CRISPR Therapeutics AG (NASDAQ: CRSP) expects to submit an application for gene therapy approval by the end of the year. This medicine already earned good evaluations from the US regulator, thus approval can be acquired even sooner than the typical timeframes.
Exa-cel (previously CTX001) gene therapy is the primary contender for CRISPR Therapeutics, with the goal of helping patients with transfusion-dependent beta-thalassemia (TDT) and sickle cell anaemia (SCD). Vertex Pharmaceuticals is CRISPR Therapeutics’ collaborator in these projects. Both businesses recently revealed plans to submit documentation to US and EU authorities for exa-cel clearance by the end of the year.
Exa-cel has already been designated as an FDA Advanced Therapy in the field of regenerative medicine. This indicates that real approval may occur sooner than the customary timeframe of 6-10 months.
Exa-cel therapy has been found in studies to be particularly successful in the treatment of patients with TDT and SCD. 42 of 44 TDT patients who received treatment did not require blood transfusions for up to 37 months. Thirty-one exa-cel-treated SCD patients did not have vaso-occlusive crises over a two to 32.3-month follow-up period. Previously, these patients had 3.9 crises every year on average.
According to a recent study, SDC patients with private health insurance spend over $1.7 million on disease-related medical expenditures during their lifetime. Exa-cel is intended to be a one-time medication that will help SDC and TDT patients lessen their financial burden. Despite the fact that the treatment is likely to be quite expensive.
Despite a modest target market and probable competition from other genetic editing approaches, exa-cel therapy might become a blockbuster. Bluebird Bio, for example, is on the approach of receiving regulatory clearance in the United States for their version of the TDT therapy.
According to the conditions of the collaboration with Vertex Pharmaceuticals, biotech CRISPR Therapeutics will earn 40% of the income and shoulder 40% of the costs of commercializing exa-cel. We believe that the arrangement with Vertex Pharmaceuticals was a wise decision since it decreases the possibility that exa-cel would meet unexpected regulatory barriers.