Capvaxive Pneumococcal Conjugate Vaccine, Elevidys for DMD, Krazati for CRC, Keytruda for Endometrial Cancer, Skyrizi for UC, Tremfya for Crohn’s Disease

New FDA Approvals

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• The FDA has approved the 21-valent pneumococcal conjugate vaccine, CAPVAXIVE™ (Merck), for the prevention of invasive disease and pneumonia in adults aged 18 years and older caused by 21 Streptococcus pneumoniae serotypes. Capvaxive includes eight serotypes not covered by other pneumococcal vaccines, addressing approximately 27% of IPD cases in adults aged 50 and older, and 30% in adults aged 65 and older, based on CDC data from 2018-2021. The approval follows an FDA Priority Review and is based on immune responses measured in the Phase 3 STRIDE-3 trial, with continued approval contingent upon verification of clinical benefit in a confirmatory trial.

• The FDA has approved delandistrogene moxeparvovec-rokl (Elevidys) for Duchenne muscular dystrophy (DMD) in ambulatory individuals aged 4 and older with a confirmed mutation in the DMD gene, as well as granting accelerated approval for non-ambulatory individuals. Elevidys, a one-time intravenous gene therapy, delivers a working copy of the DMD gene to address the muscle degeneration caused by mutations in this gene. The approvals are based on findings from a confirmatory trial that, while not meeting its primary endpoint, showed success in several secondary measures, with the Phase 3 ENVISION study underway to serve as a postmarketing requirement.

• The FDA has approved adagrasib (Krazati) plus cetuximab for adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC) who have received prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Adagrasib targets the KRAS G12C mutation, a common driver mutation in several cancers including colorectal cancer, while cetuximab enhances its antitumor activity. The approval was based on findings from the KRYSTAL-1 trial, which demonstrated a confirmed overall response rate (ORR) of 34% and a median duration of response (DOR) of 5.8 months.

• The FDA has approved pembrolizumab (Keytruda) in combination with carboplatin and paclitaxel, followed by pembrolizumab monotherapy, to treat primary advanced or recurrent endometrial carcinoma in adults, marking the third endometrial carcinoma indication for Keytruda in the US. Keytruda enhances the body's immune response against tumor cells by blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2. The approval is based on results from the phase 3 KEYNOTE-868 clinical trial, which demonstrated significant improvements in progression-free survival for patients treated with Keytruda plus chemotherapy compared to those receiving a placebo with chemotherapy.

• The FDA has approved risankizumab-rzaa (Skyrizi) for the treatment of moderately to severely active ulcerative colitis in adults, making it the first specific anti–interleukin 23 monoclonal antibody indicated for both ulcerative colitis and moderate to severe Crohn's disease. Risankizumab-rzaa inhibits interleukin-23 (IL-23), a cytokine involved in inflammatory and immune responses, thereby reducing inflammation. The approval is based on data from two phase 3 clinical trials, INSPIRE and COMMAND, which demonstrated the achievement of clinical remission and endoscopic improvement.

• A supplemental Biologics License Application (sBLA) has been submitted for guselkumab (Tremfya) for the treatment of adults with moderately to severely active Crohn's disease. Guselkumab, a fully-human, dual-acting monoclonal antibody that blocks IL-23 and binds to CD64, was previously approved for moderate-to-severe plaque psoriasis and active psoriatic arthritis. Support for the BLA is based on findings from the Phase 3 GALAXI and GRAVITI clinical trials.