It’s clear skies ahead for bluebird bio’s lovo-cel gene therapy for sickle cell disease, after the biotech confirmed that the US Food and Drug Administration (FDA) will not schedule an advisory committee meeting (FDA) to discuss the therapy.

An approval decision can now be expected for lovo-cel or lovotibeglogene autotemcel before the Prescription Drug User Fee Act (PDUFA) goal date of 20 December 2023. If greenlit, it will be bluebird bio’s third FDA-approved gene therapy, adding to Zynteglo (betibeglogene autotemce) and Skysona (elivaldogene autotemcel) for the treatment of thalassemia and cerebral adrenoleukodystrophy, respectively.

According to bluebird bio, lovo-cel is the most extensively studied gene therapy in development for sickle cell disease – a condition where long term survival is achieved by bone marrow transplants, a somewhat risky procedure. A range of medicines approved for the disease focus on preventing red blood cells from clumping together or seek to reduce symptoms.

bluebird bio’s therapy aims to treat the underlying genetic cause of the disease that approximately 100,000 Americans currently live with. Lovo-cel is a one-time treatment providing functional copies of a modified β-globin gene, which helps red blood cells produce anti-sickling haemoglobin. As part of its BLA, bluebird bio submitted efficacy data from 36 patients and safety data from 50 patients. Included in the programme are six patients with six or more years of follow-up, which the biotech says is the longest follow up of any gene programme for sickle cell disease.

bluebird bio isn’t the only one on the regulatory road for sickle cell disease – Vertex and CRISPR Therapeutics submitted a BLA to the FDA in April 2023 for its collaborative therapy exa-cel (exagamglogene autotemcel).

There have been hurdles along the way, including when the FDA suspended lovo-cel clinical trials due to a suspected unexpected serious adverse reaction. However, the case of acute myeloid leukaemia in question was later deemed unrelated. The biotech remains on track to be the first to unlock a significant portion of the sickle cell disease market – estimated to reach US sales of $4.8bn by 2030, according to a report by GlobalData.

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bluebird bio’s CEO Andrew Obenshain said: “Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease and represents the third lentiviral vector gene therapy that the Agency has reviewed from bluebird—giving us great confidence in the robustness and maturity of our BLA package. We remain focused on working with the Agency on its review in anticipation of a decision by the end of this year.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva

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