Here is information on the latest US FDA approvals, the week of February 26 – March 1, 2024
Amivantamab for NSCLC
The FDA has approved amivantamab (Rybrevant) in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test. This approval converts the May 2021 accelerated approval of amivantamab to full approval. The approval was based on the results from the Phase 3 PAPILLON study, which demonstrated that amivantamab plus chemotherapy reduced the risk of disease progression or death by 61 percent versus chemotherapy alone in patients with previously untreated NSCLC with EGFR exon 20 insertion mutations. EGFR exon 20 insertion mutations in NSCLC patients generally result in limited benefits from current third-generation EGFR tyrosine kinase inhibitors and chemotherapy, carrying a worse prognosis and shorter survival rates compared to lung cancer driven by other EGFR driver mutations. This targeted approach is the first approved for the first-line treatment of NSCLC patients with EGFR exon 20 insertion mutations. The FDA granted the approval of amivantamab to Johnson & Johnson.
https://www.prnewswire.com/news-releases/rybrevant-amivantamab-vmjw-in-combination-with-chemotherapy-is-the-first-fda-approved-therapy-for-first-line-treatment-of-patients-with-non-small-cell-lung-cancer-with-egfr-exon-20-insertion-mutations-302077581.html
First Coronary Drug-Coated Balloon
The FDA has approved the AGENT™ Drug-Coated Balloon (DCB) for coronary in-stent restenosis (ISR) in patients with coronary artery disease, marking the first drug-coated coronary balloon to receive approval in the US. This device provides an alternative treatment for ISR, aiming to minimize recurrence risk. The AGENT DCB employs a paclitaxel-coated balloon catheter, delivering a therapeutic drug dose to the vessel wall. ISR affects roughly 10% of percutaneous coronary interventions in the US. The FDA’s approval was based on positive results from the AGENT IDE trial, which enrolled 600 patients at 40 US sites. A prespecified interim analysis of the initial 480 patients showed the AGENT DCB’s statistical superiority to uncoated balloon angioplasty in reducing target lesion failure (TLF) at 12 months. The trial also reported no definite or probable stent thrombosis and a significant decrease in the risk of myocardial infarction at the target vessel, along with low adverse event rates. Boston Scientific Corporation, which was granted approval for the AGENT DCB, states the device will be available in the US in the next few months.
https://www.prnewswire.com/news-releases/boston-scientific-receives-fda-approval-for-the-agent-drug-coated-balloon-302076861.html
Ibrutinib Oral Suspension
The FDA has approved an update to the ibrutinib (Imbruvica) label, introducing an oral suspension formulation for its existing indications, including chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), Waldenström’s macroglobulinemia (WM), and chronic graft versus host disease (cGVHD) after failure of systemic therapy. This marks the first time a Bruton’s tyrosine kinase (BTK) inhibitor is available in an oral suspension form, catering to patients with difficulty swallowing pills. Ibrutinib was initially approved in 2013 for mantle cell lymphoma (MCL) in patients who had undergone at least one prior therapy. The oral suspension aims to simplify treatment for about 5% of patients facing challenges with pill-swallowing. The approval of the Imbruvica oral suspension formulation was granted to Johnson & Johnson in collaboration with Pharmacyclics LLC, an AbbVie company.
https://www.pharmexec.com/view/fda-approves-imbruvica-label-expansion-to-include-oral-suspension-formulation
Epcoritamab for FL
The FDA has approved epcoritamab-bysp (Epkinly) for adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of therapy. This approval follows the agent’s previous authorization for treating relapsed or refractory third-line diffuse large B-cell lymphoma (DLBCL), establishing it as the first and only subcutaneously administered bispecific antibody for this patient group. The mechanism of action of epcoritamab involves binding to CD3 on T cells and CD20 on B cells, inducing T-cell-mediated killing of CD20+ cells. The FDA’s decision was based on findings from the Phase 1/2 EPCORE™ NHL-1 trial in patients with relapsed or refractory FL. AbbVie and Genmab are co-developing epcoritamab, with both companies sharing commercial responsibilities in the U.S. and Japan. AbbVie is responsible for further global commercialization.
https://www.prnewswire.com/news-releases/us-food-and-drug-administration-fda-grants-priority-review-of-epcoritamab-epkinly-for-difficult-to-treat-relapsed-or-refractory-follicular-lymphoma-fl-302071777.html?
Govorestat for Galactosemia
The FDA has accepted the New Drug Application (NDA) for govorestat (AT-007), a treatment for classic galactosemia, and granted it priority review. As a novel aldose reductase inhibitor (ARI), govorestat has shown potential in reducing galactitol levels and enhancing clinical outcomes among pediatric patients. The NDA is based on findings from the phase 3 ACTION-Galactosemia Kids study among other studies. Upon approval, govorestat will be the first treatment of its kind for galactosemia. The manufacturer, Applied Therapeutics states a PDUFA target action date of August 28, 2024.
Obeticholic Acid for PBC
The FDA has accepted a supplemental New Drug Application (sNDA) for obeticholic acid (Ocaliva) for the treatment of individuals with primary biliary cholangitis (PBC). Initially receiving accelerated approval in 2016, obeticholic acid is indicated for adult patients with PBC who are either without cirrhosis or have compensated cirrhosis and lack evidence of portal hypertension. It may be used in conjunction with ursodeoxycholic acid (UDCA) if the response to UDCA is inadequate, or as monotherapy in patients who are intolerant to UDCA. The sNDA includes data from post-marketing studies COBALT and Study 401, supplemented by real-world evidence from a U.S. claims database and international PBC patient registries, to confirm clinical benefits in PBC patients. PBC, a rare and progressive autoimmune liver disease, predominantly affects women over 40 and can result in bile acid accumulation in the liver, leading to inflammation and fibrosis. Without intervention, the disease may progress to cirrhosis, necessitate a liver transplant, or lead to death. Obeticholic acid’s mechanism of action involves modulating the farnesoid X receptor (FXR), a critical regulator of bile acid, inflammatory, fibrotic, and metabolic pathways. The FDA’s review will include an Advisory Committee meeting, with the Prescription Drug User Fee Act (PDUFA) target action date set for October 15, 2024. Intercept Pharmaceuticals has been granted approval for obeticholic acid.
Biktarvy in HIV
The FDA has approved an expanded indication for Biktarvy (a combination of bictegravir, emtricitabine, and tenofovir), for the treatment of people with HIV who have suppressed viral loads and are dealing with known or suspected M184V/I resistance, a common form of treatment resistance. Biktarvy is an integrase strand transfer inhibitor (INSTI)-based single-tablet regimen (STR) first approved in 2018. The latest approval was based on 48-week data from Study 4030, a Phase 3, randomized, double-blind study focusing on virologically suppressed adults with HIV on a baseline regimen. Participants in this study were randomized to switch to Biktarvy or continue with their baseline regimen. Biktarvy is manufactured by Gilead Sciences.
Roluperidone for Schizophrenia
The FDA has rejected the NDA for Minerva Neurosciences’ roluperidone, which had been proposed to treat the negative symptoms of schizophrenia. While one study did show statistical significance, the FDA said among things in a Complete Response Letter that the study was “insufficient on its own [to provide] substantial evidence of effectiveness. Roluperidone has been shown to block serotonin, sigma and α-adrenergic receptors involved in the regulation of brain functions, including mood, cognition, sleep, and anxiety. Minerva has stated that they will request a meeting to discuss the issues raised.
https://ir.minervaneurosciences.com/news-releases/news-release-details/minerva-neurosciences-receives-complete-response-letter-fda-new
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