Tag Archives: global orphan drug market analysis

GS030 by GenSight Biologics receives orphan drug designation from EMA for Retinitis Pigmentosa

Gensight Biologics, a biopharma company specialized in developing innovative gene therapies for neurodegenerative retinal diseases and central nervous system disorders declared its leading candidate GS030 as Orphan drug designation, as approved by European Medicine Agency (EMA) for the treatment of retinitis pigmentosa. GS030 has also been classified as Advanced Therapy Medicinal product by EMA.

Currently, GS030 is undergoing preclinical development and laboratory practices are expected to initiate in September, 2017, regulatory toxicity study in non-human primates, prior to entering the clinic with a Phase I/II clinical trial in retinitis pigmentosa patients in Q3 2017, subject to toxicity results and future regulatory review.

The EMA’s designation of orphan status is granted to encourage new developments and therapies for rare diseases and disorders. Orphan status can be granted to those diseases that are rare. These incentives include protocol assistance, i.e. scientific advice specific for designated orphan medicines, and 10 years of market exclusivity once the medicine is on the market.


For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA announces orphan drug designation for TG-1101 for NMO and NMOSD

TG Therapeutics Inc., a biopharmaceutical company  focusing on developing and commercializing the medicines or treatments for B-cell malignancies and auto-immune diseases, developed TG-1101 (ublituximab), the company’s novel products has been designated as orphan drug by Food and Drug Association (FDA) for the treatment of Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD). Presently, there are no other FDA approved treatment options available in the market for NMO and NMSD. TG-1101 is the glycol-engineered anti-CD20 monoclonal antibody and a non-oncology indication providing additional protection.

Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD) are also called as Devic’s disease. It is an immune related chronic inflammatory disorder involving different parts of the central nervous system, primarily damaging optic nerve and spinal cord. The affected optic nerve and spinal cord causes loss of swelling and inflammation in eyes, vision and pain weakness or paralysis in the legs or arms, loss of sensation, and problems with bladder and bowel function. The disease rarely possess its progressive phase.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.



For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com