Orphan Drugs Report August 2016

19 August 2016

In recent months, the orphan drug space has been under the spotlight more than usual with news from the FDA that the increasing number of orphan drug applications is impacting orphan drug review times. This is seen as proof that the Orphan Drug Act is having the desired effect of spurring drug developers into targeting rare diseases.

This report features major recent developments, important catalysts and current trends occurring in the space. Including articles on Rigel Pharmaceuticals oral spleen tyrosine kinase (SYK) inhibitor, fostamatinib, for adult chronic/persistent immune thrombocytopenia (ITP). On August 30th, the company announced fostamatinib in ITP “successfully met the primary endpoint in the first of two double blind Phase III clinical trials.” On April 26th, BioPharm Insight reported “Rigel Pharmaceuticals' fostamatinib disodium's Phase III trial in persistent or chronic ITP should succeed based on past Phase II efficacy signals, experts agreed. Some added design similarities between the two trials further underscores repeated promising and meaningful platelet count readouts.”

Included in this report is the complete article mentioned above as well as an additional article on the market potential of fostamatinib. The articles featured in this report are indicative of our forward-looking investigative reporting. By interviewing KOLs and industry experts, we’re able to give our subscribers unique insight 3-12 months ahead of industry catalysts.

Companies featured in this report include: Aradigm, Eli Lilly, Infinity Pharmaceuticals, Juno Therapeutics, Kite Pharma, Novartis, Ocera Therapeutics, ProQR Therapeutics, Proteon Therapeutics, Rigel Pharmaceuticals, Ultragenyx Pharmaceutical and Vertex Pharmaceuticals.

Read the report to learn more about the orphan drug space and get a taste for our investigative reporting. Then, request a complimentary trial to get real-time updates and experience BioPharm Insight.