The RAPS store will be under maintenance Saturday, 17 April between 5 AM and 12 PM EST. Store functionality may be unavailable at times during this window.
We apologize for any inconvenience caused during this time.

Regulatory Focus™ > News Articles > 2021 > 3 > FDA Approvals Roundup: Abecma, Vyxeos, Myrbetriq

FDA Approvals Roundup: Abecma, Vyxeos, Myrbetriq

Posted 31 March 2021 | By Renee Matthews 

FDA Approvals Roundup: Abecma, Vyxeos, Myrbetriq

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
 
New approvals
Abecma cleared as first cell-based gene therapy for multiple myeloma in adults
Bristol Myers Squibb’s Abecma (idecabtagene vicleucel) has been approved for treating relapsed or refractory multiple myeloma in previously treated adults, making it the first FDA-sanctioned cell-based gene therapy for this condition.
 
Abecma is a B-cell maturation antigen‒directed, genetically modified, autologous chimeric antigen receptor (CAR) T-cell therapy. In these immunotherapies, the patient’s T cells are collected and “customized” through genetic modification, then re-infused into the patient to counter the disease.
 
Approval of Abecma was based on efficacy and safety findings in the phase 2, single-group, multicenter KarMMa study of 127 patients in the indicated population. In 100 patients evaluated for efficacy, overall response rate was 72% (95% confidence interval, 62% to 81%) and complete response was 28% (95% CI, 19% to 38%). About 65% of those attaining complete response had a remission of at least 12 months.
 
The therapy was approved with a risk evaluation and mitigation strategy. The agency has stipulated the manufacturer must conduct a post-marketing, observational study in patients treated with Abecma to evaluate its long-term safety.
 
This application was granted breakthrough therapy and orphan drug designations.
 
New indications
Vyxeos gets expanded indication for treating secondary AML in children
Jazz’s Vyxeos (daunorubicin and cytarabine) has been granted an expanded indication for treating newly diagnosed, therapy-related acute myeloid leukemia or AML with myelodysplasia-related changes in children aged 1 year or older.
 
The combination drug’s approval for the expanded indication was based on safety and pharmacokinetic findings in two single-arm trials in young patients in the indicated population: the phase 1/2 AAML1421 study in 38 patients aged between 1 and 21 years; and the phase 1 CPX-MA-1201 study in 27 patients aged between 1 and 19 years. Findings from both studies showed no differences in the safety profile based on age. Efficacy findings for Vyxeos from the CPX351-301 study in adults supported use of the drug in this younger population.
 
Vyxeos was first approved in 2017 for the same AML indication in adults.
 
Myrbetriq handed new indication for neurogenic detrusor overactivity in children
Astellas’ Myrbetriq (mirabegron extended-release tablets) and Myrbetriq Granules (mirabegron for extended-release oral suspension) have been granted a new indication for treating neurogenic detrusor overactivity (NDO) in children aged 3 years or older.
 
Myrbetriq is also indicated, in extended-release tablet form, for adults with NDO, a bladder dysfunction related to neurologic impairment.
 
Approval for the pediatric indication was based on efficacy findings in the year-long, open-label, baseline-controlled, phase 3 Crocodile study with 86 patients aged between 3 and 17 years. After 24 weeks of treatment, there were improvements from baseline in bladder capacity (3 to <12 years: mean change of 72 mL; 12-17 years: 113 mL) and number of daily leakage episodes (-2.0 and -1.0 episodes, respectively). Improvements on baselines measures over the same period were also noted for number of detrusor contractions and volume of urine retained until first contraction.
 

 

© 2021 Regulatory Affairs Professionals Society.

Tags: FDA, US

Regulatory Focus newsletters

All the biggest regulatory news and happenings.

Subscribe