Pharmaceutical Industry Information Portal

FDA grants Regenerative Medicine Advanced Therapy designation to Direct Biologics’ extracellular vesicle drug for Covid-19

Direct Biologics, an innovative biotechnology company with a groundbreaking extracellular vesicle (EV) platform drug technology, announced that the U.S. Food and Drug Administration has awarded their EV drug product ExoFlo with a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Acute Respiratory Distress Syndrome (ARDS) associated with COVID-19.

The RMAT program is designed to expedite the approval of promising regenerative medical products in the US that demonstrate clinical evidence indicating the ability to address an unmet medical need for a serious life-threatening disease or condition.

Under the RMAT designation, the FDA provides intensive guidance on drug development and post-market requirements through early and frequent interactions. Additionally, an RMAT confers eligibility for accelerated approval and priority review of biologics licensing applications (BLA).

Mark Adams, Chief Executive Officer, said:

After intensively reviewing our preclinical data, manufacturing processes, and clinical data from our Phase II multicenter, double blinded, placebo controlled randomized clinical trial, the FDA has recognized ExoFlo as a lifesaving treatment for patients suffering from Acute Respiratory Distress Syndrome due to severe or critical COVID-19,The additional attention, resources, and regulatory benefits provided by an RMAT designation demonstrate that the FDA views ExoFlo as a product that can significantly enhance the standard of care for the thousands still dying from ARDS every week in the US.

Joe Schmidt, President, said:

We are very pleased that the FDA has recognized the lifesaving potential of our platform drug technology ExoFlo. The RMAT has provided a pathway to expedite our drug development to achieve a BLA in the shortest possible time. I am very proud of our team. Everyone has been working around the clock for years in our mission to save human lives taken by a disease that lacks treatment options, both in the US and abroad. We are grateful for the opportunity to accelerate development of ExoFlo under the RMAT designation as it leads us closer to our goal of bringing our life saving drug to patients who desperately need it.

ExoFlo is an acellular human bone marrow mesenchymal stem cell (MSC) derived EV product produced with a proprietary EV platform technology by Direct Biologics, LLC. Containing signaling molecules from bone marrow MSCs, ExoFlo harnesses the anti-inflammatory and regenerative properties of bone marrow MSCs without the cost, complexity, and limitations of scale associated with stem cell transplantation.


Expert Articles