On June 6, 2023 Amphera B.V., a late-stage biotechnology company developing MesoPher cell therapy to treat cancer, reported that the EMA Committee for Orphan Medicinal Products (COMP) has recommended the granting of orphan medicinal product designation for MesoPher in pancreatic cancer (Press release, Amphera, JUN 6, 2023, View Source [SID1234632539]).

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MesoPher is comprised of autologous dendritic cells loaded with PheraLys, a lysate of tumour cell lines. PheraLys contains a broad repertoire of tumour-associated antigens, many of which are present in pancreatic cancer and other cancers.

In December 20221, Amphera reported topline results from the phase II REACTIVE trial in patients with resected pancreatic cancer. MesoPher demonstrated a statistically significant 2-year Recurrence Free Survival of 60% and an excellent safety profile.

1) View Source

Notes to Editors

About EMA Orphan Designation

Source: View Source

"The EU offers incentives to encourage companies to research and develop medicines for rare diseases that otherwise would not be developed. To access these incentives, companies can apply for orphan designation for their medicine, provided certain criteria are met.

Criteria for orphan designation:

The medicine must treat, prevent, or diagnose a disease which is life-threatening or chronically debilitating, or it is unlikely that the medicine will generate sufficient returns to justify the investment needed for its development
The disease must not affect more than 5 in 10,000 people across the EU
No satisfactory method of diagnosis, prevention or treatment exists, or if such a method already exists, the medicine must be of significant additional benefit to those affected by the condition
During an orphan medicine’s research and development, the company can benefit from incentives such as scientific advice on study protocols, various fee reductions and access to EU grants. Orphan-designated medicines that eventually make it to the market, and for which it can be demonstrated that they maintain the criteria for the designation, are granted 10 years of market exclusivity.

Orphan designation is not an authorization: Not all orphan-designated medicines reach the marketing authorisation application stage. Those that do, are evaluated by EMA’s Committee for Medicinal Products for Human Use (CHMP) using the same strict safety and efficacy standards that apply to all medicines evaluated by EMA.