European Agency Grants Orphan Medicinal Status to Experimental Therapy SB-525 for Hemophilia A

The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer.

The announcement coincides with the opening of patient enrollment for a Phase 1/2 clinical trial (NCT03061201) evaluating the safety and tolerability of SB-525 in adults with severe hemophilia A. Early data from this study is expected in late 2017 or early 2018, according to a Sangamo press release.

SB-525 was designed to reduce or eliminate the need for coagulation factor VIII replacement therapy by getting cells to naturally produce the missing protein. It works by carrying and delivering genetic information to liver cells where coagulation factors are produced using a modified, harmless form of the adeno-associated virus.

The development of SB-525 is part of a global collaboration agreement between Sangamo and Pfizer to develop and market gene therapy programs for hemophilia A. The partnership was announced in May.

The gene therapy candidate was designated an investigational new drug (IND) by the U.S. Food and Drug Administration (FDA) in January and was granted orphan drug status in May for the treatment of hemophilia A.

Gene therapy potentially could transform the treatment of hemophilia as a highly specialized, one-time therapy that targets the root cause of the disease. The technology involves introducing genetic material into the patient’s body to deliver a “correct” copy of a certain gene to compensate for a defective one.

When the collaboration agreement was announced in May, Mikael Dolsten, PhD, president of worldwide research and development at Pfizer, said the company believes SB-525 “has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of factor VIII protein with a single administration treatment.”

The EMA’s orphan medicinal product designation is granted only to medicines that are intended for the treatment, prevention or diagnosis of life-threatening or chronic conditions that affect fewer than five in 10,000 people within the European Union (EU).

The designation is meant to expedite the development and commercialization of orphan medicines, which include access to EU centralized authorizations and the potential for market exclusivity for up to 10 years.