Tag Archives: global oprhan drug pipeline

FDA grants orphan drug designation to Nintedanib for Systemic Sclerosis

Boehringer Ingelheim’s nintedanib has been approved as orphan drug for the treatment of systemic sclerosis (SSc), also called scleroderma. FDA has taken an inevitable step forward in prevention and cure of Scleroderma, including associated interstitial lung disease.

SSc is a rare disease causing scratches over skin, lungs and other organs, resulting into disability. Almost 2 million of people are suffering from SSC, out of which, 90% patients have been diagnosed with scarring in lungs. SSC holds a record of 35% of deaths caused by all the disease-related deaths in the world.

Nintedanib’s safety and efficacy in treating SSc is being evaluated in SENSCIS, the largest trial to date in this disease area. The randomized, double-blind, placebo-controlled study is designed to measure the nintedanib 150 mg which is taken twice daily for over 52 weeks up to 100 weeks in patients with SSc-ILD. The primary endpoint is the annual rate of decline in forced vital capacity, while secondary endpoints include the absolute change from baseline in the modified Rodnan Skin Score.

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

FDA grants orphan drug status to Singh Biotechnology

Singh Biotechnology, a pharmaceutical company, announces that its SBT-100, its novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (sdAb) SBT-100, has been granted as orphan drug designation by US Food and Drug Administration (FDA) for the treatment of pancreatic Cancer. It has been investigated that elevated STAT3 is linked with poor prognosis of solid tumors, including tumor of the pancreas. It can be inhibited either by directly targeting the intracellular protein or indirectly, by targeting the upstream regulators of the STAT3 pathway.

 The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020