Caladrius Biosciences, Inc. (Nasdaq:CLBS) announced on 6/19/2018 that the U.S. Food and Drug Administration (“FDA”) has granted regenerative medicine advanced therapy (“RMAT”) designation to the Company’s late-stage CD34+ cell therapy program for the treatment of refractory angina.
The FDA grants the RMAT designation to regenerative medicine therapies intended to treat a serious condition for which preliminary clinical evidence indicates a potential to address unmet medical needs for that condition. The RMAT designation affords regenerative therapies the advantages of expedited development and review of marketing applications as are available to drugs that receive breakthrough therapy designation, including increased meeting opportunities, early interactions to discuss potential surrogate or intermediate endpoints, shortened biologics license application (“BLA”) review times and the potential of accelerated approval.
“We are delighted and encouraged that the FDA has recognized our CD34+ cell therapy program with an RMAT designation. Refractory angina is a serious condition with high morbidity and no known effective treatments. We look forward to working with the FDA to define a path to registration for our therapy with the aim of providing expeditious treatment to patients suffering from this condition,” said David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius.
Caladrius acquired an exclusive worldwide license to the late-stage CD34+ program from Shire plc in March of this year. The acquisition included the data set and regulatory filings for the CD34+ cell therapy program for the treatment of refractory angina. This includes manufacturing procedures, preclinical (in vivo and in vitro) and Phase 1, Phase 2 and Phase 3 clinical study data of CD34 cell therapy as a treatment for no-option refractory angina, along with the corresponding regulatory filings.
It is estimated that as many as one million people in the United States have chronic symptomatic coronary artery disease (often referred to as refractory angina) that is recalcitrant to medical therapy and not amenable to conventional revascularization procedures. Patients have reproducible lifestyle-limiting symptoms such as chest pain and shortness of breath, and are easily fatigued. These symptoms are often due to totally occluded coronary arteries or to diffuse coronary atherosclerosis that makes revascularization problematic. As the population ages and the incidence of diabetes mellitus increases, this clinical condition is expected to become more prevalent. Patients with this condition have significant morbidity and experience a lower quality of life.
Caladrius has two CD34 related products in development. CLBS12 is to promote the development and formation of new blood vessels and thereby increase blood flow to areas affected by ischemia. CLBS14 is intended to enhance the potency of the CD34 cells for repair and regeneration of cardiovascular tissue
