Update for April 5, 2022

Regulatory Update
 
The FDA rejected vadadustat due to cardiovascular, thromboembolic and hepatotoxicity safety concerns and recommended new trials to assess safety.
 
The FDA extended the PDUFA date for vutrisiran by three months to 7/14/2022, for the treatment of transthyretin-mediated (ATTR) amyloidosis, to allow time to review information on a new packaging and labeling facility.
 
The FDA extended the PDUFA date for ublituximab for the treatment of CLL and small lymphocytic lymphoma (SLL) by three months to allow additional time to review new data. The new PDUFA date is 6/25/2022.
 
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 to recommend the FDA reject AMX0035 or the treatment of ALS and recommended an additional trial to evaluate efficacy.
 
The FDA accepted the NDA for futibatinib for the treatment of locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions and set a PDUFA date for 9/30/2022.
 
ImmunoGen submitted a BLA for mirvetuximab soravtansine for the treatment of folate receptor alpha (FRα)-high platinum-resistant ovarian cancer that has been previously treated with one to three prior systemic treatments. 
 
The FDA placed a partial hold on leronlimab HIV trials and a full hold on leronlimab COVID-19 trials in the United States. At the same time, CytoDyn decided to place a hold on its leronlimab COVID-19 Brazilian trial pending review of two cardiac events by the data safety monitoring board.
 
Announced Research Updates
 
Boehringer Ingelheim announced 12-week data for the spesolimab Effisayil 1 trial.
 
Roche announced from the SKYSCRAPER-02 trial, evaluating the addition of tiragolumab to atezolizumab, carboplatin, and etoposide in patients with extensive-stage small cell lung cancer.
 
Cytokinetics announced results from cohort III of the REDWOOD-HCM trial, evaluating aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy.
 
BMS announced results for the VALOR-HCM trial evaluating the effect of mavacamten on the need for septal reduction therapy in patients with symptomatic obstructive hypertrophic cardiomyopathy. BMS also announced long-term-extension results from the EXPLORER-LTE trial, evaluating mavacamten in the treatment of patients with NYHA Class II or III obstructive hypertrophic cardiomyopathy.
 
Published Research Updates
 
H. pylori eradication with vonoprazan compared to lansoprazole results were published.
 
Harvard researchers discuss the estimated cost effective prices for aducanumab and donanemab in a new analysis.
 
A comparison of anetumab ravtansine to vinorelbine in the treatment of unresectable locally advanced or metastatic disease overexpressing mesothelin was published.
 
Results were published for the GOAL trial evaluating pixantrone plus obinutuzumab in the treatment of diffuse large B-cell lymphoma.
 
The efficacy of CTP-543 in the treatment of moderate to severe alopecia areata was published
 
Results were published for the ROCKIES trial, comparing oral roxadustat to parenteral epoetin alfa in patients with dialysis-dependent CKD.