HEMATOLOGY

Vertex Pharmaceuticals has secured FDA approval for CASGEVY, a one-time CRISPR/Cas9 gene-edited cell therapy, for the treatment of TDT in patients ages12 and older, providing a potentially curative option for those facing significant health challenges. Roughly 1,000 patients in the U.S. are now eligible for the treatment, 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) using CRISPR/Cas9 technology in which they have employedan innovative muscle-directed adeno-associated viral(AAV) vector-mediated gene therapy. Treatment effectively normalized glycogen storage pathology,restored muscle mass and strength, and normalizedsurvival rates.

CELL & GENE THERAPY

Sarepta Therapeutics has announced that the FDA has accepted 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 from accelerated to traditional, to which the FDA has granted a Priority Review with a goal date of June 21, 2024.

HEPATOLOGY

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

CARDIOLOGY

The FDA has accepted BridgeBio Pharma’s New DrugApplication (NDA) for acoramidis, a potential treatment for transthyretin amyloid cardiomyopathy (ATTR-CM),with a Prescription Drug User Fee Act (PDUFA) set for November 29, 2024. Positive Phase 3 results from ATTRibute-CM, showed significant improvements in survival rates and hospitalizations. The company is advancing 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 the treatment of splenomegaly and symptoms in adult myelofibrosis(MF) patients with moderate to severe anemia. Ojjaara is indicated for newly diagnosed and previously treated patients, offering a unique mechanism of action targeting: JAK1, JAK2, and ACVR1.Approval is based on positive results from MOMENTUM and SIMPLIFY-1 Phase 3 trials, demonstrating significant symptom relief and spleen size reduction, while enhancing transfusionindependence. Ojjaara will be marketed as Omjjara in the EU.