval, bluebird will have to provide confirmatory long-term clinical data to the FDA from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years and from commercially treated patients.
With the accelerated approval in place, the company expects to launch Syskona commercially in the United States by the end of 2022 to enable patient access to gene therapy as soon as possible.
Syskona is the second ex-vivo lentiviral vector gene therapy to be approved by the FDA in the United States.
The first is bluebird’s Zynteglo (betibeglogene autotemcel), also known as beti-cel, which the FDA approved in August as the first cell-based gene therapy. The therapy treats beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions across all genotypes.
The FDA approvals boost bluebird bio’s growth prospects, but the successful commercialization of these therapies holds the key. Moreover, gene therapies are complex by nature.
bluebird will face stiff competition in the target market. CRISPR Therapeutics CRSP and its partner Vertex Pharmaceuticals VRTX announced that the FDA granted a rolling review to the gene therapy exagamglogene autotemcel (exa-cel), formerly known as CTX001. The companies are developing the therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Vertex intends to submit its BLA for exa-cell for a rolling review by the FDA by the start of November and expects to complete the process by the end of first-quarter 2023.
These approvals have provided bluebird two priority review vouchers, which are transferable. These vouchers can help shorten the review process of a new drug application (NDA) from 10 months to six months. With the intent of exploring additional financing opportunities, bluebird intends to monetize these vouchers in the near term.
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