Clinical Trial News

Vertex's vanzacaftor/tezacaftor/deutivacaftor triple therapy NDA accepted by FDA for cystic fibrosis, targeting those aged 6+ with at least one F508del or responsive mutation in CFTR gene. PDUFA action date set for January 2, 2025, with EMA validation also received.

FDA approves FYB203/Ahzantive® (aflibercept-mrbb), a biosimilar to Eylea®, for treating nAMD, DME, DR, and RVO, inhibiting VEGF. FYB203/Ahzantive® demonstrated comparable efficacy and safety to Eylea®, with global sales of around US$ 9 billion in 2023. A marketing authorization application for FYB203 was submitted to the EMA, with a decision expected by early 2025.

FDA approves Nypozi (filgrastim-txid), a biosimilar to Neupogen, indicated to decrease the incidence of infection in patients with non-myeloid malignancies.

SpringWorks Therapeutics completes NDA submission to FDA for mirdametinib, an investigational MEK inhibitor for treating NF1-PN in children and adults. The submission includes data from the Phase 2b ReNeu trial, showing significant response rates, deep and durable responses, and a manageable safety profile. Mirdametinib has received Orphan Drug and Fast Track designations from the FDA and is planned for MAA submission to the EMA in the second half of 2024.

FDA approves Sandoz's biosimilar Pyzchiva® (ustekinumab-ttwe), developed by Samsung Bioepis, for all indications of Stelara®, including treating moderate to severe plaque psoriasis, psoriatic arthritis, Crohn's disease, and ulcerative colitis. Pyzchiva® is set to launch in the US in February 2025, aiming to provide affordable biosimilar alternatives for patients.

Shorla Oncology's Tepylute, a liquid form of thiotepa, simplifies oncology drug use, reducing medical personnel exposure to hazardous drugs. It's Shorla's third FDA-approved drug and their first in-house developed NDA, aiming to address unmet needs in cancer treatment.

U.S. FDA approves Epkinly (epcoritamab-bysp) for relapsed or refractory follicular lymphoma after two or more lines of therapy, making it the first subcutaneous T-cell engaging bispecific antibody for this patient population. Approval based on response rate; continued approval contingent on clinical benefit verification in confirmatory trials.

FDA issues Complete Response Letter for Daiichi Sankyo and Merck's patritumab deruxtecan BLA due to inspection findings at a third-party manufacturer, without issues identified with efficacy or safety data. Patritumab deruxtecan is a potential first-in-class HER3-directed ADC, jointly developed by Daiichi Sankyo and Merck, aiming to treat EGFR-mutated NSCLC patients previously treated with two or more systemic therapies.

AbbVie received a Complete Response Letter from the FDA for ABBV-951, a treatment for motor fluctuations in advanced Parkinson's disease, due to observations during inspection of a third-party manufacturer. The CRL does not raise issues on safety, efficacy, or labeling of ABBV-951.

Ionis Pharmaceuticals' olezarsen NDA accepted for Priority Review by FDA for FCS treatment, with action date set for December 19, 2024. Phase 3 sHTG program enrollment completed. Olezarsen targets apoC-III to lower triglyceride levels, reducing acute pancreatitis risk in FCS and sHTG patients.