Janssen Seeks Expanded Use of DARZALEX®▼ (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Light Chain (AL) Amyloidosis

Janssen Seeks Expanded Use of DARZALEX^®▼ (daratumumab) Subcutaneous Formulation for the Treatment of Patients with Light Chain (AL) Amyloidosis

AL amyloidosis is a rare, multi-system disease with a high unmet medical need as there are currently no approved therapies[i]^,[ii]

BEERSE, BELGIUM, 05 November, 2020 –  The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a Type II variation application to the European Medicines Agency (EMA) seeking approval to expand the use of the DARZALEX^®▼ (daratumumab) subcutaneous (SC) formulation to include the treatment of patients with light chain (AL) amyloidosis.

AL amyloidosis is a rare and potentially fatal disease that occurs when an insoluble protein called amyloid builds up in tissues and organs, interfering with healthy tissue and organ function.[iii]^,[iv] There are currently no therapeutic options approved by regulatory bodies such as the EMA or the U.S. Food and Drug Administration (FDA) for treatment of the disease.1^,2

The submission is based on data from the Phase 3 ANDROMEDA study, presented during the 2020 European Hematology Association (EHA) Annual Congress. The study evaluated the efficacy and safety of daratumumab SC in combination with

bortezomib, cyclophosphamide, and dexamethasone (D-VCd) compared with bortezomib, cyclophosphamide, and dexamethasone (VCd) alone in the treatment of patients with AL amyloidosis. The study data showed a significantly higher haematologic complete response rate for patients with the addition of daratumumab compared with patients treated with VCd alone.[v] Overall, the safety profile of D-VCd demonstrated a consistent safety profile compared to the VCd regimen and the known safety profile of daratumumab.5

“The current management of AL amyloidosis focuses on slowing production of amyloid protein and controlling symptoms; however, there are no EMA-approved therapies for this difficult-to-treat, rare disease,” said Dr Catherine Taylor, VP, Medical Affairs Therapeutic Area Strategy, Europe, Middle East and Africa (EMEA), Janssen-Cilag Ltd. Middle East. “If approved, adding daratumumab to this combination could address a significant unmet need and offer new hope to patients with AL amyloidosis, who have poor prognoses and have long been waiting for therapeutic options.”

“Daratumumab is an important foundational therapy in the treatment of multiple myeloma and now, on the basis of the ANDROMEDA study results, has shown that it can improve outcomes in a related plasma cell disorder, AL amyloidosis,” said Craig Tendler, M.D., Vice President, Late Development and Global Medical Affairs, Janssen Research & Development, LLC. “We are excited about the potential for daratumumab, as part of a regimen for newly diagnosed patients with AL amyloidosis to alter the poor prognosis of their disease and reduce organ damage, which is an unfortunate life-threatening complication of this serious disease.”

In September 2020, Janssen submitted a supplemental Biologics Licence Application (sBLA) to the U.S. FDA seeking approval of the subcutaneous formulation of daratumumab for the treatment of patients with AL amyloidosis.[vi]

[i] Desport E, Bridoux F, Sirac C, Delbes S, Bender S, Fernandez B, Quellard N, Lacombe C, Goujon JM, Lavergne D, Abraham J. AL amyloidosis. Orphanet journal of rare diseases. 2012 Dec;7(1):54

[ii] Merlini G, Comenzo RL, Seldin DC, Wechalekar A, Gertz MA. Immunoglobulin light chain amyloidosis. Expert review of hematology. 2014 Feb 1;7(1):143-56.

[iii] Stat Pearls. Amyloidosis. Available at: https://www.ncbi.nlm.nih.gov/books/NBK470285/. Last accessed: November 2020.

[iv] Mayo Clinic. Amyloidosis Overview: Symptoms and Causes. Available at: https://www.mayoclinic.org/diseases-conditions/amyloidosis/symptoms-causes/syc-20353178 Last accessed: November 2020.

[v] Kastritis, E. et al. Subcutaneous Daratumumab + Cyclophosphamide, Bortezomib, and Dexamethasone (CyBorD) in Patients with Newly Diagnosed Light Chain (AL) Amyloidosis: Primary Results from the Phase 3 ANDROMEDA Study [LBA]. To be presented at European Hematology Association 2020 Annual Congress.

[vi] Janssen Pharmaceutical Companies of Johnson & Johnson. Janssen Submits Application Seeking U.S. FDA Approval of DARZALEX FASPRO™ (daratumumab and hyaluronidase-fihj) for the Treatment of Patients with Light Chain (AL) Amyloidosis. Available at: https://www.prnewswire.com/news-releases/janssen-submits-application-seeking-us-fda-approval-of-darzalex-faspro-daratumumab-and-hyaluronidase-fihj-for-the-treatment-of-patients-with-light-chain-al-amyloidosis-301126998.html Last accessed: November 2020.