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The FDA has accepted bluebird bio, Inc.'s biologics licence application (BLA) for the gene therapy lovotibeglogene autotemcel (lovo-cel) for sickle cell disease (SCD) patients aged 12 and older having a history of vaso-occlusive episodes (VOEs).
Sickle cell disease (SCD) is a genetic disease that affects people in a number of ways, including unpredictable pain crises, damage to vital organs, and even early death. High amounts of sickle haemoglobin (HbS) in red blood cells cause this condition, which causes the cells to become sticky, stiff, and short-lived. As a result, hemolytic anaemia, vasculopathy, and vaso-occlusion develop. Pain is a typical symptom that might be severe, necessitating hospitalisation.
In fact, more than half of adults with SCD have organ damage, and one in four experience a stroke by age 45. Unfortunately, numerous individuals with SCD do not live beyond the age of 40, so there are 100,000 people in the United States living with the disease presently.
Lovo-cel is a type of gene therapy currently researched as a treatment for sickle cell disease. The therapy involves modifying a patient's own blood stem cells so they can produce a special kind of hemoglobin that reduces the number of sickle-shaped red blood cells. bluebird bio, the company developing the therapy, has completed some clinical studies and is continuing others to check for safety and effectiveness. Some patients experienced side effects like infusion reactions and anemia, but they were mostly related to the disease or other treatments. A small number of patients also developed leukemia, but it was not due to the gene therapy itself. According to BioIntel360, the global cell and gene therapy market size is expected to record a CAGR of 26.2% during 2023-2027 to reach US$53,950.5 million by 2027, increasing from US$21,293.4 million in 2023. Over the last five years, the sector has recorded a CAGR of 28.0% to reach US$16,734.3 million in 2022.
Vertex Pharmaceuticals and CRISPR Therapeutics were able to move forward with an approved application for a rival medicine because the Massachusetts-based pharmaceutical company fell short of its deadline to submit an application by the end of March.
By late June, the organisation must determine whether to approve bluebird's application. The corporation is asking for a "priority," or expedited, review, which if approved would cut the review's duration from 10 months to six. In case a permission is given, Bluebird is preparing for an expedited evaluation and an early 2024 commercial launch.
Industry analysts have asserted that bluebird's Lovo-cel therapy is their most promising prospect for achieving commercial success. This is primarily due to the fact that a significantly higher number of individuals in the United States are affected by sickle cell disease compared to the rare disorders treated by the company's two approved gene therapies, Zynteglo and Skysona. As such, Lovo-cel has the potential to reach a larger patient population, increasing the company's overall market share and potential revenue. In light of this, Bluebird has prioritized Lovo-cel's development, investing significant resources into its clinical trials and regulatory approval processes. The success of Lovo-cel would undoubtedly cement Bluebird's position as a major player in the gene therapy space and set them on a path towards long-term success.
Beta globin is a component of the oxygen-carrying protein haemoglobin, and Lovo-cel inserts functional variants of this gene using lentiviruses. When the modified cells are reintroduced into a patient, they produce an anti-sickling haemoglobin that displaces the sickle cell-specific haemoglobin that is sticky and crescent-shaped. Clinical trials of the treatment showed an increase in total haemoglobin and the elimination of the severe pain crises that persons with the condition frequently experience. Like other gene treatments, safety concerns have hampered lovo-cel's development. Most recently, they caused a hold on a study in children and adolescents that was eventually lifted in December.
With its application, bluebird will be three weeks behind Vertex and CRISPR, who have already submitted their medication exa-cel for approval in beta thalassemia and sickle cell disease, two rare blood disorders. The therapies offered by the two companies modify a patient's harvested stem cells as well, but they do so using CRISPR to alter a gene that lowers foetal haemoglobin.
Although bluebird is a little behind in the race to find a cure for sickle cell anemia. A fierce competition has emerged between the leading biotechnology companies, including CRISPR Therapeutics, Vertex Pharmaceuticals, and bluebird bio. Despite being a key player in this race, bluebird bio has faced setbacks and has consequently seen a dip in its share market presence after the announcement.
BioIntel360 suggests that there is optimism that bluebird bio may have a future advantage due to its earlier introduction of two gene therapies and prospects will improve with the launch of its first therapy for sickle cell anemia. According to bluebird, the submission kept its timeframe "as highly similar" to that of its rivals. Being the first to launch a particular medication is highly significant for such therapies since patients undergo a single treatment, and switching is not a common practice, unlike chronic treatments.
This therapy is viewed as a great opportunity for the company to regain its competitive edge and establish itself as a key player in the field of genetic research and therapy. As the biotechnology industry continues to evolve, companies that can effectively harness the latest advancements in gene editing and genetic engineering will be well-positioned to shape the future of medicine and provide groundbreaking solutions for patients suffering from a wide range of genetic disorders.
