Update for May 10, 2023

We recently added 16 new drugs to the knowledgebase:

• Senaparib a PARP inhibitor for the treatment of BRCA-positive ovarian cancer is being developed by Junshi Biosciences and Impact Therapeutics
• VE303 a microbiome-directed therapy for the prevention of recurrent Clostridium difficile infection is being developed by Vedanta Biosciences
• Dersimelagon a melanocortin 1 receptor agonist for the treatment of erythropoietic protoporphyria and X-linked protoporphyria is being developed by Mitsubishi Tanabe Pharma
• Pozelimab a complement factor C5 monoclonal antibody for the treatment of CHAPLE disease is being developed by Regeneron Pharmaceuticals and Alnylam
• ARX788 an anti-HER2 antibody drug conjugate for the treatment of HER2 positive breast cancer is being developed by Ambrx Biopharma and NovoCodex
• Ulixacaltamide a T-type calcium channel blocker for the treatment of essential tremor and Parkinson’s disease is being developed by Praxis Precision Medicines
• MK-0616 an oral PCSK9 inhibitor for the treatment of hypercholesterolemia is being developed by Merck
• Vorasidenib an IDH1 and IDH2 inhibitor for the treatment of glioma with an IDH1 or IDH2 mutation is being developed by Servier
• Inaxaplin an APOL1 channel function inhibitor for the treatment of focal segmental glomerulosclerosis is being developed by Vertex Pharmaceuticals
• Pegadricase/SVP-rapamycin a urate oxidase and mTOR pathway inhibitor for the treatment of chronic refractory gout is being developed by Selecta Biosciences and Sobi
• Povorcitinib a JAK inhibitor for the treatment of extensive nonsegmental vitiligo, hidradenitis suppurativa and prurigo nodularis is being developed by Incyte
• Pegozafermin a glycoPEGylated analog of fibroblast growth factor 21 (FGF21) for the treatment of non-alcoholic steatohepatitis (NASH) and severe hypertriglyceridemia is being developed by 89bio
• Furmonertinib an EGFR Tyrosine Kinase Inhibitor for the treatment of locally advanced or metastatic non-squamous non-small cell lung cancer with EGFR exon 20 insertion mutations. is being developed by Allist Pharmaceuticals and ArriVent Biopharma
• Botensilimab a T-cell receptor cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) antibody for the treatment of microsatellite-stable colorectal cancer is being developed by Agenus
• Vafidemstat a KDM1A inhibitor for the treatment of autism, borderline personality disorder, ADHD, Alzheimer’s disease and Multiple Sclerosis is being developed by Oryzon Genomics
• Vilobelimab (Gohibic, InflaRx) an anti-C5a monoclonal antibody for the treatment of hospitalized COVID-19 adults receiving invasive mechanical ventilation or ECMO recently received an EUA

The Prescribe Right Pharmaceutical Pipeline Tracker now allows you to track 1,135 pipeline and recently approved drugs supported by 3,064 announced and published study results.
 
Regulatory Update
 
The FDA approved omidubicel (Omisirge, Gamida Cell), on 4/17,2023, to reduce the time to neutrophil recovery and the incidence of infection. In patients with hematologic malignancies planned for umbilical cord blood transplantation following myeloablative conditioning. Omidubicel was approved with a boxed warning regarding infusion reactions, graft versus host disease, engraftment syndrome, and graft failure.
 
The FDA approved tofersen (QALSODY, Biogen), on 4/25/2023, for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.
 
The FDA approved a fecal microbiota product (Vowst, Seres Therapeutics), on 4/26/2023, for the prevention of recurrent C. difficile infection.
 
The FDA approved GSK’s respiratory syncytial virus vaccine (Arexvy), on 5/3/2023, to prevent lower respiratory tract infection caused by RSV in individuals 60 and older.
 
The FDA rejected concizumab and requested additional information related to monitoring, dosing and the manufacturing process.

The FDA granted a Fast Track designation to VE303 for the prevention of recurrent Clostridioides difficile infection.
 
The FDA’s Antimicrobial Drugs Advisory Committee voted 12-0 to recommend approval of the combination of sulbactam and durlobactam for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused bysusceptible strains of Acinetobacter. Sulbactam/durlobactam has a PDUFA date of May 29, 2023.
  
The FDA has placed a hold on Sun Pharma’s deuruxolitinib 12 mg due to a report of a pulmonary embolism in a long-term Open Label Extension (OLE) study. The gold does not affect the 8 mg dose, since no thrombotic events have been reported with the lower dose.

The European Commission granted marketing authorization for vadadustat (Vafseo, Akebia Therapeutics) for the treatment of anemia in chronic kidney disease (CKD) dialysis patients.

Roche obtained zinpentraxin in February 2020, when it bought Promedior. Roche discontinued development of zinpentraxin, in February 2023, as a treatment for idiopathic pulmonary fibrosis after a futility analysis failed to find a benefit compared to placebo. In April 2023, Roche also abandoned development of zinpentraxin to treat myelofibrosis as part of a pipeline review and reprioritization.