Update for March 8, 2022

Regulatory Update
 
The FDA approved ciltacabtagene autoleucel (Carvykti, Johnson & Johnson and Legend Biotech), on 2/28/2022, for the treatment of patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. 

• Ciltacabtagene autoleucel was approved with a black box warning for cytokine release syndrome, neurotoxic events, prolonged cytopenias , hemophagocytic lymphohistiocytosis/macrophage activation syndrome, Parkinsonism and Guillain-Barré syndrome. 
• J&J and Legend have set WAC at $465,000. Ciltacabtagene autoleucel is only available through a Risk Evaluation and Mitigation Strategy (REMS) called the CARVYKTI REMS.

The FDA approved pacritinib (Vonjo, CTI BioPharma), on 2/28/2022, to treat intermediate or high-risk primary or secondary myelofibrosis with severe thrombocytopenia (post-polycythemia vera or post-essential thrombocythemia).
 
The FDA rejected lenacapavir due to concerns of the incompatibility of vials made of borosilicate glass with lenacapavir solution, which could potentially lead to the formation of sub-visible glass particles. Gilead plans to resubmit the NDA with a different vial type.
 
The FDA designated Pfizer’s investigational respiratory syncytial virus (RSV) vaccine, PF-06482077, a Breakthrough Therapy for prevention of RSV-associated lower respiratory tract illness in infants from birth up to six months of age by active immunization of pregnant women.
 
Announced Research Updates
 
Vaccine efficacy for PF-06425090 Clostridium difficile vaccine to prevent Clostridioides difficile in adults 50 years of age and older.
 
AP-013 effect on the WOMAC pain and function scores in patients with severe osteoarthritis of the knee.
 
Safety of fezolinetant on endometrial health in patients with moderate to severe vasomotor symptoms associated with menopause.
 
Published Research Updates
 
Effect of roluperidone on the PANSS-derived Negative Symptoms Factor Score in patients with schizophrenia presenting with moderate to severe negative symptoms.
 
Nirsevimab efficacy in preventing RSV-associated lower respiratory tract infection and hosptializations in healthy late preterm and term infants of 35 weeks or greater gestational age
 
The results form adding toripalimab to paclitaxel plus cisplatin in Chinese patients with advanced esophageal squamous cell carcinoma.
 
The effect of deucravacitinib on achievement of ACR-20 in patients with psoriatic arthritis. 
 
Use of camrelizumab, famitinib and nab-paclitaxel in Chinese patients with refractory metastatic immunomodulatory triple-negative breast cancer.
 
The efficacy of gefapixant on the 24-hour coughs per hour average the Phase III COUGH-1 and COUGH-2 trials.