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Chugai preps filings for IL-6 drug in rare disease, chasing Soliris fiercebiotech
December 20, 2018
Chugai has the positive phase 3 data it needs to file IL-6-targeting antibody satralizumab for approval in neuromyelitis optica spectrum disorder (NMOSD), a rare autoimmune disease, next year.
STEM Healthcare appoints Jason McKenna as CEO pharmaphorum
December 19, 2018
STEM Healthcare has promoted Jason McKenna (pictured above) to CEO, a role he takes over from the benchmarking company’s co-founder Rob Wood.
Sutimlimab shows promise for hard-to-treat, rare blood disorder worldpharmanews
December 19, 2018
In a first-in-human clinical trial reported today in Blood, the investigational drug sutimlimab appeared to be effective in treating cold agglutinin disease, a rare chronic blood disorder for which there are currently no approved treatments.
EUSA Pharma Appoints President of US Operations americanpharmaceuticalreview
December 06, 2018
EUSA Pharma, a global biopharmaceutical company with a focus on oncology and rare disease, has announced the appointment of Jeffrey S. Hackman as President.....
FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue firstwordpharma
December 02, 2018
The Food and Drug Administration designates some drugs as "orphan drugs," which allows manufacturers to obtain incentives (like tax breaks) for developing drugs that treat rare diseases. But we found that FDA reviewers don’t always include all the require
Quanticate Links Up with Lancaster University contractpharma
November 18, 2018
The partnership will see Quanticate support research into optimizing clinical trial design for rare disease and the role of response-adaptive randomisation. STOR-i was established in 2010 as an Engineering and Physical Sciences Resaearch Council EPSRC Cen
Quanticate Links Up with Lancaster University contractpharma
November 18, 2018
The partnership will see Quanticate support research into optimizing clinical trial design for rare disease and the role of response-adaptive randomisation. STOR-i was established in 2010 as an Engineering and Physical Sciences Resaearch Council EPSRC Cen
Included in the Medical Insurance of China, A Comparison of Prices of Anticancer Drugs in and outside China 1℃
October 24, 2018
Maximum Price Reduction by 74%!
CANbridge Pharmaceutical and WuXi Biologics Enter into Strategic Partnership for Rare Disease Therapeutics en-cphi.cn
October 24, 2018
CANbridge Plans to Submit First Rare Disease IND in China in 2019
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease worldpharmanews
August 13, 2018
The U.S. Food and Drug Administration today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

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