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CRISPR Therapeutics and Vertex Pharmaceuticals Win RMAT for CTX001™

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  • CRISPR Therapeutics and Vertex Pharmaceuticals Have Been Awarded an FDA RMAT Designation for CTX001™ in the Treatment of Severe Hemoglobinopathies

  • CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the European Medicines Agency (EMA) for sickle cell disease and transfusion-dependent beta thalassemia

ZUG, Switzerland and Cambridge, MA, May 11, 2020 — CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. FDA RMAT designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). [Read more…]

What Is An RMAT? List of RMAT Designations (44)

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To date, 44 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 139 requests and issued 48.

Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.

The most recent addition to this list was the RMAT jointly awarded to CRISPR Therapeutics and Vertex Pharmaceuticals for CTX001™ in the treatment of severe hemoglobinopathies.

Prior to that, it was Immunicum’s ilixadencel on May 6, 2020. Ilixadencel consists of activated allogeneic dendritic cells used as an immune primer for the treatment of metastatic Renal Cell Carcinoma (mRCC).

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Immunicum Wins RMAT for Ilixadencel, Its Dendritic Cell Immunotherapy Against Kidney Cancer

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  • Immunicum AB has received an FDA RMAT for Ilixadencel, its allogeneic dendritic cell immunotherapy for the treatment of metastatic renal cell carcinoma (mRCC).

  • Ilixadencel is the 43rd FDA RMAT to be announced.

Immunicum AB (publ; IMMU.ST) has received and FDA Regenerative Medicine Advanced Therapy (RMAT) designation for the Company’s lead product candidate, ilixadencel, a cell-based, off-the-shelf immune primer for the treatment of metastatic Renal Cell Carcinoma (mRCC). [Read more…]

Novartis’ Kymriah® Wins Latest FDA RMAT (42 Announced)

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Novartis Kymriah® receives FDA Regenerative Medicine Advanced Therapy designation in follicular lymphoma.

  • If approved, relapsed or refractory (r/r) follicular lymphoma would become the third B-cell malignancy indication for Kymriah, joining approvals in children and young adults with r/r ALL, and adults with r/r DLBCL
  • The Regenerative Medicine Advanced Therapy (RMAT) designation reflects the unmet need for patients with r/r follicular lymphoma
  • US regulatory filing for Kymriah in r/r follicular lymphoma anticipated in 2021

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TissueTech Receives RMAT from FDA for Human Umbilical Cord Product, TTAX02

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RMAT designation reinforces clinical significance of investigational biologic product TTAX02 used during in-utero fetal surgical repair of spina bifida.

MIAMI, April 16, 2020 — TissueTech, Inc., the pioneer in the development and clinical application of regenerative human birth tissue products, announced today that the U.S. Food and Drug Administration (FDA) has granted their cryopreserved human umbilical cord investigational biologic product TTAX02 RMAT designation for the treatment of spina bifida in-utero.
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