HEMATOLOGY

Vertex Pharmaceuticals has secured FDA approval for CASGEVY, a one-timeCRISPR/Cas9 gene-edited cell therapy, for the treatment of TDT in patients ages12 and older, providing a potentially curative option for those facing significanthealth challenges. Roughly 1,000 patients in the U.S. are now eligible for thetreatment, alleviating the $5 million lifetime cost burden for existing treatments.

CELL & GENE THERAPY

Researchers have successfully generated the first ratmodel for Infantile-Onset Pompe Disease (IOPD) usingCRISPR/Cas9 technology in which they have employedan innovative muscle-directed adeno-associated viral(AAV) vector-mediated gene therapy. Treatmenteffectively normalized glycogen storage pathology,restored muscle mass and strength, and normalizedsurvival rates.

CELL & GENE THERAPY

Sarepta Therapeutics has announced that the FDA hasaccepted and filed the company's efficacy supplementfor ELEVIDYS, a gene transfer therapy for Duchennemuscular dystrophy (DMD) approved for ambulatorypediatric patients aged 4-5 with a confirmed DMD genemutation. The efficacy supplement aims to expand the labeledindication for ELEVIDYS to remove age and ambulationrestrictions and to convert the initial approval fromaccelerated to traditional, to which the FDA has granteda Priority Review with a goal date of June 21, 2024.

HEPATOLOGY

CymaBay Therapeutics announced that the FDA hasaccepted the New Drug Application (NDA) forseladelpar, a potential treatment for primary biliarycholangitis (PBC), granting it Priority Review with atarget action date of August 14, 2024. Seladelpar, aninvestigational PPARδ agonist, has shown promisingresults in reducing disease progression and addressingPBC-related symptoms such as pruritus in clinical trials.

CARDIOLOGY

The FDA has accepted BridgeBio Pharma’s New DrugApplication (NDA) for acoramidis, a potential treatmentfor transthyretin amyloid cardiomyopathy (ATTR-CM),with a Prescription Drug User Fee Act (PDUFA) set forNovember 29, 2024. Positive Phase 3 results fromATTRibute-CM, showed significant improvements insurvival rates and hospitalizations. The company isadvancing regulatory submissions globally, includingacceptance by the European Medicines Agency.

HEMATOLOGY

On January 29, 2024, the European Commission authorized GSK'sOjjaara (momelotinib), the first medicine in the EU indicated for thetreatment of splenomegaly and symptoms in adult myelofibrosis(MF) patients with moderate to severe anemia. Ojjaara is indicatedfor newly diagnosed and previously treated patients, offering aunique mechanism of action targeting: JAK1, JAK2, and ACVR1.Approval is based on positive results from MOMENTUM andSIMPLIFY-1 Phase 3 trials, demonstrating significant symptomrelief and spleen size reduction, while enhancing transfusionindependence. Ojjaara will be marketed as Omjjara in the EU.