FDA orphan drug designation for treatment of rare blood cancer
pharmafile | February 3, 2022 | News story | Business Services |
The FDA has granted orphan drug designation (ODD) to tamibarotene for the treatment of myelodysplastic syndrome (MDS). The Office of Orphan Drugs Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the US.
Myelodysplastic syndromes (MDS) are a type of rare blood cancer involving poorly formed or dysfunctional blood cells, and too few healthy blood cells. As the condition progresses, bone marrow is gradually taken over by immature blood cells, make the number of functional cells entering the bloodstream lower and lower. In some people it can develop into a type of leukaemia called acute myeloid leukaemia (AML).
Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARa) agonist, is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 Phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS (HR-MDS).
Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials, and an exemption from FDA application fees.
“The FDA’s orphan drug designation is an important milestone in the development of tamibarotene as a treatment for MDS,” said David A. Roth, M.D., Syros’ Chief Medical Officer. “We believe tamibarotene’s novel mechanism of action, promising clinical activity data, oral delivery, and favourable tolerability profile supports a potential new option for the approximately 30% of HR-MDS patients who are RARA-positive. We are focused on developing the first potential therapy for a targeted population in HR-MDS as we continue to advance our ongoing SELECT-MDS-1 pivotal trial.”
The ongoing SELECT-MDS-1 Phase III clinical trial is evaluating the safety and efficacy of tamibarotene in combination with azacytidine for RARA-positive patients with newly diagnosed HR-MDS.
Syros Pharmaceuticals is a leader in the development of medicines that control the expression of genes, aiming to develop medicines that provide a significant benefit for patients with diseases that have eluded other genomic-based approaches.
Data from the SELECT-MDS-1 trial are expected in the fourth quarter of 2023, or the first quarter of 2024. A potential new drug application filing is expected in 2024.
Ana Ovey
