The FDA has approved Amondys 45 (casimersen), an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 45 skipping. This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45, which is reasonably likely to predict clinical benefit for those patients who are exon 45 amenable.
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The FDA has approved Azstarys (dexmethylphenidate/serdexmethylphenidate), a once-daily central nervous system stimulant for the treatment of attention-deficit/hyperactivity disorder in patients aged 6 years and older. Azstarys capsules are available as 26.1mg/5.2mg, 39.2mg/7.8mg, and 39.2mg/7.8mg capsules.
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The FDA has approved Breyanzi (lisocabtagene maraleucel), a cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or who have relapsed after, at least two other types of systemic treatment. Breyanzi, a chimeric antigen receptor T cell therapy, is the third gene therapy approved by the FDA for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma. Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.
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The FDA has approved Cosela (trilaciclib) for injection to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small-cell lung cancer. It is the first and only therapy designed to help with myeloprotection when administered prior to treatment with chemotherapy.
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The FDA has approved Evkeeza (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol-lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia. Evkeeza is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3, a protein that plays a key role in lipid metabolism.
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The FDA has approved Nulibry (fosdenopterin) for injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency Type A. This is the first therapy of its kind.
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The FDA has approved Pepaxto (melphalan flufenamide), in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. This indication has been granted under accelerated approval. Pepaxto is the first anticancer peptide-drug conjugate approved in multiple myeloma.
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The FDA has approved Ukoniq (umbralisib) for the treatment of adult patients with relapsed or refractory marginal zone lymphoma who have received at least one prior anti-CD20 based regimen and adult patients with relapsed or refractory follicular lymphoma who have received at least three prior lines of systemic therapy. Ukoniq is the first and only oral, once-daily, inhibitor of phosphoinositide 3 kinase delta and casein kinase 1 epsilon.
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The FDA has approved Vazalore 325mg and Vazalore 81mg (referred to together as “Vazalore”), the first-ever novel, liquid-filled aspirin capsule. Vazalore provides patients with vascular disease and diabetic patients who are candidates for aspirin therapy based on physician recommendation with fast, reliable, and predictable platelet inhibition as compared to enteric-coated aspirin. It also reduces the risk of stomach erosions and ulcers, as compared to immediate-release aspirin, common in an acute setting.
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The FDA has approved an expanded indication for Entresto (sacubitril/valsartan). The indication is to reduce the risk of cardiovascular death and hospitalization for heart failure in adult patients with chronic heart failure. For the first time, there is a treatment with benefit for patients diagnosed with guideline-defined heart failure that includes both those with heart failure with reduced ejection fraction and many with heart failure with preserved ejection fraction.
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The FDA has approved Libtayo (cemiplimab-rwlc) for the first-line treatment of patients with advanced non-small-cell lung cancer whose tumors have high PD-L1 expression (tumor proportion score ≥50%), as determined by an FDA-approved test. This is the third approval for Libtayo.
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