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Voxelotor Granted Priority Review for Children With Sickle Cell Disease

The U . S . Food and Drug Administration ( FDA ) has accepted Global Blood Therapeutics ’ ( GBT ’ s ) supplemental New Drug Application ( sNDA ) seeking accelerated approval for voxelotor for the treatment of children ages 4 to 11 with sickle cell disease ( SCD ). Voxelotor is a once-daily oral therapy that modulates hemoglobin ( Hb ) affinity for oxygen by binding to and stabilizing Hb S .
Additionally , the FDA has accepted GBT ’ s New Drug Application ( NDA ) for a new dispersible tablet dosage form of voxelotor appropriate for pediatric patients . The 300 mg tablets are grape flavored , intended to be dispersed in roomtemperature clear drinks for ease of swallowing , and allow for weight-based dosing .
Previously , the drug was FDA approved for the treatment of SCD in patients ages 12 and older . Voxelotor inhibits Hb polymerization , reduces sickling , and improves red blood cell survival .
GBT ’ s pediatric sNDA and NDA are based on data from the phase IIa HOPE-KIDS 1 Study ( GBT440-007 ), which demonstrated improved Hb and reduced hemolysis in patients who received weight-based treatment with voxelotor dispersible tablets .
The agency has granted priority review status to both applications , with a Prescription Drug User Fee Act target action date of December 25 , 2021 .
Source : Global Blood Therapeutics press release , September 7 , 2021 .

FDA Grants Accelerated Approval to Zanubrutinib for Marginal Zone Lymphoma

Zanubrutinib has received accelerated approval from the FDA for the treatment of adult patients with relapsed or refractory marginal zone lymphoma ( MZL ) previously treated with at least one anti-CD20-based regimen .
Accelerated approval is based on efficacy data from the phase II MAGNOLIA trial and the phase I / II BGB-3111-AU-003 trial , with overall response rate ( ORR ) as assessed by independent review committee per 2014 Lugano Classification as the primary endpoint . Additional endpoints were complete response ( CR ) rate and duration of response ( DOR ).
In the phase II MAGNOLIA trial , 66 evaluable patients had the following subtypes of relapsed / refractory MZL :
• extranodal ( n = 26 )
• nodal ( n = 26 )
• splenic ( n = 12 )
• unknown ( n = 4 )
The ORR , based on CT scan assessment , was 56 % ( 95 % CI 43-68 %). The CR rate using CT scan was 20 %. Based on assessment using PET-CT scan , the ORR was 67 % ( 95 % CI 54-78 %) and the CR rate was 26 %. At a median follow-up of 8.3 months , median DOR was not reached . At 12 months , 85 % of responders were still in remission ( 95 % CI 67-93 %). Responses were observed across all subtypes .
A total of 20 patients were evaluated in the BGB-3111-AU-003 trial , including the following subtypes :
• extranodal ( n = 9 )
• nodal ( n = 5 )
• splenic ( n = 6 )
The ORR and CR rates based on CT scan assessment were 80 % ( 95 % CI 56-94 %) and 20 %, respectively . The median DOR was not reached at a median follow-up of 31.4 months . At 12 months , 72 % of responders were still in remission ( 95 % CI 40-88 %).
Common adverse events ( AEs ) occurring in ≥30 % of the 847 patients in the pooled safety population were decreased neutrophil count , upper respiratory tract infection , decreased platelet count , hemorrhage , decreased lymphocyte count , rash , and musculoskeletal pain .
Recommended dosing for this indication is either 160 mg twice daily or 320 mg once daily orally with or without food . The dose may be reduced for patients with severe hepatic impairment and certain drug interactions .
Source : BeiGene press release , September 15 , 2021 .

Ruxolitinib Approved for Chronic Graft- Versus-Host Disease

The FDA has approved ruxolitinib for the treatment of chronic graft-versus-host disease ( cGVHD ) in adult and pediatric patients 12 years and older whose disease is refractory to one or two lines of systemic therapy .
This decision is supported by efficacy data from the REACH-3 trial comparing 10 mg daily ruxolitinib to best available therapy ( BAT ) for 329 patients whose cGVHD was refractory to corticosteroids after allogeneic stem cell transplantation . Overall response rate ( ORR ), as defined by 2014 NIH Response Criteria , was 70 % ( 95 % CI 63-77 %) for patients treated with ruxolitinib , compared with 57 % ( 95 % CI 49-65 %) in the BAT arm . Median duration of response from first response to progression , death , or new systemic therapy for cGVHD was 4.2 months ( 95 % CI 3.2- 6.7 ) in the ruxolitinib group and 2.1 months ( 95 % CI 1.6-3.2 ) for patients receiving BAT . The median time from first response to death or new systemic therapy in the ruxolitinib and BAT arms was 25 months ( 95 % CI 16.8 to not estimable ) and 5.6 months ( 95 % CI 4.1-7.8 ), respectively .
Anemia and thrombocytopenia occurred in > 35 % of patients receiving ruxolitinib . The most common nonhematologic AEs , occurring in ≥20 % of patients , were infections .
Recommended starting dose of ruxolitinib for the treatment of cGVHD is 10 mg orally twice daily .
Source : FDA press release , September 22 , 2021 .

After a Pandemic- Driven Boom , Telehealth Visits Level Off

According to recent data , telehealth visits are leveling off from their pandemic highs to below 20 % of total medical appointments .
Overall telehealth use for doctor ’ s office visits and outpatient health care was 78 times higher in April 2020 than in February 2020 , consulting firm McKinsey reported in July . The analysis also found that telehealth use has since stabilized at levels 38 times higher than pre-pandemic , ranging from 13 % to 17 % of visits across all specialties .
A recent survey by KLAS Research and the Center for Connected Medicine ( CCM ) found that about 40 % of hospital executives reported conducting up to 10 % of appointments virtually , while a similar percentage reported that between 11 % and 20 % of their appointments are virtual . The 12 % of survey respondents who said they are currently using telehealth for more than 30 % of visits feel that this number is still inflated because of the COVID-19 pandemic and expect it to decline in the future .
FAIR Health , a nonprofit organization that provides information on health care coverage and insurance , reported that national telehealth utilization declined 10 % in June .
In all regions of the U . S ., most telehealth diagnoses have been mental health conditions , according to FAIR Health . The KLAS Research and CCM survey found that hospitals are using telehealth most frequently for primary care and behavioral health , while two areas likely to see telehealth expansion include chronic care management and urgent care .
Source : Fierce Healthcare , September 9 , 2021 .
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