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- Fasenra Pediatric Asthma Expansion: No details provided for this update.

- Beqvez for Hemophilia B: FDA approved gene therapy, Beqvez, for adults with moderate to severe hemophilia B, which enables the production of clotting protein factor IX. This one-time treatment by Pfizer, derived from Spark Therapeutics, aims to replace frequent infusion therapies, demonstrating superior efficacy in a late-stage trial.

- Anktiva for Bladder Cancer: Anktiva received FDA approval for treating BCG-unresponsive non-muscle invasive bladder cancer, enhancing NK and T cell proliferation. Based on a trial with 77 patients, it showed a 62% complete response rate, surpassing international clinical benchmarks, leading to its designation as a breakthrough therapy.

- Pivya for UTI: FDA approved pivmecillinam (Pivya) for treating uncomplicated urinary tract infections caused by specific bacterial strains. This marks the first new antibiotic for such infections in the U.S. in over 20 years, backed by effective outcomes in three clinical trials.

- Tovorafenib for Pediatric Low-Grade Glioma: Tovorafenib was approved for pediatric low-grade glioma patients with specific BRAF alterations, showing a 67% response rate in the FIREFLY-1 trial. It has been designated for accelerated approval due to its potential in treating these brain tumors.

- Entyvio Maintenance for Crohn’s Disease: Vedolizumab (Entyvio) received approval for subcutaneous administration as Crohn’s disease maintenance therapy after initial intravenous induction. Supported by the VISIBLE 2 Study, it proved effective in maintaining clinical remission at 52 weeks.

- Alecensa for NSCLC: Alecensa was approved as an adjuvant treatment post-tumor resection for ALK-positive non-small cell lung cancer. In the ALINA trial, it significantly extended disease-free survival compared to chemotherapy, particularly in early-stage patients.

- SPG601 for Fragile X Syndrome: The FDA cleared SPG601 for a phase 2a trial in Fragile X Syndrome, addressing synaptic function through BK channel activation. This marks an advance for treating the core symptoms of the most common inherited intellectual disability.

- Lumisight for Visualizing Breast Cancer: The FDA approved Lumisight and the Lumicell Direct Visualization System for use during lumpectomy surgeries to detect residual cancer tissues. This system, shown in the INSITE trial, improves surgical outcomes by reducing the need for second operations.

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- The FDA has expanded the approval of benralizumab (Fasenra) to include children aged 6 to 11 with severe eosinophilic asthma, building on its previous approval for patients aged 12 and older. Benralizumab is a monoclonal antibody that targets and reduces eosinophilic inflammation, crucial in the severe asthma pathway. This approval was supported by the Phase 3 TATE study, which confirmed that the drug's effects in younger children were consistent with those seen in older patients.

- The FDA has approved new administration methods for cenobamate (Xcopri), allowing the tablets to be crushed for oral suspension or delivered via nasogastric tube for adults with partial-onset seizures. Cenobamate, initially approved in 2019, works by inhibiting neuronal firing and modulating the GABA ion channel. The approval follows a study confirming that these new methods are bioequivalent to the original oral intake of intact tablets.

- The FDA's advisory committee has recommended adopting minimal residual disease (MRD) as a new endpoint for the accelerated approval of multiple myeloma treatments. This recommendation is based on the EVIDENCE study, which highlighted MRD's ability to detect very low levels of disease, offering a quicker and more sensitive measure of treatment efficacy. If adopted, MRD could allow for faster approvals and potentially better patient outcomes in multiple myeloma.

- Labcorp's Mpox PCR Test Home Collection Kit has received FDA Emergency Use Authorization for diagnosing mpox in adults, marking the first at-home collection kit for this purpose. The kit uses PCR technology to detect the virus from lesion swabs collected by patients at home. This development comes in response to rising mpox cases and aims to improve accessible diagnostic options during public health emergencies.

- The FDA has approved the use of dolutegravir/lamivudine (Dovato) for HIV treatment in adolescents, making it the first oral, two-drug, single-tablet regimen for this age group. The combination targets crucial steps in the HIV replication process and aims to simplify lifelong treatment regimens, enhancing adherence. Approval was based on the DANCE study's findings, which demonstrated effective viral suppression in adolescents, consistent with adult data.

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• Cilta-cel for Myeloma: The FDA approved ciltacabtagene autoleucel (Carvykti; cilta-cel) for adults with relapsed or refractory multiple myeloma who have tried at least one prior therapy including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. This CAR T-cell therapy, initially approved in 2022, was confirmed effective in the phase 3 CARTITUDE-4 study, showing significant reduction in disease progression or death risk by 59% compared to standard care.
• Enhertu for HER2-positive Solid Tumors: Fam-trastuzumab deruxtecan-nxki (Enhertu) received FDA approval for treating unresectable or metastatic HER2-positive solid tumors in adults who have had previous systemic treatment and lack satisfactory alternative options. This therapy, a conjugate of an anti-HER2 antibody and a cytotoxic drug, was first approved in 2019 and targets HER2-expressing cancer cells to potentially minimize damage to normal tissues.
• Fanapt for Bipolar: Iloperidone (Fanapt) has been approved for the acute treatment of manic or mixed episodes in adults with bipolar I disorder. Previously approved for schizophrenia, iloperidone targets neurotransmitters like dopamine and serotonin. It demonstrated efficacy in a pivotal trial, showing significant improvement on the Young Mania Rating Scale.
• Zevtera for Multiple Bacterial Infections: Ceftobiprole medocaril sodium (Zevtera) was approved for treating adults with Staphylococcus aureus bloodstream infections, right-sided infective endocarditis, and acute bacterial skin and skin structure infections. Also approved for pediatric community-acquired bacterial pneumonia, ceftobiprole is a broad-spectrum cephalosporin that combats various bacteria including MRSA.
• TriClip for Tricuspid Regurgitation: The FDA approved the TriClip™ transcatheter edge-to-edge repair system for treating tricuspid regurgitation. This minimally invasive option clips the tricuspid valve leaflets to improve blood flow and prevent the need for surgery. The TRILUMINATE Pivotal trial showed significant improvements in TR severity and quality of life with a good safety profile.
• Revumenib for Acute Leukemia: The FDA granted priority review to revumenib (SNDX-5613) for treating adult and pediatric patients with relapsed or refractory acute leukemia with KMT2A rearrangements. As a new therapeutic agent, revumenib inhibits the menin-MLL protein interaction crucial in leukemic transformation. Early trial results show promising remission rates, with a PDUFA action date scheduled for September 26, 2024.