FDA Approvals Roundup: Azstarys, Lorbrena, Yescarta
A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).New approvals
Azstarys approved as once-daily treatment for ADHD in children
Corium’s Azstarys (serdexmethylphenidate [SDX] and dexmethylphenidate [d-MPH] capsule) has been approved in three dosage strengths for treating attention deficit hyperactivity disorder (ADHD) symptoms in patients aged 6 years or older.
The approval was supported by findings from a Phase 3 multicenter, double-blind, randomized, placebo-controlled, laboratory classroom study in 150 children aged 6 to 12 years diagnosed with ADHD. After a week of treatment, raters used the SKAMP-Combined rating scale to evaluate the children’s attention and behavior in a classroom setting. Primary efficacy endpoint was mean change in scores from baseline. Children treated with Azstarys had an average SKAMP-C score reduction of -5.4 points, compared with those receiving placebo.
Because Azstarys’ d-MPH moiety is classified by the Drug Enforcement Administration (DEA) as a Schedule II stimulant, Azstarys also carries a Schedule II controlled substance classification pending DEA’s final scheduling decision. Other methylphenidate- and dexmethylphenidate-containing medicines also carry Schedule II classifications. Serdexmethylphenidate is a pro-drug of d-MPH.
New indications
Lorbrena gets expanded indication as first-line therapy for ALK-positive NSCLC
Pfizer’s Lorbrena (lorlatinib) has received an expanded indication as a first-line therapy for patients with
anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test.
The agency concurrently approved the Ventana ALK (D5F3) CDx Assay as a companion diagnostic for Lorbrena.
This review was conducted in collaboration with the FDA’s Project Orbis partners, the Australia’s Therapeutic Goods Administration, Brazil’s Health Regulatory Agency, Health Canada, and the UK Medicines and Healthcare Products Regulatory Agency. It used the real-time oncology review pilot program and the Assessment Aid. In addition, the application was granted priority review and orphan drug designation.
Lorbrena was first approved in 2018 to treat second- or third-line ALK-positive metastatic NSCLC.
Yescarta wins extended indication previously treated follicular lymphoma
Kite Pharma’s Yescarta (axicabtagene ciloleucel) has received an accelerated approval for treating adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.
Approval in follicular lymphoma was based on the single-arm, open-label, multicenter ZUMA-5 trial. Findings showed that among 81 patients, overall response rate was 91% in patients receiving Yescarta, and complete remission was 60%. Median duration of response was not reached, but the 1-year rate of continued remission was 76.2%.
This indication was granted accelerated approval based on response rate, and continued approval for the new indication will depend on demonstration of clinical benefit in follow-up findings. The application was granted priority review and breakthrough and orphan drug designations.
Yescarta was first approved in 2017 and is also used to treat adult patients with certain types of large B-cell lymphoma.