The evaluation of drugs by FDA has always been the focus of pharmaceutical research all over the world, and the approval of NME (New molecular entity) to market is the top priority. From January to June 2019, FDA approved a total of eight NME launches, including the first drug for indications, the first target inhibitor, which is worthy of understanding.
In the first half of 2019, FDA approved a total of eight NME. They are Triclabendazolet (clonorchiasis), Brexanolone (postpartum depression), Sslriamfetol (narcolepsy), Siponimod Fumaric Acid (recurrent multiple sclerosis), Erdafitinib (uroepithelial carcinoma), Tafamidismeglumine (polyneuropathy), Alpelisib (advanced metastatic breast cancer), Bremelanotide acetate (Sexual desire disorder in postmenopausal women).
- Trichlorbendazole
Trichlorbendazole, a DNA methyltransferase inhibitor developed by Novartis, was approved by FDA on February 13, 2019, for the treatment of clonorchiasis in patients aged 6 and older, trade name Egaten®. Its approval is expected to facilitate wider access to this important drug in the United States and in affected countries around the world.
- Brexanolone
Brexanolone, γ-aminobutyric acid A receptor regulator, developed by Ligand (authorized by SAGE Therapeutics in 2011), to be awarded breakthrough therapy for postpartum depression by FDA in the United States in 2016. In November 2017, a phase III clinical project, Hummingbird, provided positive data that showed that Brexanolone significantly alleviated PPD symptoms compared with placebo, with efficacy lasting up to 30 days, while Brexanolone was safe and well tolerated.
In April 2018, the company submitted NDA, to FDA on March 19, 2019, which was approved by FDA in the United States for the treatment of postpartum depression. The drug is the first and only drug to obtain indications for postpartum depression, under the trade name Zulresso®.
- Solriamfetol
Solriamfetol, an orphan drug originally developed by SK Biopharmaceuticals of Korea, is a selective dopamine and norepinephrine reuptake inhibitor (NRI). Approved by the United States FDA on March 20, 2019, for the treatment of daytime excessive narcolepsy in adults with paroxysmal narcolepsy or obstructive sleep apnoea, sold by Jazz Pharmaceuticals in the United States under the name Sunosi®.
Siponimod, the Sphinginol-1-phosphate receptor (SIPR) regulator, selectively binds to SIPR1 and SIPR5, preventing lymphocytes from leaving the lymph nodes, therefore preventing them from entering the central nervous system. The mechanism of therapeutic role in multiple sclerosis is unclear and may be related to reducing the migration of lymphocytes to the central nervous system. The drug is used in the treatment of adult patients with recurrent multiple sclerosis (MS). These include clinical isolation syndrome, recurrent and remission multiple sclerosis and active secondary progressive multiple sclerosis, known as Mayzent®.
Erdafitinib, is a kinase inhibitor that binds to and inhibits the activity of fibroblast growth factor receptor FGFR1/FGFR2/FGFR3/FGFR4. In March 2018, FDA awarded Erdafitinib a breakthrough therapy for urothelial cancer. On April 12, 2019, it was approved by the United States FDA for the treatment of adult patients with locally advanced or metastatic uroepithelial cancer (who carry specific FGFR3 or FGFR2 gene mutations and continue to progress after platinum chemotherapy) under the trade name Balversa®.
- Tafamidis Meglumine
Tafamidis meglumine, is a new type of specific thyroxine protein stabilizer for the treatment of transthyroxine protein familial amyloid polyneuropathy (TTR-FAP). In 2012, Tafamidis meglumine was certified by the United States FDA and the European Union EMA as an orphan drug for the treatment of thyroxine protein familial amyloid polyneuropathy (TTR-FAP). In 2018, Tafamidis meglumine was certified by Japanese PMDA as an orphan drug for transthyroxine protein familial amyloid polyneuropathy (TTR-FAP). In May 2018, Tafamidis meglumine was certified by the United States FDA as a breakthrough therapy for transthyroxine protein cardiomyopathy. On May 3, 2019, it was approved by the FDA of the United States for the treatment of thyroxine familial amyloid polyneuropathy under the trade name Vyndaqel®.
Alpelisib, is a phosphatidylinositol 3-kinase α (PI3K- α) inhibitor. PI3K-AKT-mTOR signal cascade is a key regulator of angiogenesis, which can up-regulate the metabolic activity of tumor cells. It was approved for sale by FDA in the United States on May 24, 2019. It was combined with the endocrine therapeutic drug Fulvestrant. Used to treat postmenopausal women and men with specific advanced or metastatic breast cancer, which is also the first FDA approved PI3K inhibitor, trade name Piqray ®.
Bremelanotide, was developed by AMAG Pharmaceuticals and targeted at melanin 4 receptor (MC4R). It was approved by FDA on June 21, 2019, for the treatment of sexual disorder in premenopausal women under the trade name Vyleesi®.
References
1. Barone, A., Casey, D., McKee, A. E., & Reaman, G. (2019). Cancer drugs approved for use in children: Impact of legislative initiatives and future opportunities. Pediatric blood & cancer, e27809.
2. Markman, J., Gudin, J., Rauck, R., Argoff, C., Rowbotham, M., Agaiby, E., … & Lu, L. (2019). SUMMIT-07: a randomized trial of NKTR-181, a new molecular entity, full mu-opioid receptor agonist for chronic low-back pain. Pain, 160(6), 1374-1382.
3. Blumenthal, G. M., & Pazdur, R. (2019). Approvals in 2018: a histology-agnostic new molecular entity, novel end points and real-time review. Nat Rev Clin Oncol, 16(3), 139-141.