Berotralstat capsules

Trade Name: Orladeyo™
Company: BioCryst Pharmaceuticals
Approval Dates/Comments: In December 2020, the US FDA approved oral, once-daily berotralstat, a plasma kallikrein inhibitor, indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients aged ≥12 years. The approval is based on data from the Phase 3 APeX-2 trial, which showed that berotralstat could significantly reduce attacks at the 24-week mark, with the reduction sustained through 48 weeks. Subjects who completed 48 weeks of treatment in the Phase 3 trial experienced reductions in HAE attack rates, from an average of 2.9 attacks per month at baseline, to a mean of 1 attack per month. In the long-term APeX-S trial, those who completed 48 weeks of therapy had an average monthly attack rate of 0.8.

Vixarelimab for SC use

Trade Name: KPL-716
Company: Kiniksa Pharmaceuticals

Approval Dates/Comments: The FDA granted Breakthrough Therapy designation to vixarelimab in November 2020 for the treatment of pruritus associated with prurigo nodularis. Vixarelimab is a fully-human monoclonal antibody (mAb) that targets oncostatin M receptor beta (OSMRβ), which mediates signaling of interleukin-31 and oncostatin M, two key cytokines implicated in pruritus, inflammation, and fibrosis. Vixarelimab is believed to be the only mAb in development that simultaneously targets both pathways.

Ivermectin lotion 0.5%

Trade Name: Sklice®
Company: Arbor Pharmaceuticals

Approval Dates/Comments: In October 2020, the FDA approved a lotion to treat head lice for nonprescription, or over-the-counter (OTC), use through a process called a prescription (Rx)-to-OTC switch. It was initially approved for treating head lice infestation in patients ≥6 months of age as a prescription drug in February 2012.

Gene therapy for DEB

Trade Name: AGLE-102™
Company: Aegle Therapeutics

Approval Dates/Comments: The FDA granted Rare Pediatric Disease designation in October 2020 to AGLE-102™ for the topical treatment of dystrophic epidermolysis bullosa (DEB). AGLE-102™ is a composite of mesenchymal stem cell-derived extracellular vesicles that deliver proteins, genetic material and regenerative healing factors to diseased and damaged tissue. AGLE-102™ has the potential to be the first multifaceted approach to treat this rare patient population.

Ingenol mebutate gel

Trade Name: Picato®
Company: Leo Pharma

Approval Dates/Comments: Following Health Canada’s safety review that concluded a potential link between Picato® and an increased risk of nonmelanoma skin cancer, Leo Pharma has withdrawn this drug from the Canadian market. Picato® was an approved topical treatment for non-hyperkeratotic, non-hypertrophic actinic keratosis in adults. On October 26, 2020, recall of the drug was initiated from the Canadian market to the retail pharmacy level. Healthcare professionals are advised to:

  • not prescribe or dispense Picato®.
  • contact patients under their care who are currently being treated with Picato® to stop treatment, and review alternative treatment options.
  • counsel patients who have been treated with Picato® to contact a healthcare professional if they experience signs or symptoms of skin cancer, such as new scaly red patches on their skin, open sores, or elevated or warty growths within the treatment area, which could occur after stopping treatment.

Leo Pharma has already withdrawn or is in the process of withdrawing Picato® in the Europe, Australia, and the US.

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