Update for September 15, 2022

Regulatory Update
 
The FDA approved terlipressin (Terlivaz, Mallinckrodt), on 9/14/2022, to improve kidney function in hepatorenal syndrome (HRS) with rapid reduction in kidney function. Terlipressin was approved with a black box warning regarding the potential for serious or fatal respiratory failure.
 
The FDA approved eflapegrastim (Rolvedon, Spectrum Pharmaceuticals), on 9/9/2022, to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia.
 
The FDA approved deucravacitinib (Sotyktu, Bristol Myers Squibb ), on 9/9/2022, for the treatment of moderate-to-severe plaque psoriasis in patients who are candidates for systemic therapy or phototherapy.
 
The FDA accepted the BLA for velmanase alfa, for the treatment of alpha-mannosidosis, suggesting a PDUFA date for 2/12/2023.
 
The FDA accepted the NDA for trofinetide for the treatment of Rett syndrome and set a PDUFA date for 3/12/2023.
 
Seres completed a rolling BLA submission for SER-109 for the prevention of recurrent C. difficile infection in September 2022.
 
Announced Research Updates
 
Hutchmed announced that in the 691 patient, Phase III, FRESCO-2 trial, treatment with fruquintinib resulted in 7.4 month overall survival compared to 4.8 months with placebo in patients with advanced, refractory metastatic colorectal cancer.
 
Almirall announced that in a one-year extension of the ADvocate 1 trial, 74% of patients who received lebrikizumab every four weeks and 76% of patients who received the drug every two weeks maintained an IGA score of clear or almost clear. EASI-75 was achieved by 79% in both the every four weeks group and every two weeks group. Almirall also announced that in a one-year extension of the ADvocate 2 trial, 81% of patients who received lebrikizumab every four weeks and 65% of patients who received the drug every two weeks maintained an IGA score of clear or almost clear. EASI-75 was achieved by 85% in the every four week group and 77% of patients in the every two weeks group.
 
Allakos announced that in the 24-week, 93 patient, Phase III, EoDyssey trial, treatment with lirentelimab reduced eosinophils more than placebo in patients with biopsy confirmed moderate to severe eosinophilic duodenitis. But lirentelimab did not improve a patient reported symptom score compared to placebo.
 
BeiGene announced that in the 674 patient, Phase III, RATIONALE 301 trial, treatment with tislelizumab resulted in overall survival of 15.9 months compared to 14.1 months with sorafenib in patients with unresectable hepatocellular carcinoma. The results were non-inferior, but did not achieve superiority.
 
SpringWorks announced that in the 142 patient, Phase III, DeFi trial, treatment with nirogacestat resulted in a 71% lower risk of disease progression compared to placebo in patients with progressive desmoid tumors.
 
Akero announced that in the 24-week, 100 patient, Phase IIb, HARMONY trial, treatment with efruxifermin achieved at least a one-stage improvement in liver fibrosis with no worsening of NASH in 39% who received 28 mg and 41% with 50 mg compared to 20% with placebo in patients with pre-cirrhotic NASH fibrosis stage 2 or 3.
 
Oramed announced that in a 12-week, 32 patient, Phase II trial, treatment with oral insulin reduced liver fat compared to placebo in patients with type 2 diabetes and non-alcoholic steatohepatitis (NASH).
 
Published Research Updates
 
In a final report, ICER found evidence to support efficacy for AMX0035 in the treatment of amyotrophic lateral sclerosis (ALS) to be comparable or better compared to standard of care alone. ICER felt the evidence suggested a survival benefit in ALS patient treated with AMX0035 with minimal adverse effects. However, ICER felt that a new trial would be needed to prove that AMX0035 increased survival or slowed disease progression.  If AMX0035 is priced similar to intravenous edaravone at $171,000 a year, the drug would exceed cost effectiveness thresholds. ICER calculated an annual health-benefit price benchmark (HBPB) range for AMX0035 of $9,100 – $30,700.