FDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers

The U.S. Food and Drug Administration granted Fast Track designation to Syros Pharmaceuticals’ tamibarotene for the treatment of higher-risk myelodysplastic syndrome.

Photo: David Roth, chief medical officer of Syros Pharmaceuticals

Myelodysplastic syndromes (MDS) are a rare group of blood cancers characterized by abnormal development of blood cells within the bone marrow. Approximately 15,000 people are diagnosed with MDS in the United States each year, and no new treatments have been approved in 14 years. The average survival rate for those with lower-risk MDS is six years and approximately 18 months for those with higher-risk MDS.

Individuals with MDS have abnormally low blood cell levels with symptoms including dizziness, fatigue, weakness, shortness of breath, bruising and bleeding, frequent infections, and headaches. In some cases, MDS may progress to bone marrow failure or an acute leukemia. The exact cause of MDS is unknown. It sometimes runs in families, but no disease-causing gene has been identified. Treatment depends on the affected individual’s age, general health, and type of MDS and may include red cell and/or platelet transfusions and antibiotics.

Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine for the treatment of newly diagnosed HR-MDS patients with RARA gene overexpression.

Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. The purpose is to help speed development of new drugs, making them available to the patient earlier. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the therapeutic candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.

Syros is evaluating tamibarotene in combination with azacitidine in newly diagnosed HR-MDS patients with RARA overexpression in the ongoing SELECT MDS-1 phase 3 trial. This randomized, double-blind, placebo-controlled study is intended to enroll 190 patients. Syros currently has over 75 clinical sites open for recruitment in 12 countries. Syros expects to complete patient enrollment in SELECT-MDS-1 in the fourth quarter of 2023, with pivotal data expected in the third quarter of 2024.

Syros is also evaluating tamibarotene in combination with venetoclax and azacitidine in newly diagnosed unfit AML patients with RARA overexpression, with initial data from the randomized portion of the SELECT-AML-1 phase 2 trial expected in the fourth quarter of 2023 and additional data in 2024.

“Receipt of Fast Track designation for tamibarotene underscores both the potential of tamibarotene and the unmet need for HR-MDS patients, who have a poor prognosis due to the progressive nature of the disease,” said David Roth, chief medical officer of Syros Pharmaceuticals. “No new therapies beyond hypomethylating agents have been approved since 2006, and approximately half of all patients diagnosed with HR-MDS patients ultimately progress to AML.”

Author: Rare Daily Staff