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What Is An RMAT? List of RMAT Designations (47)

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To date, 47 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 149 requests and issued 55.

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 Orca Bio for its experimental cell therapy, Orca-T. Orca-T is a formulation of hematopoietic stem cells and T cells provided to cancer patients who are eligible for a hematopoietic stem cell transplant (HSCT). Thus far, only two companies (Abeona Therapeutics and Athersys) have received two RMAT designations from the U.S. FDA.

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FDA Grants RMAT to MultiStem Cell Therapy for Treatment of ARDS

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ARDS program well-positioned for an expedited path to commercialization with RMAT and Fast Track designation

September 23, 2020; Cleveland — Athersys, a leading regenerative medicine company in late-stage clinical development, announced today that MultiStem® cell therapy was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for the acute respiratory distress syndrome (ARDS) program. [Read more…]

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…]

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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