Update for August 17, 2021

Regulatory Update
 
The FDA approved belzutifan (Welireg, Merck), on 8/13/2021, to treat von Hippel-Lindau (VHL) disease for patients who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery.
 
The FDA agreed with the Advisory Committee recommendation and rejected AstraZeneca’s and FibroGen’s roxadustat for the treatment of anemia in patients with chronic kidney disease and requested a new efficacy trial.
 
The FDA rejected Sesen Bio’s oportuzumab monatox, for the treatment of BCG-unresponsive non-muscle invasive bladder cancer, and requested additional clinical and manufacturing data. 
 
In August 2021, the FDA delayed the PDUFA for Merck’s gefapixant, from December 2021 to March 2022, to allow more time for a full review of the drug. 
 
Ipsen withdrew the NDA for palovarotene to allow time for additional analyses and evaluation of data.
 
The FDA has placed a hold for a clinical trial evaluating Aprea Therapeutics’ eprenetapopt with acalabrutinib or with venetoclax and rituximab for the treatment of lymphoid malignancies. Three trials evaluating eprenetapopt in combination with azacitidine for the treatment of myelodysplastic syndrome has also been put on hold.
 
Announced Research Updates
 
The Department of Veterans Affairs will not include aducanumab on its national formulary due to safety and efficacy concerns. Use of the drug is restricted to specific patients when prescribed by dementia experts. Biogen is restricted from promoting the drug to VA prescribers.
 
Lilly announced 16-week results from the 52-week, Phase III, ADvocate 1 and ADvocate 2 trials, where more patients treated with lebrikizumab achieved an Investigator Global Assessment (IGA) score of clear or almost clear skin with a 2-point or greater improvement in the IGA score and a 75% or greater improvement in their EASI score compared to placebo in patients with moderate-to-severe atopic dermatitis.
 
Travere announced interim results after 36-weeks from the first 280 patients enrolled in the Phase III, PROTECT trial, where treatment with sparsentan reduced the urine protein-to-creatinine ratio by 49.8% compared to a 15.1% decrease with irbesartan in IgA nephropathy (IgAN) patients with persistent proteinuria despite active ACE or ARB treatment. Travere plans to file an NDA and MAA in the first half of 2022.
 
Published Research Updates
 
In the 52-week, 1,444 patient, Phase III SURPASS-3 trial, treatment with tirzepatide reduced HbA1c by 1.93% with 5 mg, 2.2% with 10 mg and 2.37% with 15 mg compared to 1.34% with insulin degludec in insulin-naive patients with type 2 diabetes and inadequate glycemic control on stable doses of metformin with or without an SGLT-2 inhibitor.The reduction in HbA1c met the criteria for non-inferiority. Tirzepatide also decreased bodyweight from 7.5 kg to 12.9 kg compared to gain of 2.3 Kg with insulin degludec. 
 
In the three-month, 2,199 patient, Phase III, CONCERTO trial, treatment with laquinimod did not improve the time to confirmed disease progression compared to placebo in patients with relapsing-remitting multiple sclerosis.
 
In an 8-week, 25 patient, open-label, phase II trial, treatment with remetinostat 1% gel resulted in at least a 30% decrease in tumor diameter in 70% of tumors and complete resolution in 54.8% of tumors in patients with basal cell carcinoma.