New FDA Approvals Emma Hitt Nichols, PhD

Visit nascentmc.com/podcast for full show notes.
- 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.
Vist LearnAMAStyle.com for several essential tip sheets that you can download as well as a free course on using AI in medical writing, and an AMA style quiz to test your knowledge

·       Nascentmc.com for medical writing assistance for your company.
Visit nascentmc.com/podcast for full show notes
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.  

Visit nascentmc.com/podcast for full show notes
Winrevair for PAH: Sotatercept-csrk (Winrevair) is FDA-approved for treating adults with pulmonary arterial hypertension (PAH), enhancing exercise capacity, improving WHO functional class, and reducing clinical worsening events. It's the first FDA-approved activin signaling inhibitor for PAH, addressing the imbalance in vascular cell proliferation underlying the condition. The approval is based on the Phase 3 STELLAR trial, demonstrating significant improvements in walk distance and reduced risk of clinical worsening events [1]. Vafseo for CKD: Vadadustat (Vafseo) is approved for managing anemia due to chronic kidney disease (CKD) in adult dialysis patients. As an oral HIF-PH inhibitor, it stimulates endogenous erythropoietin production, offering a novel approach to anemia management. Approval is supported by efficacy and safety data from the INNO2VATE program and post-marketing safety data from Japan [2]. Ultomiris for NMOSD: Ravulizumab-cwvz (Ultomiris) is FDA-approved for treating neuromyelitis optica spectrum disorder (NMOSD) in patients with anti-AQP4 antibodies. It's a terminal complement C5 inhibitor administered once every two months, demonstrating efficacy in preventing relapses. Approval is based on the Phase 3 CHAMPION-NMOSD study, showing significant reductions in relapse risk compared to placebo [3]. Evolut FX+ for TAVR: The Evolut™ FX+ transcatheter aortic valve replacement (TAVR) system is FDA-approved for treating symptomatic severe aortic stenosis. It features enhancements for improved catheter maneuverability without compromising effectiveness. The approval expands treatment options for patients across all risk categories [4]. Vemlidy for Pediatric HBV: Tenofovir alafenamide (Vemlidy) is FDA-approved for treating chronic hepatitis B virus (HBV) infection in pediatric patients aged 6 years and older with compensated liver disease. It's a preferred or first-line treatment option, addressing a significant medical need. Approval is based on the Phase 2 clinical trial 1092, demonstrating efficacy and safety in this patient population [5]. Pemgarda for COVID: Pemgarda, a monoclonal antibody, is FDA-approved for preventive use in immunocompromised individuals aged 12 and older against COVID-19. It prevents virus attachment and has shown promising results in reducing symptomatic COVID-19 cases. Approval is based on emergency use authorization and preliminary data from the CANOPY Phase 3 clinical trial [6].

Nascentmc.com for medical writing assistance for your company.
Visit nascentmc.com/podcast for full show notes
Visit learnAMAstyle.com for free downloads regarding editing and the AMA Manual of Style tip sheet. 
Nexletol and Nexlizet for LDL Lowering and CV Risk: The FDA approved bempedoic acid (Nexletol) and its combination with ezetimibe (Nexlizet) for reducing cardiovascular risk and treating primary hyperlipidemia. Bempedoic acid inhibits a cholesterol synthesis enzyme, while ezetimibe blocks cholesterol absorption, both lowering LDL-C levels. The approval, for high-risk patients not yet having cardiovascular events, was based on the CLEAR Outcomes trial. Iclusig for ALL: The FDA granted accelerated approval to ponatinib (Iclusig) with chemotherapy for treating previously untreated Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib, a tyrosine kinase inhibitor, is the first targeted agent for this leukemia type in combination with chemotherapy, based on the PhALLCON trial results. Further confirmatory data may be required to verify its clinical benefit. Elahere for Ovarian Cancer: Mirvetuximab soravtansine-gynx (Elahere) received FDA approval for treating folate receptor alpha-positive, platinum-resistant ovarian cancer, marking it as the first antibody-drug conjugate in the U.S. for this condition. The treatment targets cancer cells by binding to the folate receptor alpha, delivering a cytotoxic agent. Approval was based on the Phase 3 MIRASOL trial. Duvyzat for DMD: The FDA approved givinostat (Duvyzat) for patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare neuromuscular condition. Givinostat is an HDAC inhibitor that mitigates muscle damage and slows disease progression. Approval was based on the EPIDYS trial results, granted to Italfarmaco S.p.A. Spevigo for Psoriasis: Spesolimab-sbzo (Spevigo) received FDA approval for treating generalized pustular psoriasis (GPP) in adults and pediatric patients, expanding its indication from initial approval for GPP flares. As the first targeted therapy for GPP, it acts as an IL-36 receptor antagonist. The expanded approval was based on the Effisayil 2 trial, showing significant reduction in GPP flares. Tryvio for Hypertension: The FDA approved aprocitentan (Tryvio) in combination with other antihypertensive drugs for adults not adequately controlled on other medications, marking it as the first new oral antihypertensive therapy pathway in nearly 40 years. Based on the PRECISION trial, aprocitentan was shown to be effective in patients with resistant hypertension. Opsynvi for PAH: Macitentan and tadalafil (Opsynvi) was approved by the FDA for adults with pulmonary arterial hypertension (PAH) and WHO functional class II-III, as the first once-daily single-tablet combination therapy for PAH. The approval was based on the A DUE study, demonstrating greater reduction in pulmonary vascular resistance compared to monotherapies. Lenmeldy for Juvenile Metachromatic Leukodystrophy: The FDA approved Lenmeldy (atidarsagene autotemcel) gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. The therapy uses the patient's own genetically modified stem cells to produce the missing enzyme. Approval was based on significant improvements in survival, mobility, and cognitive functions observed in clinical trials and an expanded access program.

