The FDA has approved nusinersen (Spinraza – Biogen) for treatment of spinal muscular atrophy (SMA), a hereditary neurodegenerative disease that occurs in about one in every 10,000 births. It is the first drug to be approved in the US for this indication.

The FDA has approved avelumab (Bavencio – EMD Serono) and durvalumab (Imfinzi – AstraZeneca), two new immune check point inhibitors, and pembrolizumab (Keytruda – Merck), a checkpoint inhibitor that has been available in the US since 2014, for treatment of locally advanced or metastatic bladder cancer. Nivolumab (Opdivo) and atezolizumab (Tecentriq) were approved earlier for this indication.

The FDA has approved an oral powder formulation of the amino acid L-glutamine (Endari – Emmaus) to reduce the acute complications of sickle cell disease in patients ≥5 years old. Endari is only the second drug to be approved for use in sickle cell disease; the antimetabolite hydroxyurea (Droxia) was approved 20 years ago. L-glutamine oral powder is also available by prescription as NutreStore for treatment of short bowel syndrome and over the counter as a dietary supplement.

The oral kinase inhibitors selpercatinib (Retevmo – Lilly) and pralsetinib (Gavreto – Blueprint Medicines/Genentech) have been approved by the FDA for treatment of advanced or metastatic RET-mutant or RET fusion-positive cancers. They are the first drugs to be approved specifically for certain RET-driven cancers. Both drugs were granted accelerated approval based on overall response rates and the duration of response.

Amlodipine (am loe' di peen) besylate (Norvasc - Pfizer), a dihydropyridine calcium-channel blocker, has been approved by the US Food and Drug Administration for once-daily oral treatment of hypertension, chronic stable angina, and vasospastic (Prinzmetal's) angina.

Felbamate (Felbatol - Wallace Laboratories), a phenyl dicarbamate structurally similar to meprobamate (Equanil, and others), was recently approved by the US Food and Drug Administration for use alone or with other drugs in adults with partial seizures with or without secondary generalization. It was also approved for use in addition to other drugs in children with the multiple types of seizures associated with the Lennox-Gastaut syndrome, a severe epileptic encephalopathy.

Mirtazapine (mir taz a peen; Remeron - Organon) has been approved by the US Food and Drug Administration for treatment of depression. A tetracyclic piperazinoazepine, it is an analog of mianserin, an antidepressant available in Europe, but is structurally unrelated to antidepressants previously available in the USA.

Augmentation with a second-generation (atypical) antipsychotic is a treatment option for patients with major depressive disorder (MDD) whose symptoms persist following antidepressant monotherapy. Aripiprazole (Abilify), olanzapine in a fixed-dose combination with fluoxetine (Symbyax), and extended-release quetiapine (Seroquel XR) have been approved by the FDA for such use.

The FDA has recently approved intradetrusor injection of onabotulinumtoxinA (Botox – Allergan) for treatment of overactive bladder in patients who cannot tolerate or have an inadequate response to anticholinergic therapy. Botox is also approved by the FDA for use in detrusor overactivity associated with a neurologic condition such as multiple sclerosis or spinal cord injury, and for chronic migraine, upper limb spasticity, axillary hyperhidrosis, cervical dystonia, blepharospasm, strabismus, and cosmetic reduction of wrinkles.