Crunch Time At EMA For Luspatercept And Glasdegib
Executive Summary
Eight new marketing applications including ones from BMS/Acceleron Pharma and Pfizer are up for an opinion at the April meeting of the European Medicines Agency’s drug evaluation committee, the CHMP.
The European Medicines Agency is this week deciding whether to recommend pan-EU marketing approval for eight new drugs including luspatercept, Bristol-Myers Squibb Co. and Acceleron Pharma’s treatment for myelodysplastic syndromes (MSD) and beta-thalassemia that this month secured a second key approval in the US.
Luspatercept last November became the first US Food and Drug Administration-approved treatment for anemia in beta thalassemia (US brand name Reblozyl). The US regulator on April 3 approved an extended indication for the product, which according to BMS and Acceleron, marked the first new FDA-approved treatment option in over a decade for patients with MDS who require red blood cell (RBC) transfusions and have failed an erythropoiesis stimulating agent. (Also see "Keeping Track: Oncology Submissions From Blueprint, Y-mAbs, BMS/bluebird; Approvals For BMS’ Reblozyl, LFB’s SevenFact" - Pink Sheet, 5 Apr, 2020.)
Pfizer’s glasdegib, for the treatment of newly diagnosed de novo or secondary acute myeloid leukemia (AML), is also one of the eight. Glasdegib, an oral hedgehog pathway inhibitor, was approved in the US for AML in 2018 under the brand name Daurismo.
The EU marketing authorization applications (MAAs) for luspatercept and glasdegib – both of which have orphan status – are listed as being up for an opinion on the agenda of the latest monthly meeting of the EMA’s drug evaluation committee, the CHMP, which is taking place on 28-30 April. The indication being sought for luspatercept is for both the treatment of adult patients with very low- to intermediate-risk MDS associated-anemia and the treatment of adults with beta-thalassemia-associated anemia who require RBC transfusions.
The other products up for an opinion by the CHMP this week include:
-
paliperidone – Janssen Pharmaceutical’s treatment for schizophrenia that the company filed with the EMA under an informed consent application. In an informed consent application, reference is made to an authorized medicine where the marketing authorization holder of the reference medicine has given consent to the use of their dossier in the application procedure.
-
two versions of indacaterol/glycopyrronium/mometasone, both for treating asthma and for reducing asthma exacerbations. Notably, two asthma products made by Novartis received a positive marketing recommendation during the CHMP’s meeting in March – Atectura Breezhaler and Bemrist Breezhaler (both indacaterol/mometasone furoate). (Also see "Zolgensma and Seven Other Products Get CHMP Thumbs Up" - Pink Sheet, 27 Mar, 2020.)
In addition, the CHMP is due to adopt an opinion on the biosimilar insulin apart, for diabetes, and two generics – cabazitaxel, for treating adult patients with metastatic castration resistant prostate cancer previously treated with a docetaxel-containing regimen, and fingolimod, for treating multiple sclerosis.
Oral Explanations
Fingolimod is also listed in the CHMP agenda as being scheduled for an oral explanation meeting. These meetings are held when the CHMP wants sponsors to address concerns that remain at the final stages of the regulatory review process.
Oral explanation meetings are also scheduled for two other products in the pre-authorization setting: Novartis’s advanced breast cancer drug, Piqray (alpelisib), and the generic erlotinib, for treating lung and pancreatic cancers. (Also see "Novartis Faces The Music Over EU Piqray Review" - Pink Sheet, 29 Apr, 2020.)
In addition, an oral explanation meeting was due on 28 April for D&A Pharma regarding its alcohol dependence treatment, Hopveus (sodium oxybate), which the CHMP last October said should not be recommended for approval. D&A had asked the CHMP to re-examine its negative opinion.
This is the second time D&A has been listed in a CHMP meeting agenda as being scheduled for an oral explanation regarding the negative opinion for Hopveus. (Also see "Novartis, D&A Pharma Prepare For High-Stake EMA Meetings" - Pink Sheet, 25 Feb, 2020.) The latest oral explanation meeting was to hear a report from an ad-hoc expert group meeting held on 6 April and include patient representative participation.
The Outcomes
The key outcomes of April’s CHMP meeting will be made public on 1 May.
CHMP opinions are sent to the commission, which has 67 days to take a legally binding decision valid throughout the EU.