Update for August 25, 2021

Regulatory Update

The FDA approved difelikefalin (Korsuva, Cara Therapeutics,Vifor Pharma) on 8/23/2021, for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis.
 
The FDA approved tick-borne encephalitis vaccine (Ticovac, Pfizer), on 8/13/2021, for the prevention of tick-borne encephalitis in individuals one year of age and older. The vaccine has been available in Europe since 1976. Pfizer bought the vaccine from Baxter in 2014.
 
The FDA accepted the NDA for mitapivat for the treatment of pyruvate kinase (PK) deficiency and set a PDUFA for 2/17/2022
 
The FDA and EMA have accepted the BLA for tebentafusp for the treatment of HLA-A*02:01-positive adult patients with metastatic uveal melanoma (mUM). The FDA set a PDUFA date for 2/23/2022
 
The EMA approved roxadustat to treat anemia in both non-dialysis-dependent and dialysis-dependent chronic kidney disease patients.
 
Stealth BioTherapeutics submitted an NDA for elamipretide for the treatment of Barth syndrome.
 
Announced Research Updates
 
Coherus BioSciences and Junshi Biosciences announced interim results from 218 patients enrolled in the 465 patient, Phase III, CHOICE-01 trial, where adding toripalimab to chemotherapy resulted in PFS of 8.3 months compared to 5.6 months with chemotherapy alone in patients with advanced squamous or non-squamous non-small cell lung cancer. 
 
Published Research Updates
 
ICER estimates an annual cost for mavacamten of $12,513 for a threshold of $100,000 per QALY and $15,451 for a threshold of $150,000 per QALY. In beginning their calculations, ICER used a placeholder price of $75,000 per year based on an analyst’s projection for mavacamten. ICER estimated the annual cost for competing therapies as $8,559 for disopyramide plus dose initiation hospitalization, $122,750 for myectomy and $55,706 for septal ablation. The cost for metoprolol and verapamil were < $70 per year.
 
In a 38 patient, Phase II, open-label trial, treatment with camrelizumab plus famitinib resulted in an overall response rate of 60.5% in Chinese patients with advanced or metastatic renal cell carcinoma.
 
In the 12-week, 285 patient, Phase III, JADE TEEN trial, the IGA endpoint was achieved by 46.2% of patients treated with abrocitinib 200 mg and 41.6% with 100 mg compared to 24.5% with placebo in patients 12 to 17 years of age with moderate to severe AD who also received topical therapy. EASI-75 was achieved by 72% of patients who received abrocitinib 200 mg and 68.5% with 100 mg compared to 41.5% with placebo. 
 
In a 12-week, 79 patient, Phase IIa Trial, 57.3% of patients treated with bimekizumab had a 50% or greater decrease in their Hidradenitis Suppurativa Clinical Response (HiSCR50) score compared to 26.1% with placebo in patients with hidradenitis suppurativa. HiSCR75 was achieved by 46% of bimekizumab patients and 32% achieved HiSCR90 compared to 35% and 15% with adalimumab and 10% and 0 with placebo.
 
In the 14.9 month, 233 patient, Phase III, open-label, ASCEMBL trial, 25.5% of patients treated with asciminib achieved a major molecular response (MMR) rate compared to 13.2% with bosutinib in patients with chronic myeloid leukemia in chronic phase who were resistant or intolerant to two or more tyrosine kinase inhibitors.
 
In a long-term follow-up, of at least 35-months, of patients enrolled in the L-MIND trial, treatment with tafasitamab plus lenalidomide resulted in an overall response rate of 57.5%, which included a complete response in 40% of patients. Overall survival was 33.5 months and progression-free survival was 11.6 months.
 
In a 12-month, 42 patient, Phase II/III trial, patients treated with arimoclomol slowed disease progression with a 0.76 point increase in the NPC Clinical Severity Scale (NPCCSS) compared to 2.15 increase with placebo in patients, age 2-18 years with Niemann-Pick Type C disease (NPC). In patients receiving miglustat, the addition of arimoclomol reduced the increase in NPCCSS by 2.06 points.
 
In a 29-day, 21 patient, open-label, dose-escalation, Phase II trial, treatment with a single dose of cipargamin 150 mg resulted in PCR-corrected and uncorrected adequate clinical and parasitological response (ACPR) of 75% at 14-days and 65% at 28-days, which was less than artemether-lumefantrine (> 90% at both time periods) in sub-Saharan African patients with malaria.
 
In the 90 patient, Phase III, MAPP1 trial, patients treated with MDMA-assisted psychotherapy had a 24.4 point decrease in their Clinician-Administered PTSD Scale (CAPS-5) compared to a 13.9 point decrease with psychotherapy alone in patients with severe PTSD.
 
In an analysis of 568 patients from the SAKURA trials, four-weeks after the first injection of daxibotulinumtoxinA, 57.9% as judged by patients and 64,8% as judged by investigors, had no static Glabellar lines (GL). After a second injection 68.1% and 75% reported no GL and 71.5% and 77.6% after the third treatment in patients with mild and moderate static Glabellar lines. 
 
In the 482 patient, Phase III SEAMLESS trial, treatment with sapacitabine plus decitabine did not improve overall survival compared to decitabine alone in elderly patients with newly diagnosed acute myeloid leukemia.