Update for January 31, 2023

Regulatory Update
 
The FDA approved elacestrant (Orserdu, Stemline Therapeutics), on 1/27/2023, for the treatment of estrogen receptor–positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.
 
The FDA approved pirtobrutinib (Jaypirca, Lilly), on 1/27/2023, for the treatment of relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton's tyrosine kinase inhibitor. 
 
The FDA’s Antimicrobial Drugs Advisory Committee voted 14 to 1 to recommend approval of rezafungin for the treatment of candidemia and invasive candidiasis.
 
The FDA accepted the NDA for gepirone or the treatment of major depressive disorder and set a PDUFA date of 6/23/2023.
 
The FDA granted Moderna’s mRNA vaccine, mRNA-1345, a Breakthrough Therapy Designation for the prevention of RSV-associated lower respiratory tract disease in adults aged 60 years or older.
 
The EMA validated the MAA for exagamglogene autotemcel, initiating a full review by CHMP.
 
CHMP recommended that palovarotene to treat fibrodysplasia ossificans progressiva not be approved by the EMA.
 
Announced Research Updates
 
Deciphera announced that in a 453 patient, open-label, Phase III, INTRIGUE trial, treatment with ripretinib did not improve PFS compared to sunitinib in patients with GIST previously treated with imatinib.
 
Peregrine announced that in a 28 patient, Phase II trial, treatment with bavituximab plus pembrolizumab resulted in an ORR of 32.1% in patients with previously untreated advanced hepatocellular carcinoma.
 
Published Research Updates
 
In the 52-week, 159 patient, Phase III, XTEND-1 trial, once weekly treatment with efanesoctocog alfa resulted in an annualized bleeding rate of 0.71 in 133 patients with severe hemophilia A. 26 patents in XTEND-1 received on-demand efanesoctocog alfa and 97% of bleeding episodes resolved after one injection of efanesoctocog alfa.
 
In the 12-week, 140 patient, Phase IIb, ADVISE trial, treatment with etrasimod did not improve the Eczema Area and Severity Index (EASI) compared to placebo in patients with moderate-to-severe atopic dermatitis. 
 
In the 24-week, 195 patient, Phase III, MOMENTUM trial, 25% of patients treated with momelotinib achieved a reduction of at least 50% in their Myelofibrosis Symptom Assessment Form (MFSAF) TSS compared to 9% with danazol in patients with myelofibrosis who were symptomatic and anemic and had been previously treated with an FDA-approved JAK inhibitor.