Telix granted FDA orphan drug designation for bone marrow conditioning treatment

pharmafile | March 29, 2022 | News story | Manufacturing and Production  

The FDA has granted Orphan Drug Designation (ODD) for TLX66, for conditioning treatment prior to hematopoietic stem cell transplant (HSCT).

This ODD for TLX66 makes Telix qualified for various drug development incentives, which may include FDA administered market exclusivity for seven years, waived FDA prescription drug user fees, and tax credits for R&D and clinical development costs.

Bone marrow conditioning is performed prior to HSCT, a procedure where the patient’s bone marrow is cleared of cells and instead replaced with stem cells (cells that can develop into different types of cells), to encourage the production of new bone marrow that produces healthy blood cells. Traditional conditioning regimens are typically associated with morbidity and mortality from chemotherapy, limiting their use particlarly in paediatric and rare diseases.

TLX66 could potentially add to the depth of conditioning, removing additional disease-causing cells. The treatment’s potential to reduce the toxicity of existing conditioning regiments could also increase the number of patients that are eligible for transplant.

Telix CMO, Dr Colin Hayward said, “The granting of an Orphan Drug Designation by the FDA for TLX66, combined with recent encouraging data from prior studies in haematological malignancies and autoimmune disease provides a strong impetus to advance our development plans for TLX66. This treatment has potential application in a number of haematological cancers and rare diseases and potentially also in the future for conditioning for cell and gene therapies.”

TLX66 has also previously been granted ODD status by the EMA in Europe, for treatment in haemotopoietic stem cell transplantation. TLX66 has not received a marketing authorisation approval in any jurisdiction.

Lina Adams

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