Update on Drugs & Devices: May – June 2020

Trade Name: Koselugo®
Company: AstraZeneca, Merck

Approval Dates/Comments: The US FDA approved oral selumetinib in April 2020 for the treatment of pediatric patients ≥2 years of age with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib is the first drug approved by the FDA to treat this debilitating, progressive and often disfiguring rare disease that typically begins in early life. NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with a range of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (café au lait spots); in 30-50% of patients tumors develop on the nerve sheaths (PN). PN can cause disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction. Selumetinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) and functions by blocking a key enzyme that interferes with the growth of tumor cells. Serious side effects may result from treatment and include cardiomyopathy, ocular toxicity, severe diarrhea, skin rash, and muscle problems (rhabdomyolysis). Common side effects include vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, pyrexia, acneiform rash, stomatitis, headache, paronychia, and pruritus.

Trade Name: Eucrisa®
Company: Pfizer

Approval Dates/Comments: In March 2020, the FDA approved a supplemental new drug application to expand the indication of crisaborole to include treatment of mild-to-moderate atopic dermatitis (AD) in children ages 3 months to 24 months. Crisaborole is a topical phosphodiesterase-4 inhibitor that was previously approved in 2016 for patients aged ≥2 years. This new approval was based on the Phase 4 CrisADe CARE 1 study, a 4-week, multicenter, open-label, single-arm trial investigating the safety of twice daily crisaborole 2% use. There were 137 pediatric patients aged 3 months to <24 months who had mild-to-moderate AD involving at least 5% of BSA, excluding the scalp. The study found that the drug was well tolerated and effective with no difference in safety outcome compared with previous studies.

Trade Name: Avsola™
Company: Amgen Canada

Approval Dates/Comments: Health Canada approved this anti-tumor necrosis factor-alpha monoclonal antibody in March 2020 for all approved indications of the reference product, Remicade® (infliximab), including chronic severe plaque psoriasis and psoriatic arthritis.

Trade Name: Olumiant®
Company: Eli Lilly and Company, Incyte Corporation

Approval Dates/Comments: The FDA granted Breakthrough Therapy designation in March 2020 for baricitinib for alopecia areata (AA). Baricitinib is a oncedaily, oral Janus kinase inhibitor approved in the US under the brand name Olumiant® to treat moderate-to-severe rheumatoid arthritis. The expedited status is based on the positive Phase 2 results of the adaptive Phase 2/3 study BRAVE-AA1, which evaluated treatment with baricitinib vs. placebo in adults with AA. In the Phase 2 portion of the BRAVE-AA1 study up to Week 36, there were no new safety signals with no serious adverse events reported. The reported treatment-emergent adverse events were mild or moderate and the most common included upper respiratory tract infections, nasopharyngitis, and acne. Based on the interim results of the Phase 2 part of the study, the Phase 3 portion of BRAVE-AA1 and an additional Phase 3 double-blind study (BRAVE-AA2) are currently assessing the efficacy and safety of the 2 mg and 4 mg doses of baricitinib vs. placebo.