|Alnylam Pharmaceuticals Inc., of Cambridge, Mass.||Lumasiran||RNAi therapeutic||Primary hyperoxaluria type 1||U.K.’s Medicines and Healthcare products Regulatory Agency granted a positive scientific opinion through EAMS (early access to medicines scheme), allowing eligible PH1 patients to gain access to drug prior to marketing authorization|
|Clinigen Group plc, of Burton Upon Kent, U.K.||Aldesleukin||Non-glycosylated interleukin-2 product||Amyotrophic lateral sclerosis||FDA granted orphan designation|
|Cytodyn Inc., of Vancouver, Washington||Leronlimab||CCR5 antagonist||HIV||Received a refuse to file letter from FDA regarding BLA seeking approval as combination therapy with HAART for highly treatment-experienced patients; company intends to request type A meeting with FDA to discuss request for additional information; FDA request does not require any additional clinical trials|
|Enzychem Lifesciences Inc., of Englewood, N.J.||EC-18||CCL26 gene inhibitor; TLR modulator||Acute respiratory distress syndrome due to COVID-19 pneumonia||Filed IND with FDA for a phase II trial testing use as preventive therapeutic|
|Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany||BNT-162b1 and BNT-162b2||mRNA-based vaccines||COVID-19||FDA granted fast track designation to both|
|Protokinetix Inc., of Marietta, Ohio||PKX-001||AAGP molecule||Type 1 diabetes||Submitted amendments to Health Canada; upon approval, company will deliver 75 grams of drug to University of Alberta to accommodate ongoing trial; study will treat islet cells prior to transplantation in type 1 diabetes patients|
For more information about individual companies and/or products, see Cortellis.