Background Analysis: US FDA Advisory Committee to Review Mesoblast’s Proposed Stem Cell Therapy, Ryoncil – AUG 13, 2020 (ODAC)

Announcement

The US FDA has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting for Thursday, August 13, 2020. The committee will discuss a biologics license application (BLA) for Ryoncil (remestemcel-L), submitted by Mesoblast, Inc (Mesoblast), for the proposed use to treat steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients. Ryoncil consists of a suspension of ex-vivo culture-expanded adult human mesenchymal stromal cells for intravenous infusion,

Agenda

The morning session will discuss issues related to the characterization and critical quality attributes of remestemcel-L as they relate to clinical effectiveness.  The afternoon session will discuss results from clinical trials included in the BLA.

Clinical Background

The BLA is primarily based on the results of an open-label phase 3 trial in 55 children with SR-aGVHD, 89% of whom had Grade C/D disease (www.clinicaltrials.gov identifier for this trial: NCT02336230). According to the company, the treatment significantly improved the day 28 overall response rate (OR) compared with the historical control OR value of 45% (70.4% versus 45%, P = .0003). In additional, the company says that overall survival was 74.1% at day 100 and 68.5% at day 180. With respect to safety, the company has reported that the treatment was well tolerated with no identified infusion-related toxicities or other safety concerns.

Regarding manufacturing processes for Ryoncil, the company claims that technical properties inherent with its mesenchymal lineage cells allow for scalable expansion to produce “off-the-shelf” commercial quantities with batch to batch consistency and reproducibility. The company also says that proprietary formulations, advances in the development of 3D bioreactor technology and automation may deliver step-changes improvement in product yield.

Regulatory Background

US Regulatory Background

September 30, 2020 – PDUFA date

February 2, 2020 – Mesoblast announced the completion of their rolling BLA submission (BLA #125706).

What’s Next?

Tarius will send a Briefing Summary after briefing materials are posted to FDA’s website (typically within 2 days of the meeting). This report will provide a summary of the FDA and the Sponsor’s briefing materials.

Tarius will send a Results Wire soon after the meeting. This report will include the voting outcomes, if applicable, and key outcomes of the discussion.

METADATA: Sponsor: Mesoblast, Inc. Drug Name: Ryoncil (remestemcel-L) Drug Class: stem cell therapy Indication: steroid-refractory acute graft-versus-host disease

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DISCLAIMER: The information in this document is for informational purposes only. The SAC Tracker Background Analysis contains information from publicly available sources, including FDA, sponsor, scientific, and clinical websites. Tarius A/S assumes no liability for any inaccurate or incomplete information, or for any actions taken in reliance thereon. © Tarius A/S. All rights reserved.