FDA Approvals Roundup: Evrysdi, Kymriah, Tibsovo

RoundupsRoundups | 01 June 2022 |  By 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New indications
Evrysdi gets label extension for use in babies with spinal muscular atrophy
Genentech’s Evrysdi (risdiplam; powder for oral solution) has been granted a new indication to include babies younger than 2 months with spinal muscular atrophy (SMA).
The approval is based on interim findings from the RAINBOWFISH study in newborns demonstrating that the most of the pre-symptomatic babies treated with Evrysdi were able to sit and stand, with half walking after 12 months of treatment.
Evrysdi was originally approved in 2020 for treating SMA in patients aged 2 months or older.
Kymriah okayed for relapsed/refractory follicular lymphoma
Novartis’s Kymriah (tisagenlecleucel; suspension for infusion) has been granted a new indication for treating relapsed/refractory follicular lymphoma adults who have received two or more lines of systemic therapy.
The approval of Kymriah, a CAR-T cell therapy, was based on findings from the multicenter, single-arm, open-label on the ELARA trial in 90 patients from the indication population. Overall response rate was 86% (complete response, 68%). Median duration of response was not reached, with 75% of responders still in response at 9 months.
This indication was approved under accelerated approval based on patient response rate. Continued approval for the indication is contingent on verification and description of clinical benefit in a confirmatory trial (or trials).
The review used the assessment aid. The application was granted priority review and regenerative medicine advanced therapy and orphan drug designations.
Kymriah was originally approved in 2017 for pediatric and young adult patients with a certain form of acute lymphoblastic leukemia. It comes with a boxed warning for cytokine release syndrome and neurological toxicities.
Tibsovo combo get expanded use for newly diagnosed acute myeloid leukemia
Servier’s Tibsovo (ivosidenib; tablets), in combination with azacytidine, has been granted a new indication for treating newly diagnosed acute myeloid leukemia (AML) with a susceptible IDH1 mutation, as detected by an FDA-approved test, in patients aged 75 years or older or who have comorbidities precluding them from receiving intensive induction chemotherapy.
Tibsovo, an IDH1 inhibitor, was originally approved in 2018 for treating adults with relapsed/refractory AML with IDH1 mutation. It is also used in patients with previously treated locally advanced/metastatic cholangiocarcinoma. The drug comes with a boxed warning for differentiation syndrome.
Approval of Tibsovo was based on findings from a randomized, multicenter, double-blind, placebo-controlled AGILE study in 146 patients from the indicated population randomized 1:1 to receive daily the Tibsovo or placebo, both combined with azacytidine, until disease progression, unacceptable toxicity, or having to have hematopoietic stem cell transplantation.
Event-free survival (EFS) events occurred in 65% of patients receiving the Tibsovo combination, compared with 84% of the placebo combination patients. (EFS was the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurred first.) Median overall survival was 24.0 months and 7.9 in the Tibsovo and placebo groups, respectively. Complete response was 47% and 15%, respectively. Median duration of CR was not estimable in the study drug group and 11.2 months in the placebo patients.
The review used the real-time oncology review (RTOR) pilot program and the assessment aid. The application was granted priority review and breakthrough designation.
Opdivo nabs new indication as combo therapy for esophageal SCC
Bristol-Myers Squibb’s Opdivo (nivolumab; injection), combined with the company’s Yervoy (ipilimumab) or fluoropyrimidine- and platinum-based chemotherapy, has been granted a new indication for treating patients with advanced/metastatic esophageal squamous cell carcinoma (ESCC).
The approval was based on findings from the randomized, active-controlled, open-label CHECKMATE-648 trial in 970 patients from the indicated population who were randomized 1:1:1 to receive Opdivo plus fluoropyrimidine- and platinum-based chemotherapy, Opdivo plus Yervoy, or fluoropyrimidine- and platinum-based chemotherapy alone. Findings demonstrated statistically significant improvements in overall survival for both Opdivo combinations, compared with chemotherapy alone.
This review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada, Israel’s Ministry of Health Pharmaceutical Administration, and Swissmedic. The review used the real-time oncology review pilot program and the assessment aid.
Opdivo was granted orphan drug designation for esophageal cancer.
The PD-1-blocking antibody was originally approved in 2014 for unresectable/metastatic melanoma and disease progression following Yervoy treatment. It has expanded indications for non-small cell lung cancer, renal cell carcinoma, and other cancers.



© 2022 Regulatory Affairs Professionals Society.

Tags: FDA, US

Discover more of what matters to you