FDA grants orphan drug status to Syros’ myelodysplastic syndrome therapy

The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Syros Pharmaceuticals’ oral therapy, tamibarotene, to treat myelodysplastic syndrome (MDS).

Tamibarotene is a selective retinoic acid receptor alpha (RARα) agonist.

At present, oral therapy is being analysed along with azacitidine in the Phase III SELECT-MDS-1 clinical trial to treat RARA-positive individuals with recently detected higher-risk MDS (HR-MDS).

This ongoing trial is assessing the safety and efficacy of the combination treatment.

The company anticipates results from the pivotal trial in the fourth quarter of next year or the first quarter of 2024.

Furthermore, Syros plans to submit a potential new drug application in 2024.

Syros Pharmaceuticals chief medical officer David Roth said: “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.”

In addition, tamibarotene is analysed by the company along with azacitidine and venetoclax to treat RARA-positive people with recently diagnosed unfit acute myeloid leukaemia (AML).

The oral therapy has already obtained orphan drug designation for this indication.

Safety lead-in results from Phase II SELECT-AML-1 trial underway are anticipated in the second half of this year.

The Office of Orphan Drug Products of the regulatory agency provides orphan status to facilitate the development of therapeutics to treat rare diseases that affect less than 200,000 individuals in the country.

Orphan drug designation can offer specific benefits such as market exclusivity for seven years on obtaining drug approval, tax credits for eligible trials and an FDA application fee exemption.

Apart from tamibarotene, Syros’ pipeline comprises SY-2101, a new oral form of arsenic trioxide for acute promyelocytic leukaemia and SY-5609, an oral inhibitor of CDK7 for blood cancers and solid tumours.

In December 2019, Syros and Global Blood Therapeutics entered an agreement to discover, develop and market therapies for the treatment of two blood disorders.