FDA Approvals Roundup: Voxzogo, Fyarro, Livtencity

RoundupsRoundups | 24 November 2021 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA). 
New approvals 
Voxzogo cleared as growth-improvement therapy for children with dwarfism 
BioMarin’s Voxzogo (vosoritide injection) has been approved for improving growth in children aged 5 years or older with achondroplasia and open epiphyses, or growth plates. 
Achondroplasia is the most common form of dwarfism. Children with open epiphyses still have the potential to grow.  
Approval of Voxzogo, a C type natriuretic peptide analog, was based on findings from a year-long Phase 3, randomized, double-blind, placebo-controlled study in 121 children from the indicated population who were randomized to receive Voxzogo or placebo. The children’s annualized growth velocity, or rate of height growth, was measured at the end of 1 year. Those receiving Voxzogo grew an average 1.57 cm taller compared with those receiving placebo.  
The FDA approved Voxzogo under the accelerated approval pathway, under which a post-marketing study is required to assess final adult height. The application also received priority review designation. 
Fyarro nabs go-ahead for aggressive form of sarcoma 
Aadi’s Fyarro (sirolimus protein-bound particles for injectable suspension; albumin-bound) has been approved for treating adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor.  
The approval of Fyarro, an mTOR inhibitor, was based on findings from the Phase 2, interventional, single-group assignment AMPECT trial in 31 patients from the indicated population. Overall response rate was 39%, with 2 patients achieving a complete response after extended follow-up. The median duration of response has not been reached with a median follow-up of 36 months. In all, 92% of responders had a response lasting 6 months or longer; 67% had a response of 12 months or longer; and 58% had a response of 2 or more years. 
Livtencity approved for post-transplant cytomegalovirus infection  
Takeda’s Livtencity (maribavir) has been approved for treating adults and children aged 12 years or older with post-transplant cytomegalovirus (CMV) infection that is resistant to other treatments. 
The approval of the oral antiviral drug was based on findings from a Phase 3 multicenter, open-label, active-controlled trial in 352 transplant recipients from the indicated population who received Livtencity or a researcher-assigned treatment for 8 weeks. At the end of that period, 56% of the 235 patients receiving Livtencity had levels of CMV DNA levels below what was measurable, compare with 24% of the 117 patients receiving the investigator-assigned treatment. 
Livtencity received breakthrough therapy and priority review designations for this indication.  
New indications 
Keytruda use extended as adjuvant treatment for renal cell carcinoma 
Merck’s Keytruda (pembrolizumab injection) has been granted a new indication as an adjuvant treatment for patients with renal cell carcinoma (RCC) who are at risk of recurrence following nephrectomy or who have undergone nephrectomy and resection of metastatic lesions. 
Approval for this indication was based on findings from the multicenter, randomized, double-blind, placebo-controlled KEYNOTE-564 trial in 994 patients from the indicated population. The patients were randomized 1:1 to receive Keytruda or placebo for up to 1 year until disease recurrence or unacceptable toxicity. The primary outcome measure was disease-free survival (DFS), defined as time to recurrence, metastasis, or death. Overall survival (OS) was a secondary outcome measure. 
A significant improvement in DFS was demonstrated at a prespecified interim analysis, with 109 (22%) events in the Keytruda arm and 151 (30%) events in the placebo arm. Median DFS was not reached in either arm. At the time of the DFS analysis, OS data were not mature, with 5% deaths in the overall population. 
This review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada, and Swissmedic. 
This review used the assessment aid, and the application was granted priority review. 
Keytruda, a human programmed death receptor-1‒blocking antibody, was originally approved in 2014 for use in previously treated metastatic melanoma and is used in numerous other cancers, including non-small cell lung cancer and triple-negative breast cancer. 


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Tags: FDA, US

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