Novel FDA Drug Approvals 2Q 2023

drug development, drug discovery,

26 new drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER) for 1H! Lots of exciting new mechanisms and modalities.

Highlights from the 2Q include –

💉 Tofersen (Qalsody), an antisense oligonucleotide indicated for the treatment of adult amyotrophic lateral sclerosis caused by SOD1 mutation.

💉 Pegunigalsidase alfa (Elfabrio), a recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease.

💊 Fezolinetant (Veozah), a selective neurokinin-3 (NK3) receptor antagonist used to treat moderate to severe vasomotor symptoms due to menopause.

👁 Perfluorohexyloctane (Miebo) is a drug used to treat dry eye disease. Check out structure! Mechanism unknown.

💉Epcoritamab (Epkinly), a bispecific CD20-directed CD3 T-cell engager used to treat relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in adults.

💊 Sulbactam/durlobactam (Xacduro), a co-packaged medication used for the treatment of bacterial pneumonia caused by Acinetobacter baumannii-calcoaceticus complex. It contains sulbactam, a beta-lactam antibacterial and beta-lactamase inhibitor; and durlobactam, a beta-lactamase inhibitor.

💊 Nirmatrelvir/ritonavir (Paxlovid), a co-packaged medication used as a treatment for COVID‑19. It contains the antiviral medications nirmatrelvir and ritonavir. Nirmatrelvir is a SARS-CoV-2 main protease inhibitor while ritonavir is a HIV-1 protease inhibitor and strong CYP3A4 inhibitor.

💉Flotufolastat F-18 (Posluma), a radiopharmaceutical diagnostic agent used in PET imaging to visualize PSMA-positive lesions in men with prostate cancer with suspected metastasis or recurrence.

💊 Sotagliflozin (Inpefa), a dual SGLT1/2 inhibitor used to reduce the risk of CV, hospitalization for heart failure, and urgent heart failure visit in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors

💉Glofitamab (Columvi), a bispecific mAb directed against CD20 and CD3 which is used for the treatment of relapsed or refractory diffuse large B-cell lymphoma.

💊 Ritlecitinib (Litfulo), a dual JAK3/TEC kinase family inhibitor used to treat severe alopecia areata in adults and adolescents 12 years and older.

💉Rozanolixizumab (Rystiggo), a humanized mAb targeting the human neonatal Fc receptor (FcRn) used to treat generalized myasthenia gravis.

💉Somatrogon (Ngenla), a long-acting recombinant human growth hormone used as the long-term treatment of pediatric patients who have growth failure due to growth hormone deficiency.

Again, a fascinating collection from Chris de Savi!

New clinical trial upcoming using cyclodextrin as active

carbohydrates, , ,

Cyclo Therapeutics, Inc. sponsors Phase II trial for early Alzheimer’s disease. The study will enroll approximately 90 male and female patients aged 50 to 80 years who will be given a minimum active dose of 500 mg/kg of Trappsol Cyclo (equivalent to 18,500 mg/m2) as an intravenous (IV) infusion once every 28 days for 6 months.
We wish the best of success to our colleagues at Cyclo Therapeutics and hope this therapy will prove useful for the benefit of these patients.
Having programs ourselves in developing NCEs for neurodegenerative diseases, we find this study particularly essential.

Read more about the clinical trial here