Three regenerative medicines are designated as Orphan Drug Designation (ODD) by Minister of Health and Labor Welfare on November 13, 2019. ODD has merits of priority review ( 9months review than 12 months) and longer exclusivity ( max 10 years than 6 years)
- NSR-REP1 (Biogen, Origin came from Nightstar therapeutics)
- bb2121 (Celgene Corporation and bluebird bio, Inc.)
- HLCM051 (Healios and Athersys)
NSR-REP1 (Biogen, Origin came from Nightstar therapeutics)
NSR-REP1 for the treatment of choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder, which leads to blindness and has no approved treatments. CHM primarily affects males and is caused by loss of function in the CHM gene which encodes the Rab escort protein-1 (REP-1). The REP-1 protein plays a role in intracellular protein trafficking, and loss of function in the CHM gene leads to abnormal intracellular protein trafficking and impaired elimination of waste products from the retinal pigment epithelium and photoreceptors. Initially, patients with CHM experience poor night vision, and over time progressive visual loss ultimately leads to complete blindness.
http://investors.biogen.com/news-releases/news-release-details/biogen-announces-agreement-acquire-nightstar-therapeutics
bb2121 (Celgene Corporation and bluebird bio, Inc.)
a chimeric antigen receptor T-cell (CAR-T) therapy targeting b-cell maturation antigen (BCMA) in previously treated patients with multiple myeloma
https://ir.celgene.com/press-releases/press-release-details/2017/Celgene-Corporation-and-bluebird-bio-Announce-bb2121-Anti-BCMA-CAR-T-Cell-Therapy-Has-Been-Granted-Breakthrough-Therapy-Designation-from-FDA-and-Prime-Eligibility-from-EMA-for-Relapsed-and-Refractory-Multiple-Myeloma/default.aspx
HLCM051 (Healios and Athersys)
MultiStem® cell therapy treatment of patients who suffer from acute respiratory distress syndrome (“ARDS”)
https://www.athersys.com/investors/press-releases/press-release-details/2019/HEALIOS-KK-Announces-Enrollment-of-First-Patient-in-Japan-in-the-ONE-BRIDGE-Study-of-MultiStem-Treatment-for-Acute-Respiratory-Distress-Syndrome/default.aspx