Both the U.K. MHRA and the U.S. FDA approved their first CRISPR-based gene therapy in 2023. Crispr Therapeutics AG and partner Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) was approved by the MHRA in November and the FDA on Dec. 8. The U.K. approval is for both severe sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT). In the U.S., the approval is for severe SCD, with a PDUFA date for TDT coming up in spring 2024.
All treatment groups in Vertex Pharmaceuticals Inc.’s phase II study of the non-opioid VX-548 showed meaningful reductions in pain from baseline in treating diabetic peripheral neuropathy (DPN), pointing the company toward pivotal phase III studies and prompting analysts to applaud enthusiastically.
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.
Vertex Pharmaceuticals Inc. has divulged heteroaryl compounds acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
Sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers have been reported in a Vertex Pharmaceuticals Inc. patent as potentially useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
The BioWorld Biopharmaceutical Index (BBI) fell through October, ending the month up just 0.98%. It remains above its low point of the year, when it was down 3.65% at the end of February.
Vertex Pharmaceuticals Inc. has identified cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
Vertex Pharmaceuticals Inc. has divulged cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
