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Ingrezza for Huntington’s:

• The FDA has approved valbenazine (INGREZZA® SPRINKLE) capsules for adults with tardive dyskinesia and chorea associated with Huntington's disease.
• This new oral granules formulation provides an alternative for those with swallowing difficulties, maintaining the same dosage strengths as the original capsule form.
• Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor designed to reduce uncontrollable movements by inhibiting dopamine release.

Tivdak for Cervical Cancer:

• Tisotumab vedotin-tftv (TIVDAK) has been approved for recurrent or metastatic cervical cancer after chemotherapy failure.
• This approval was based on results from a Phase 3 trial showing a significant overall survival benefit compared to chemotherapy.
• The approval highlights a significant advancement in treating this aggressive form of cancer, granted to Pfizer and Genmab A/S.

Xolremdi for WHIM:

• Mavorixafor (Xolremdi) has been approved for WHIM syndrome in patients 12 years and older to increase circulating neutrophils and lymphocytes.
• WHIM syndrome is a rare genetic disorder impairing immune function, and mavorixafor significantly improves cell counts and reduces infection risk.
• Approval was based on positive results from a double-blind trial and granted to X4 Pharmaceuticals.

Esprit Stent for CLTI-BTK:

• The FDA has approved the Esprit™ BTK Everolimus Eluting Resorbable Scaffold System for chronic limb-threatening ischemia below-the-knee.
• This is the first approval of its kind in the U.S., offering an alternative to traditional balloon angioplasty.
• The approval was based on positive outcomes from the LIFE-BTK trial, showing significant disease progression reduction compared to standard care, granted to Abbott Laboratories.

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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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