StemCyte's HPC Cord Blood Receives FDA RMAT Designation for Long COVID-19 Treatment
Key Insights
StemCyte (search)'s HPC Cord Blood therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for treating Long COVID-19 syndrome (search).
The RMAT designation allows StemCyte (search) to expedite the development of HPC Cord Blood, including priority review and accelerated approval opportunities.
A Phase II clinical trial of HPC Cord Blood for Long COVID-19 was successfully completed, with results expected to be unblinded soon, informing a planned Phase III trial.
StemCyte (search)'s Hematopoietic Progenitor Cell Cord Blood therapy (search) (HPC Cord Blood) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of Long COVID-19 syndrome (search). This designation, announced on October 3, 2024, positions StemCyte among a select few cell therapy companies in the United States to achieve this milestone.
Addressing Unmet Needs in Long COVID-19
Long COVID-19 syndrome (search) presents a significant and persistent health challenge, with many patients experiencing debilitating symptoms for which there are currently no effective treatments. StemCyte (search)'s HPC Cord Blood is the first cord blood cell therapy to receive FDA approval for a Phase II clinical trial specifically targeting Long COVID-19. The RMAT designation, largely based on positive outcomes from this Phase II trial, enables StemCyte to work closely with the FDA to accelerate development, gain priority review, and pursue accelerated approval pathways for future indications.
Clinical Trial Progress and Future Plans
The Phase II clinical trial of HPC Cord Blood was completed within eight months in the United States, achieving full patient enrollment. StemCyte (search) anticipates unblinding the results soon. With the RMAT designation, StemCyte is now planning a Phase III clinical trial under FDA guidance. The company has also made significant progress on its Biologics License Application (BLA) for the HPC Cord Blood product (RegeneCyte), with expectations of receiving FDA approval by the end of the year to utilize umbilical cord blood-derived hematopoietic stem cells for treating cancers (search), blood disorders (search), and immune deficiencies (search).
Significance of RMAT Designation
The FDA's RMAT designation is reserved for products demonstrating breakthrough therapeutic potential and addressing significant unmet medical needs. As of June 2024, the FDA reported 279 RMAT applications submitted worldwide, with successful approvals including CAR-T therapies like Kymriah®, Abecma®, and Breyanzi®. StemCyte (search) highlights its position as the only cell therapy company to achieve this designation for its HPC Cord Blood in the Long Covid-19 indication.
StemCyte's Focus on Regenerative Medicine
StemCyte (search) is focused on regenerative medicine, developing innovative therapies utilizing cord blood. The company aims to bring advanced cellular therapies to market, improving health outcomes for patients globally.