Pharm-Olam Conducts Pivotal Orphan Disease Trial, Leads to First Therapeutic Approved for the Treatment for Acquired Thrombotic Thrombocytopenic Purpura
Pharm-Olam, a global, midsized CRO offering full-service clinical development solutions for oncology, infectious diseases and vaccines, and rare and orphan diseases, extends its congratulations to Ablynx, a Sanofi company, for its launch of Cablivi® (caplacizumab) in the German market for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). Pharm-Olam is proud to have contributed to the execution and completion of its pivotal Phase III study.
aTTP is a rare, life-threatening, autoimmune-based blood clotting disorder. The European Commission has granted marketing authorization for the drug. In the U.S., the FDA will give priority review to the drug, with a decision expected in early 2019.
Pharm-Olam, a leader in orphan and rare disease research, was contracted in April 2015 to conduct the pivotal Phase III HERCULES study. The company has also been contracted to run the three-year follow-up study to characterize the long-term impact of the drug and evaluate its repeated use.
Despite the challenges of enrolling patients into an orphan disease study, Pharm-Olam completed enrollment for the HERCULES study ahead of schedule — the team managed 92 sites across 16 countries to dose 145 patients.
“This achievement reinforces our experience and expertise in orphan diseases and blood disorders,” said Maria Jesus Seivane, project director at Pharm-Olam. “With more than 100 studies in this category, our team has become a well-suited partner to ensure success.”
“Helping to bring a new treatment to patients is something we can be immensely proud of,” said David L. Grange, CEO at Pharm-Olam. “We also want to congratulate Ablynx. This is not only the first therapeutic drug for the treatment of aTTP, it’s the first Nanobody®-based medicine on the market.”
Pharm-Olam is Helping Create a Healthier World as a global, midsized CRO that offers flexible, innovative and highly personalized clinical solutions to pharmaceutical, biotechnology and life science companies. Our team is well-known for producing quality results with reduced risk, costs and timelines in challenging international trials. Learn more about our full-service solutions, data protection services and expertise in oncology, infectious diseases and vaccines, rare and orphan diseases, pediatrics and general medicine at pharm-olam.com.