Update for January 6, 2023

Regulatory Update
 
The FDA approved lenacapavir (Sunlenca, Gilead Sciences), on 12/22/2022, for the treatment of HIV-1 infection in heavily treatment-experienced adults with multi-drug resistant HIV-1 infection. Lenacapavir is initiated as a combination of oral tablets with the first subcutaneous injection. After the loading dose, a subcutaneous injection of lenacapavir is given every six months. According to Endpoints News WAC for the first year of lenacapavir will be $42,250 with subsequent years costing $39,000.
 
The FDA approved mosunetuzumab (Lunsumio, Genentech, Roche), on 12/22/2022, for the treatment of relapsed or refractory follicular lymphoma in patients who have received at least two previous systemic therapies.
 
The FDA approved ublituximab (Briumvi, TG Therapeutics), on 12/28/2022, for the treatment of relapsing forms of multiple sclerosis in adults.
 
The FDA delayed a decision on toripalimab, until a site inspection of the manufacturing facility can be completed. Due to COVID-19 related restrictions on travel in China, an inspection had not been competed in December 2022.
 
The FDA rejected palovarotene and requested additional details on clinical trials in December 2022.
 
The FDA accepted the resubmitted BLA for bimekizumab for the treatment of moderate to severe plaque psoriasis with approval expected in 2Q23.
 
The FDA has placed a hold on the Phase III VITESSE trial, evaluating Viaskin Peanut, until DBV makes changes to the study protocol regarding statistical analysis and total number of patients. DBV modified the VITESSE protocol, and the FDA lifted the hold in December 2022.
 
SpringWorks submitted an NDA for nirogacestat for the treatment of adults with desmoid tumors.
 
Published Research Updates
 
In a review of fezolinetant, ICER found evidence to support a health benefit for fezolinetant in the treatment of vasomotor symptoms to be insufficient compared to menopausal hormone therapy. Longer-term safety and efficacy data are needed to determine the place in therapy for fezolinetant. ICER estimated a Health Benefit Price Benchmark for fezolinetant to be $2,000 to $2,500 per year.
 
ICER found evidence to support use of lecanemab to slow the development of mild cognitive impairment in early Alzheimer's disease to be promising but inconclusive with the possibility for harm due to ARIA. ICER estimated a range to meet cost-effectiveness criteria to be $8,500 to $20,600 per year. 
 
ICER found evidence to support use of donanemab to slow the development of mild cognitive impairment in early Alzheimer's disease to be insufficient, with only data from a Phase II trial available. There is also the with the possibility for harm due to ARIA. ICER assumed the drug would be as effective as lecanemab to estimate a range to meet cost-effectiveness criteria to be $14,500 and $46,900 per year. 
 
An ICER review concluded there was a moderate certainty of a comparable, small, or substantial health benefit with etranacogene dezaparvovec compared to factor IX therapy for the treatment of severe hemophilia B. While etranacogene dezaparvovec offers a clinical benefit, ICER was concerned about the duration of the benefit with long-term data only available through 18 months for 54 patients. There was also concern about potential long-term harms to liver function and hepatocellular carcinoma. ICER estimated a Health Benefit Price Benchmark (HBPB) of $2.93 million to $2.96 million for a single dose of etranacogene dezaparvovec.
 
An ICER review concluded there was a moderate certainty of a comparable, small, or substantial health benefit with valoctocogene roxaparvovec compared to factor VIII prophylaxis for the treatment of severe hemophilia A without inhibitors. When comparing valoctocogene roxaparvovec to emicizumab, ICER found limitations due to the small amount of data available to compare the drugs. They concluded there was low certainty for a net health benefit with valoctocogene roxaparvovec compared to emicizumab. While valoctocogene roxaparvovec offers a clinical benefit, ICER was concerned about the duration of the benefit and potential long-term harms to liver function and hepatocellular carcinoma. ICER estimated a Health Benefit Price Benchmark (HBPB) of $1.96 million for a single dose of valoctocogene roxaparvovec.