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Regulatory Focus™ > News Articles > 2021 > 4 > FDA Approvals Roundup: Qelbree, Sarclisa, Tyvaso

FDA Approvals Roundup: Qelbree, Sarclisa, Tyvaso

Posted 07 April 2021 | By Renee Matthews 

FDA Approvals Roundup: Qelbree, Sarclisa, Tyvaso

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Qelbree okayed as nonstimulant therapy option for children with ADHD
Supernus’s Qelbree (viloxazine extended-release capsules) has been approved for the treatment of  attention deficit hyperactivity disorder (ADHD) in children and adolescents aged 6 to 17 years.
The serotonin norepinephrine modulating agent provides a nonstimulant alternative to other ADHD treatment options that are considered controlled substances. However, Qelbree carries a black box warning for increased risk for suicidal ideation during early treatment or dosing changes.
Qelbree’s approval was based on findings from three phase 3, multicenter, randomized, double-blind, three-arm, placebo-controlled studies in a total of 1,100 patients aged between 6 and 17 years (studies 1 and 2, in children aged 6 to 11 years; and Study 3, in those aged 12 to 17 years). Patients received 100 mg, 200 mg, or 400 mg Qelbree, or placebo. The primary endpoint was the reduction from baseline to study end in ADHD Rating Scale score, an assessment of hyperactivity, impulsivity, and inattention. In all three studies, patients treated with any of the doses of Qelbree had a significantly greater reduction in the rating score compared with those receiving placebo. Total duration of treatment was 6 weeks for studies 1 and 3, and 8 weeks for Study 2.
Supernus resubmitted a new drug application in February 2021 after receiving a complete response letter (CRL) from the FDA in November 2020. The agency said in the CRL that the review process for the application was complete, but the application was not ready for approval because of agency concerns around relocation of the company’s in-house laboratory. The two parties had a Type A meeting in January to discuss the letter and NDA resubmission requirements, and the company removed reference to the in-house laboratory and addressed other issues in the CRL before resubmitting the NDA. The agency reclassified the NDA review to accelerate the review.
New indications
Tyvaso indication extended to ILD-related pulmonary hypertension
United Therapeutics’ Tyvaso (treprostinil inhalation solution) has been granted an expanded indication for treating patients with pulmonary hypertension associated with interstitial lung disease to improve exercise ability.

Approval of the supplemental new drug application was supported by findings from the multicenter, randomized, double-blind, placebo-controlled INCREASE study in 326 patients in the indicated population. The primary endpoint was improvement in six-minute walk distance. Participants were randomized 1:1 to receive Tyvaso or placebo. After 16 weeks, the least squares mean difference between the two groups in change from baseline in the six-minute walk distance was 31.12 meters (95% CI, 16.85-45.39; P < .001). Tyvaso patients showed a 15% reduction in levels of N-terminal pro-B-type natriuretic peptide, a marker for cardiac function, compared with an increase of 46% in the placebo group (treatment ratio, 0.58; 95% CI, 0.47-0.72; P < .001). In addition, 37 patients (22.7%) in the Tyvaso group experienced clinical worsening, compared with 54 (33.1%) in the placebo group (HR, 0.61; 95% CI, 0.40- to 0.92; P = .04).
Tyvaso was approved in 2009 for treating adults with pulmonary arterial hypertension.
Sarclisa combo approved for relapsed/refractory multiple myeloma
Sanofi’s Sarclisa (isatuximab-irfc IV infusion), in combination with standard-of-care (SoC) carfilzomib and dexamethasone, has received an expanded indication for treating relapsed or refractory multiple myeloma in previously treated adults.
Approval of the monoclonal antibody was based on efficacy and safety findings in the phase 3, multinational, randomized, open-label, two-arm, IKEMA study in 302 patients in the indicated population. Participants were randomized 3:2 to receive the Sarclisa combination therapy or SoC until disease progression or unacceptable adverse events. The main efficacy outcome measure was progression-free survival (PFS). Median PFS was not reached for the Sarclisa combination and was 20.27 months for SoC (hazard ratio, 0.548; 95% CI, 0.366-0.822; P = .0032), a 45% reduction in the risk of disease progression or death in the Sarclisa combination group over SoC alone.
The application review used the assessment aid. Sarclisa has orphan drug designation for the treatment of multiple myeloma.
Sarclisa was first approved in 2020 and is used in combination with pomalidomide and dexamethasone to treat relapsed/refractory multiple myeloma in previously treated adults.
Praluent approved as add-on for genetic form of severe high cholesterol
Regeneron’s Praluent (alirocumab injection) has received an expanded indication as an adjunct therapy for adults with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening genetic condition causing severely high cholesterol.
Patients with HoFH have dangerously high circulating levels of low-density lipoprotein cholesterol (LDL-C), also known as “bad cholesterol.” They can develop premature cardiovascular disease as teenagers or young adults. Many do not respond to other cholesterol-lowering drugs and are at risk of dying before age 30.
Praluent’s approval was based on findings in 12-week, double-blind, randomized ODYSSEY HoFH evaluating the drug’s effectiveness and safety in 69 patients in the indicated population. In all, 45 patients received Praluent and 24 received placebo in addition to their regular LDL-C‒lowering medications. At week 12 of the study, patients in the Praluent group had an average 27% decrease in LDL-C, compared with an average 9% increase in the placebo group.
Praluent was first approved in 2015 for treating adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease.
Erbitux gets news dosage regimen for colorectal, head and neck cancers
ImClone’s Erbitux (cetuximab) has received approval for a new dosage regimen of 500 mg/m2 as a 120-minute intravenous infusion every 2 weeks for patients with K-Ras wild-type, EGFR-expressing colorectal cancer or squamous cell carcinoma of the head and neck.
Erbitux was first approved in 2004 and is used for treating advanced colorectal and head and neck cancers.



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Tags: FDA, US

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