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New FDA Orphan Drug Designation: Capricor Therapeutics And Duchenne Muscular Dystrophy

May 2, 2015

Capricor Therapeutics, a Beverly Hills, California-based clinical stage biotechnology company, is developing new treatments for heart disease. Capricor Therapeutics’ investigational product, CAP-1002, “aims to attenuate and potentially improve damage to the heart caused by a heart attack.” CAP-1002 is the company’s allogeneic Cardiosphere-Derived Cell (CDC) product. The company announces April 22nd that CAP-1002 receives FDA Orphan Drug Designation (ODD) for the treatment of cardiomyopathy associated with Duchenne Muscular Dystrophy (DMD).

Per Linda Marban, Ph.D., Chief Executive Officer of Capricor Therapeutics:

“Duchenne cardiomyopathy is a devastating complication of the disease that impacts virtually all those affected by the disease and is the leading cause of death in patients. We believe CDCs are uniquely suited to treat the cardiac component of DMD and could potentially be used in conjunction with any of the therapeutics currently in use or under investigation for the skeletal muscle pathology … We anticipate filing an IND with the FDA in the near future and hope to be treating patients with Duchenne later this year subject to regulatory approval. ”

FDA ODD Database Record For Capricor Therapeutics

Generic Name: Allogeneic cardiosphere-derived cells
Trade Name: n/a
Date Designated: 04-21-2015
Orphan Designation: Duchenne Muscular Dystrophy (DMD)
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Capricor, Inc. 8840 Wilshire Blvd. 2nd Floor Beverly HIlls, CA 90211

.

References

Capricor Therapeutics April 22, 2015 Press Release

Capricor Therapeutics Pipeline

CureDuchenne & Capricor Therapeutics January 2015 Webinar Recording.

Please Note: “Chemicals in Flasks by Joe Sullivan (Flickr) [CC-BY-2.0] | Wikimedia Commons.

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