The FDA approved vosoritide (Voxzogo, BioMarin) on 11/19/2021, to treat achondroplasia with open epiphyses in children five years of age and older.
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The FDA granted a Fast Track Designation to omaveloxolone for the treatment of Friedreich’s ataxia.
Announced Research Updates
Mesoblast announced that in the 30-month, 565 patient, Phase III, DREAM-HF trial, treatment with a single dose of rexlemestrocel-L added to standard of care did not reduce the cumulative rate of recurrent non-fatal decompensation heart failure events compared to standard of care alone in patients with chronic heart failure with reduced ejection fraction.
PhaseBio announced interim results from 150 patients enrolled in the Phase III REVERSE-IT trial, where treatment with bentracimab reduced PRU by 135% within four hours and 98.4% of patients achieved effective hemostasis within 24 hours in ticagrelor patients requiring urgent surgery or an invasive procedure or experiencing uncontrolled major or life-threatening bleeding.
UCB announced interim data after from the 16-week, 852 patient, Phase III, BE OPTIMAL trial, where more paints treated with bimekizumab achieved ACR50 compared to placebo in patients with psoriatic arthritis, who have not been treated with a biologic drug.
Daiichi Sankyo announced that in the 539 patient, Phase III, QuANTUM-First trial, adding quizartinib to standard induction and consolidation chemotherapy, then continuing quizartinib alone as maintenance therapy improved overall survival compared to standard treatment alone plus placebo maintenance therapy in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML)
Published Research Updates
ICER found the evidence of a health benefit for adding mavacamten to optimal treatment of heart failure promising but inconclusive compared to optimal therapy alone or adding the drug to disopyramide. Some concern was expressed for the long-term effects of reduction in left ventricular ejection fraction and myocardial thickness. Some analysts have forecast an annual cost for mavacamten of $75,000, but ICER estimated a health-benefit price benchmark (HBPB) of $12,000-$15,000. Myectomy and septal ablation were found to be more cost effective than mavacamten.
In the 14-day, 1,242 patient, Phase II, AXIOMATIC-TKR trial, venous thromboembolism developed in 21% of patients treated with milvexian 25 mg BID, 11% with 50 mg BID, 9% with 100 mg BID, 8% with 200 mg BID, 25% with 25 mg QD, 24% with 50 mg QD and 7% with 200 mg QD compared to 21% who received enoxaparin QD in patients undergoing knee arthroplasty.
In a three-year, 18 patient, Phase I/II trial, treatment with SPK-8011 resulted in continued factor VIII expression in 16 of 18 participants and the annualized bleeding rate decreased from 8.5 events per year to 0.3 events per year, a 91.5% decrease, in patients with Hemophilia A.
Published in the same issue of Lancet Gastroenterology last week:
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