Orphan drug designation granted in the EU

  • On November 13, 2017
  • Regulatory press release

Regulatory press release: ITB-Med´s application for Orphan Drug Designation for Siplizumab in the EU received positive opinion by the European Medicines Agency (EMA) on September 7, 2017, and final approval on October 16, 2017.

Commenting on the announcement, Erik Berglund, CEO of ITB-Med, said:

“Granting Siplizumab Orphan Drug Designation status is a very important regulatory milestone. The opinion that the regulatory agency EMA recognizes Siplizumab to be of significant benefit in the treatment of transplant patients attests to Siplizumab´s ability to revolutionize the field of transplantation by becoming the first therapy to enable long-term tolerance induction in transplant patients. The designation provides development and commercial incentives, including 10 years of market exclusivity, protocol assistance on the development of Siplizumab, including clinical studies, and certain exemptions from or reductions in regulatory fees. The outcome of the clinical trials with Siplizumab have been revolutionizing for the majority of the treated transplant patients, and we look very much forward to progress toward registration.”

Based on the positive opinion from the Committee for Orphan Medicinal Products (COMP), the European Commission grant Orphan Drug Designation to drugs intended for the treatment of life-threatening or chronically debilitating rare diseases where no therapeutic options are either authorized or where the drugs will be of significant benefit to those affected by the condition. Rare diseases are those defined as having a prevalence of no more than five in 10,000 people in Europe.

In the grounds for the opinion on Siplizumab´s orphan medicinal product designation, EMA wrote:

“…although satisfactory methods of treatment of the condition exist in the European Union, the sponsor has provided sufficient justification for the assumption that the medicinal product containing Siplizumab will be of significant benefit to those affected by the condition. The sponsor has provided preclinical and preliminary clinical data supporting long term tolerance of the transplant, with the potential to withdraw immunosuppression therapy. The Committee considered that this constitutes a clinically relevant advantage.

Thus, the requirement under Article 3(1)(b) of Regulation (EC) No 141/2000 on orphan medicinal products is fulfilled.”