Axatilimab IV injection
Trade Name: SNDX-6352
Company: Syndax Pharmaceuticals
Approval Dates/Comments: The US FDA granted Orphan Drug designation to axatilimab in March 2021 for the treatment of patients with chronic graft versus host disease (cGVHD). Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor (CSF-1R), a cell surface protein known to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor was shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, thereby blocking the development of cutaneous and pulmonary cGVHD. The Phase 1 trials enrolled 15 subjects, with 67% experiencing significant improvements in symptoms. Axatilimab data has demons trated durable responses and multiorgan clinical benefit in patients refractory to multiple therapeutic agents to date, and is currently being evaluated in a pivotal Phase 2 AGAVE-201 trial in patients with cGVHD.
Binimetinib tablets + encorafenib capsules
Trade Name: Mektovi® + Braftovi®
Company: Pfizer Canada
Approval Dates/Comments: In March 2021, Health Canada approved these kinase inhibitors indicated for oral combination treatment of patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation, as detected by a validated test. Binimetinib (a small molecule MEK inhibitor) + encorafenib (a small molecule BRAF inhibitor) in combination is the first targeted treatment to demonstrate over 30 months median overall survival in a Phase 3 trial. Both drugs target key enzymes in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Inappropriate activation of proteins in this pathway has been shown to occur in many cancers including melanoma. FDA approval for this combination therapy was gained in June 2018.
Encapsulated cell therapy for Fabry disease
Trade Name: SIG-007
Company: Sigilon Therapeutics
Approval Dates/Comments: The FDA granted Orphan Drug designation to SIG-007 in March 2021 for the treatment of Fabry disease, a progressive, life-threatening lysosomal disorder. SIG-007 is comprised of cells that are genetically modified with a non-viral vector to express human alpha-galactosidase A, or AGAL.
Oritavancin IV injection
Trade Name: Kimyrsa™
Company: Melinta Therapeutics
Approval Dates/Comments: In March 2021, the FDA approved oritavancin, a lipoglycopeptide antibiotic, for the treatment of adult patients with acute bacterial skin and skin structure infections (ABSSSI) caused by susceptible isolates of designated Grampositive microorganisms, including methicillin-resistant Staphylococcus aureus (MRSA). Oritavancin is administered as a one-time, single-dose by intravenous (IV) infusion. The efficacy and safety of Kimyrsa™ were demonstrated in the SOLO clinical trials with another oritavancin product, Orbactiv®. The SOLO trials were randomized, double-blind, multicenter studies that investigated a single 1,200 mg IV dose of oritavancin against twice-daily vancomycin for the treatment of ABSSSI in 1,987 adult patients and assessed one of the largest subsets of documented MRSA infection (405 patients). These trials showed that 1,200 mg one-dose IV oritavancin infusion was as effective as 7-10 days of twice-daily vancomycin (1 g or 15 mg/kg) for the primary and secondary endpoints.