Liso-cel for CLL/SLL, Tislelizumab for Esophageal SCC, Resmetirom for NASH, Maralixibat for Cholestatic Pruritus, Guselkumab for UC

New FDA Approvals

Please visit nascentmc.com/podcast for all the details. Go to learnAMAstyle.com for lots of freebies on AMA Style and the use of AI in medical writing and editing Here is information on the latest US FDA approvals, the week of March 11 – March 15, 2024 Liso-cel for CLL/SLL - The FDA approved lisocabtagene maraleucel (liso-cel; Breyanzi) for adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior therapies. It is a CAR T-cell therapy that modifies patient's T cells to target tumor antigens. The approval was based on the TRANSCEND CLL 004 study, showing a 20% complete response rate and a median duration of response not reached by the data cutoff.

Tislelizumab for Esophageal SCC - Tislelizumab-jsgr (Tevimbra) received FDA approval for unresectable or metastatic esophageal squamous cell carcinoma (ESCC) patients after prior systemic chemotherapy. The approval was based on the phase 3 RATIONALE 302 trial, which showed significant improvement in overall survival compared to chemotherapy (8.6 months vs. 6.3 months). This marks a critical advancement for patients with limited treatment options after first-line failures.

Resmetirom for NASH - The FDA approved resmetirom (Rezdiffra) for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced fibrosis, to be used alongside diet and exercise. This is the first medication approved to directly address liver damage in NASH patients with significant liver scarring, acting as a partial activator of a thyroid hormone receptor to reduce liver fat. The approval, based on a 54-month trial, used a surrogate endpoint at 12 months to demonstrate improvement in liver scarring and inflammation.

Maralixibat for Cholestatic Pruritus - Maralixibat (Livmarli) oral solution was approved for treating cholestatic pruritus in patients aged 5 years and older with progressive familial intrahepatic cholestasis (PFIC). It is an orally administered ileal bile acid transporter inhibitor, showing efficacy in the Phase 3 MARCH clinical trial across various genetic types of PFIC. Additionally, a higher concentration formulation is under consideration to extend its use to younger PFIC patients.

Guselkumab for UC - A supplemental Biologics License Application (sBLA) has been submitted for guselkumab (Tremfya) for treating adults with moderate-to-severely active ulcerative colitis (UC). The submission is based on the QUASAR program results, demonstrating significant clinical remission at Week 44 compared to placebo. Guselkumab, a novel IL-23 inhibitor, has previously been approved for moderate-to-severe plaque psoriasis and active psoriatic arthritis, marking its potential expansion into UC treatment.