Research & Development
Pharm-Olam Conducts Pivotal Orphan Disease Trial, Leads to First Therapeutic Approved for the Treatment for Acquired Thrombotic Thrombocytopenic Purpura
1 November 2018 - - US-based midsized CRO Pharm-Olam supported Ablynx, a Sanofi company, for its launch of Cablivi (caplacizumab) in the German market for the treatment of acquired thrombotic thrombocytopenic purpura, the company said.

Pharm-Olam 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 US, 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.


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