When it comes to innovation in the development of new drugs and therapeutic biological products, FDA’s Center for Drug Evaluation and Research (CDER) supports the pharmaceutical industry at every step of the process. The FDA continuously makes strides in approving essential drugs for the medical industry to improve the lives of many from newborns to adulthood.
- Amondys 45– February 25
Amondys 45 (casimersen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. It binds to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping.
- Nulibry– February 26
Nulibry (fosdenopterin) is an injection created to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A, a rare, genetic, metabolic disorder that typically presents in the first few days of life, causing intractable seizures, brain injury and death. Patients with Molybdenum Cofactor Deficiency Type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP) that can now be replaced with Nulibry.
- Pepaxto– February 26
Pepaxto is the first prescription medicine used in combination with dexamethasone to treat adults with multiple myeloma (MM) who did not respond to or stopped responding to at least four prior medicines, including at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. It is designed to leverage aminopeptidases, which are overexpressed in MM cells and cause the release of the cytotoxic payload.
- Azstarys– March 2
Azstarys is a once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients ages six and older. ADHD is one of the most common mental disorders affecting children. Azstarys consists of serdexmethylphenidate (SDX), KemPharm’s prodrug of d-methylphenidate (d-MPH), co-formulated with immediate-release d-MPH.
- Fotivda– March 10
Fotivda® (tivozanib) is an oral, once daily, differentiated vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI). It is a potent, selective inhibitor of VEGFRs 1, 2, and 3 with a long half-life designed to improve efficacy and tolerability. Fotivda has been approved for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models.
- Ponvory– March 18
Ponvory™ is a prescription medicine that is used to treat adults with relapsing forms of multiple sclerosis. The approval covers clinically isolated syndrome, relapsing-remitting MS (RRMS), and active secondary progressive MS (SPMS), in adults.
- Zegalogue– March 22
Zegalogue (dasiglucagon) injection is the first and only prescription medicine used to treat very low blood sugar (severe hypoglycemia) in adults and children with diabetes aged 6 years and older. It essentially kickstarts the pancreas to release glucagon, which tells the liver and muscle cells to convert stored energy into glucose and release that into the bloodstream to raise sugar levels.
- Qelbree– April 2
Qelbree (i.e. SPN-812) is a nonstimulant ADHD medication containing a serotonin norepinephrine modulating agent approved by the FDA to treat attention deficit hyperactivity disorder (ADHD or ADD) in patients 6 to 17 years of age. It is the first nonstimulant ADHD medication to receive FDA approval for use in children since Intuniv was approved in 2009.
- Nextstellis– April 15
Nextstellis is the first and only contraceptive pill containing estetrol, a native estrogen, now produced from a plant source. Estetrol is the first new estrogen introduced in the US in more than 50 years. In two phase 3 clinical studies it was shown to be both safe and effective. It also delivered excellent results on a variety of secondary endpoints that demonstrated outstanding cycle control, bleeding control, safety, and tolerability.
- Jemperli– April 22
Jemperli is indicated for the treatment of adult patients with mismatch repair deficient (dMMR) recurrent or advanced endometrial cancer (EC), as determined by an FDA-approved test, that has progressed on or following prior treatment with a platinum-containing regimen. This indication is approved under accelerated approval based on tumor response rate and durability of response.
- Zynlonta– April 23
Zynlonta™ is indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from low-grade lymphoma, and high-grade B-cell lymphoma. This CD19-targeted antibody drug conjugate (ADC) has been granted accelerated approval by the FDA based on overall response rate.
- Empaveli– May 14
Empaveli is the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, chronic, life-threatening blood disorder caused by an acquired mutation, which leads to uncontrolled complement activation and the destruction of red blood cells through intravascular and extravascular hemolysis. Empaveli has the potential to improve the lives of patients with PNH by increasing hemoglobin and reducing blood transfusion requirements.
For drug approvals for 2020 and previous years, check out the FDA website to learn more about each of these pharmaceutical advances.