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.

Development of abuse-deterrent opioid products, including reformulation of existing products, has become a priority for drug manufacturers and public health advocates. Since our last article on this subject, several new abuse-deterrent opioid formulations have been approved by the FDA, including an oxycodone tablet formulation (Roxybond – Inspirion) that is the first immediate-release opioid product FDA-approved to include claims of abuse deterrence in its labeling. No opioid formulation prevents consumption of a large number of intact dosage units, the most common method of...

Deflazacort (Emflaza – PTC Therapeutics), an oral corticosteroid, has been approved by the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients ≥5 years old. It has been available outside the US for many years. Deflazacort is the second drug to be approved for treatment of DMD; eteplirsen (Exondys 51), an antisense oligonucleotide approved for IV administration in patients with mutations of the dystrophin gene amenable to exon 51 skipping (about 13% of DMD cases), was the first.

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 a progestin-only oral contraceptive ("minipill") containing drospirenone (Slynd – Exeltis). All other progestin-only oral contraceptives available in the US contain norethindrone (Camila, and others). Progestin-only oral contraceptives are similar in efficacy to combination oral contraceptives. They are used predominantly by breastfeeding women and by those in whom estrogen is poorly tolerated or contraindicated. Combination oral contraceptives containing drospirenone and ethinyl estradiol have been available for years.

Caplacizumab-yhdp (Cablivi – Sanofi /Genzyme), a von Willebrand factor-directed antibody fragment, has been approved by the FDA for use in combination with plasma exchange and immunosuppressive therapy for treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP; formerly called acquired thrombotic thrombocytopenic purpura [aTTP]) in adults. It is the first drug to be approved in the US for this indication.

Mobocertinib (Exkivity – Takeda), an oral kinase inhibitor, has received accelerated approval from the FDA for treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations in adults whose disease has progressed on or after platinum-based chemotherapy. Accelerated approval was based on the overall response rate and duration of response. Mobocertinib is the second drug to become available in the US for this indication; the IV EGFR-MET bispecific antibody amivantamab (Rybrevant) was approved...

The oral kinase inhibitors dabrafenib (Tafinlar – GSK) and trametinib (Mekinist – Novartis) have received accelerated approval by the FDA for use together for a fifth indication: treatment of unresectable or metastatic solid tumors with a BRAF V600E mutation in patients ≥6 years old who have progressed following prior treatment and have no satisfactory alternative treatment options. The combination is not approved for treatment of colorectal cancer because of known intrinsic resistance to BRAF inhibition and dabrafenib is not approved for use in patients with wild-type BRAF...

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.

Colchicine (Colcrys, and others), which has been available in the US for decades for prophylaxis and treatment of gout flares and other indications in oral formulations that contain 0.6 mg of the drug, has now been approved in 0.5-mg tablets as Lodoco (Agepha) to reduce the risk of myocardial infarction (MI), stroke, coronary revascularization, and cardiovascular death in adults with established atherosclerotic disease or multiple risk factors for cardiovascular disease.