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.
 

Here is information on the latest US FDA approvals, the week of March 4 – March 8, 2024
·         ChatGPT4 in medical writing and editing—visit learnAMAstyle.com
·       Nascentmc.com for medical writing assistance for your company.

Visit nascentmc.com/podcast for full show notes 
 - **OTC Glucose Monitor**: The FDA has approved the Dexcom Stelo Glucose Biosensor System for over-the-counter sale, a first for a continuous glucose monitor. Designed for people aged 18 and older not using insulin, it helps manage diabetes with oral medications or monitors the impact of diet and exercise on blood sugar levels. Scheduled for release in Summer 2024, the system offers a 15-day sensor wear time and does not alert users to low blood sugar episodes.
 
- **Semaglutide in CVD Risk**: The FDA has approved semaglutide (Wegovy) for reducing cardiovascular risk in adults with known heart disease who are overweight or obese, specifically targeting the reduction of major adverse cardiovascular events. This approval makes semaglutide the first weight-loss medication also indicated for preventing life-threatening cardiovascular events in patients with established cardiovascular disease and obesity or overweight.
 
- **Juvederm Additional Indication**: JUVÉDERM® VOLUMA® XC, a hyaluronic acid dermal filler, has received FDA approval for treating moderate to severe temple hollowing in adults over 21, marking it as the first HA filler for this purpose. With effects lasting up to 13 months, clinical studies show significant improvement and patient satisfaction with facial symmetry post-treatment. This approval highlights Allergan Aesthetics' commitment to innovation in aesthetic treatments.
 
- **Nivolumab in mUC**: The FDA approved nivolumab in combination with cisplatin and gemcitabine for first-line treatment of metastatic urothelial carcinoma, based on significant improvements in survival outcomes from the CHECKMATE-901 trial. This expands nivolumab's indications, which include treatments for melanoma and lung cancer, among others, demonstrating its broad applicability in cancer treatment.
 
- **Zanubrutinib in FL**: The FDA has granted accelerated approval to zanubrutinib and obinutuzumab for relapsed or refractory follicular lymphoma patients after two or more systemic therapies. This combination targets key pathways in B cell survival, offering a new treatment option for patients. Approval was based on the ROSEWOOD trial, highlighting significant patient outcome improvements.
 
- **Donanemab and Alzheimer’s**: The FDA has postponed the decision on the approval of Eli Lilly's donanemab for Alzheimer's treatment to convene an advisory meeting for further examination of safety and efficacy data, indicating a significant delay. This reflects the complex nature of Alzheimer's drug approval and Eli Lilly's confidence in donanemab's potential benefits.
 
- **Tocilizumab Biosimilar**: Tyenne® (tocilizumab-aazg), the first FDA-approved biosimilar to Actemra® for various inflammatory diseases, is now available in both IV and subcutaneous formulations. This approval introduces a new treatment option for patients with conditions like rheumatoid arthritis and juvenile idiopathic arthritis, emphasizing advancements in biosimilar medications.
 
- **Denosumab Biosimilars**: The FDA approved Jubbonti and Wyost as interchangeable biosimilars to Prolia and Xgeva, respectively, marking a first for biosimilars targeting the RANKL inhibitor used in osteoporosis and cancer-related bone conditions. These approvals offer new treatment options for managing bone health, underlining the importance of biosimilar development in expanding patient care.
 
- **Clobetasol Propionate Eye Drops**: The FDA's approval of clobetasol propionate 0.05% eye drops for post-operative eye inflammation and pain introduces the first ophthalmic formulation of this corticosteroid and the first new steroid in ophthalmo